type: Other Identifier

title: A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.

summary:
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:

Stage 1 is a dose-escalation study.
Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.

All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).

Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.

ReferenceCitation: Sebastien B, Cheverton P, Magnin C, Aouni J, Castan R. Development and validation of a predictive model to guide the use of plerixafor in pediatric population. Bone Marrow Transplant. 2022 Sep 26. doi: 10.1038/s41409-022-01831-2. [Epub ahead of print]

ReferenceCitation: Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rossler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Krivan G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Netherlands
Hungary
France
Israel
Belgium
Germany
Czechia

location_facility:
Investigational Site Number 21
Investigational Site Number 92
Investigational Site Number 42
Investigational Site Number 13
Investigational Site Number 83
Investigational Site Number 51
Investigational Site Number 93
Investigational Site Number 26
Investigational Site Number 72
Investigational Site Number 94
Investigational Site Number 82
Investigational Site Number 35
Investigational Site Number 33
Investigational Site Number 31
Investigational Site Number 61
Investigational Site Number 91
Investigational Site Number 24
Investigational Site Number 71
Investigational Site Number 84
Investigational Site Number 23
Investigational Site Number 11
Investigational Site Number 81
Investigational Site Number 36
Investigational Site Number 22
Investigational Site Number 34
Investigational Site Number 85
Investigational Site Number 43


title: European Ankylosing Spondylitis Infliximab Cohort (EASIC) Follow up Registration Study

acronym: EASIC registry

summary: Long term data on efficacy and safety of anti-TNF treatment with infliximab in patients with ankylosing spondylitis (AS) beyond 5 years is lacking. These data are important because patients with AS usually are younger and withdrawal of anti-TNF therapy in these patients almost always leads to a disease relapse. Furthermore it is still unclear whether long term anti-TNF treatment in AS patients can inhibit radiographic progression. Patients who participated in the EASIC and the DIKAS trial respectively who were treated with infliximab within these studies for 7 and 10 years respectively are followed up by using clinical outcome parameters every 6 months assessing efficacy and safety of long term treatment. Furthermore radiographs of the spine, if done for clinical indication, are analyzed. It is hypothesized that anti-TNF treatment with infliximab is effective and safe over a time period of 9 and 12 years respectively and that long term anti-TNF therapy may inhibit radiographic progression of the spine.

status: Unknown status

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Zochling J, van der Heijde D, Burgos-Vargas R, Collantes E, Davis JC Jr, Dijkmans B, Dougados M, Géher P, Inman RD, Khan MA, Kvien TK, Leirisalo-Repo M, Olivieri I, Pavelka K, Sieper J, Stucki G, Sturrock RD, van der Linden S, Wendling D, Böhm H, van Royen BJ, Braun J; 'ASsessment in AS' international working group; European League Against Rheumatism. ASAS/EULAR recommendations for the management of ankylosing spondylitis. Ann Rheum Dis. 2006 Apr;65(4):442-52. Epub 2005 Aug 26.

ReferenceCitation: Baraliakos X, Listing J, Brandt J, Zink A, Alten R, Burmester G, Gromnica-Ihle E, Kellner H, Schneider M, Sörensen H, Zeidler H, Rudwaleit M, Sieper J, Braun J. Clinical response to discontinuation of anti-TNF therapy in patients with ankylosing spondylitis after 3 years of continuous treatment with infliximab. Arthritis Res Ther. 2005;7(3):R439-44. Epub 2005 Feb 21. Erratum in: Arthritis Res Ther. 2005;7(3):113. Zink, Angela [added];Alten, Rieke [added]; Burmester, Gerd [added]; Gromnica-Ihle, Erika [added]; Kellner, Herbert [added]; Schneider, Matthias [added]; Sörensen, Helmut [added]; Zeidler, Henning [added]; Rudwaleit, Martin [added].

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Alten R, Burmester G, Gromnica-Ihle E, Haibel H, Schewe S, Schneider M, Sörensen H, Zeidler H, Visvanathan S, Sieper J, Braun J. Safety and efficacy of readministration of infliximab after longterm continuous therapy and withdrawal in patients with ankylosing spondylitis. J Rheumatol. 2007 Mar;34(3):510-5. Epub 2007 Feb 1.

ReferenceCitation: Braun J, Baraliakos X, Listing J, Fritz C, Alten R, Burmester G, Krause A, Schewe S, Schneider M, Sörensen H, Zeidler H, Sieper J. Persistent clinical efficacy and safety of anti-tumour necrosis factor alpha therapy with infliximab in patients with ankylosing spondylitis over 5 years: evidence for different types of response. Ann Rheum Dis. 2008 Mar;67(3):340-5. Epub 2007 Oct 29.

ReferenceCitation: Dijkmans B, Emery P, Hakala M, Leirisalo-Repo M, Mola EM, Paolozzi L, Salvarani C, Sanmarti R, Sibilia J, Sieper J, Van Den Bosch F, van der Heijde D, van der Linden S, Wajdula J. Etanercept in the longterm treatment of patients with ankylosing spondylitis. J Rheumatol. 2009 Jun;36(6):1256-64. doi: 10.3899/jrheum.081033. Epub 2009 May 1. Erratum in: J Rheumatol. 2010 Oct;37(10):2198.

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Sieper J, Braun J. Radiographic progression in patients with ankylosing spondylitis after 2 years of treatment with the tumour necrosis factor alpha antibody infliximab. Ann Rheum Dis. 2005 Oct;64(10):1462-6. Epub 2005 Mar 18.

ReferenceCitation: van der Heijde D, Landewé R, Baraliakos X, Houben H, van Tubergen A, Williamson P, Xu W, Baker D, Goldstein N, Braun J; Ankylosing Spondylitis Study for the Evaluation of Recombinant Infliximab Therapy Study Group. Radiographic findings following two years of infliximab therapy in patients with ankylosing spondylitis. Arthritis Rheum. 2008 Oct;58(10):3063-70. doi: 10.1002/art.23901.

ReferenceType: derived

ReferenceCitation: Essers I, van Tubergen A, Heldmann F, Baraliakos X, Braun J, Kiltz U, Boonen A. Do patients with ankylosing spondylitis adapt to their disease? Evidence from a 'then-test' in patients treated with TNF inhibitors. RMD Open. 2015 Nov 17;1(1):e000164. doi: 10.1136/rmdopen-2015-000164. eCollection 2015.

lead_sponsor: Rheumazentrum Ruhrgebiet

location_country:
United Kingdom
Netherlands
Finland
France
Belgium
Germany

location_facility: Universitair Ziekenhuis, Afdeling Rheumatologie

location_facility: Klinikum der Universität München, Rheumaeinheit

location_facility: Klinik für Immunologie und Rheumatologie der MHH

location_facility: University Central Hospital, Division of Rheumatology

lead_sponsor_type: industriel

status_simplified: Unknown


type: EudraCT Number

title: A phase1 Multi-center, Open Label, Dose-escalation Study of Oral LEE011 in Patients With Advanced Solid Tumors or Lymphoma

summary:
LEE011 is a new oral drug designed to inhibit the activity of an enzyme known as CDK4/6. CDK4/6 is involved in the process that allows both normal and cancer cells to divide and multiply. Cancer cells are often driven to divide and multiply by abnormalities that increase the activity of CDK4. Hence there is hope that blocking the activity of CDK4 may slow the growth of some cancers. LEE011 has shown anti-cancer activity in several different tumor models in animals.

Because CDK4 is important in both normal and cancerous cells, LEE011 is expected to decrease the ability of the bone marrow to make white blood cells, platelets, and red blood cells. Although these effects are expected to be reversible, they can increase the risk of infection, bleeding and fatigue.

The primary purpose of this study is to find the highest dose of LEE011 that can be safely given to adult patients with advanced solid tumors or lymphomas for which no further effective standard treatment is available. It will provide information about the side effects that may occur following treatment. The study will also possibly provide early evidence for LEE011's anti-tumor activity.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Novartis Pharmaceuticals

location_country:
Netherlands
United States
France

location_facility: University of Michigan Comprehensive Cancer Center SC

location_facility: Sarah Cannon Research Institute DeptofSarahCannonRes.Inst. (2)

intervention_type: Drug

status_simplified: Completed


title: A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

summary: A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.

lead_sponsor: BioMarin Pharmaceutical

location_country:
United Kingdom
Australia
United States
France
Germany

location_facility: Hôpital de I´Archet- Centre Hospitalier Universitaire Nice

location_facility: Univ of California San Diego School of Medicine

location_facility: University of Florida College of Medicine

location_facility: Old Queen Elizabeth Hospital, Department of Medicine

intervention_type: Biological


title: Double Blind Randomized Phase III Study of Maintenance Pazopanib Versus Placebo in NSCLC Patients Non Progressive After First Line Chemotherapy. MAPPING, an EORTC Lung Group Study.

summary:
RATIONALE: Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. It is not yet known whether pazopanib hydrochloride is more effective than a placebo in treating patients with non-small cell lung cancer that has not progressed after first-line chemotherapy.

PURPOSE: This randomized phase II/III trial is studying how well giving pazopanib hydrochloride works and compares it with giving a placebo in treating patients with non-small cell lung cancer who have received first-line chemotherapy.

status: Terminated

design_allocation: Randomized

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Slovenia
Greece
United Kingdom
Netherlands
France
Belgium
Germany
Egypt

location_facility: Centre Hospitalier Regional De La Citadelle

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Nottingham University Hospitals NHS Trust

location_facility: Assistance Publique - Hôpitaux de Marseille - Assistance Publique - Hôpitaux de Marseille - Hopital Nord

intervention_type: Other


type: Registry Identifier

title: A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.

summary:
This trial is conducted in Europe, Japan and the United States of America (USA).

The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.

design_allocation: Non-Randomized

ReferenceCitation: Negrier C, Knobe K, Tiede A, Giangrande P, Møss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.

ReferenceCitation: Collins PW, Møss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost. 2012 Nov;10(11):2305-12. doi: 10.1111/jth.12000.

lead_sponsor: Novo Nordisk A/S

location_country:
Denmark
Spain
United Kingdom
Sweden
United States
France
Japan
Germany


title: A Randomised, Open-labelled, Single Dose, Dose-escalation Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pegylated Long-acting Human Growth Hormone (NNC126-0083) Compared to Norditropin NordiFlex® in Growth Hormone Deficient Children

source: euctr

ReferenceType: result

ReferenceCitation: de Schepper J, Rasmussen MH, Gucev Z, Eliakim A, Battelino T. Long-acting pegylated human GH in children with GH deficiency: a single-dose, dose-escalation trial investigating safety, tolerability, pharmacokinetics and pharmacodynamics. Eur J Endocrinol. 2011 Sep;165(3):401-9. doi: 10.1530/EJE-11-0536. Epub 2011 Jul 1.

ReferenceCitation: Papathanasiou T, Agersø H, Damholt BB, Højby Rasmussen M, Kildemoes RJ. Population Pharmacokinetics and Pharmacodynamics of Once-Daily Growth Hormone Norditropin(®) in Children and Adults. Clin Pharmacokinet. 2021 Sep;60(9):1217-1226. doi: 10.1007/s40262-021-01011-3. Epub 2021 Apr 17.

location_country: Macedonia, The Former Yugoslav Republic of

summary: This trial is conducted in Europe. The aim of this clinical trial is to investigate the safety, tolerability, pharmacokinetics (the determination of the concentration of the administered medication in blood over time) and pharmacodynamics (the determination of the effect over time and the duration of action) of a long-acting growth hormone (NNC126-0083) in growth hormone deficient children.


title: Clinical Investigation of the Freedom SOLO Stentless Heart Valve

summary: This is a trial to demonstrate the safety and effectiveness of the Freedom SOLO heart valve when used to replace a diseased or dysfunctional aortic valve or aortic valve prosthesis.

ReferenceCitation: Thalmann M, Grubitzsch H, Matschke K, Glauber M, Tan E, Francois K, Amorim MJ, Hensens AG, Cesari F, Feyrer R, Diegeler A, Veit F, Repossini A; Freedom Solo Investigators. A European Multicenter Study of 616 Patients Receiving the Freedom Solo Stentless Bioprosthesis. Ann Thorac Surg. 2016 Jan;101(1):100-8. doi: 10.1016/j.athoracsur.2015.06.096. Epub 2015 Oct 9.

lead_sponsor: LivaNova

location_country: Portugal Italy Austria Netherlands Switzerland France Belgium Germany

location_facility: Herzzentrum Dresden Universitätsklinik an der Technischen Universität Dresden

location_facility: Universitätsklinikum Erlangen, Klinik für Herzchirurgie

location_facility: Ospedale del Cuore, Fondazione G. Monasterio

intervention_type: Device


title: An Open Label, Single-arm, Baseline-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Immunogenicity of Subcutaneous HX575 Administered Once a Week (qw) and Once Every Two Weeks (q2w) in the Treatment of Anemia Associated With Chronic Kidney Disease in Pre-dialysis and Dialysis Subjects

acronym: SWEEP

summary:
This is an open-label, single-arm, baseline-controlled, multicenter efficacy and safety switch study involving 500 CKD subjects suffering from anemia and treated previously with a stable dose of ESA s.c.

Correction of anemia will be maintained by s.c. administration of HX575 in two frequencies (i.e. qw and q2w), in order to maintain an Hb target range of 10.0-12.0 g/dL.

status: Withdrawn

lead_sponsor: Sandoz

location_country: Spain France Romania Germany Bulgaria

location_facility: Gesundheitszentrum Alzey Internistische Gemeinschaftspraxis

location_facility: Spitalul Clinic de Nefrologie Dr. Carol Davila

location_facility: Spitalul Clinic Municipal "Dr. Gavril Curteanu"

location_facility: Spitalul Universitar de Urgenta Bucuresti

location_facility: Centre Hospitalier Universitaire Limoges, Hôpital Dupuytren

location_facility: Gemeinschaftspraxis Dr. Spitthöver, Dr. Knee, Dr. Gröschel

location_facility: Nierenzentrum Weinheim Kreiskrankenhaus Weinheim


title: A Phase II Uncontrolled Study of BAY73-4506 in Previously Untreated Patients With Metastatic or Unresectable Renal Cell Cancer (RCC)

lead_sponsor: Bayer

ReferenceCitation: Eisen T, Joensuu H, Nathan PD, Harper PG, Wojtukiewicz MZ, Nicholson S, Bahl A, Tomczak P, Pyrhonen S, Fife K, Bono P, Boxall J, Wagner A, Jeffers M, Lin T, Quinn DI. Regorafenib for patients with previously untreated metastatic or unresectable renal-cell carcinoma: a single-group phase 2 trial. Lancet Oncol. 2012 Oct;13(10):1055-62. doi: 10.1016/S1470-2045(12)70364-9. Epub 2012 Sep 6.

location_country:
Poland
United Kingdom
Finland
United States
France
Germany

summary: This is a uncontrolled, open-label, non-randomized Phase II study of oral BAY73-4506 to evaluate the response rate of BAY73-4506 in previously untreated patients with metastatic or unresectable renal cell cancer (RCC).


title: Efficacy Evaluation of Intra-articular Hyaluronic Acid (Sinovial®) vs Synvisc® in the Treatment of Symptomatic Knee Osteoarthritis. A Double-blind, Controlled, Randomised, Parallel-group Non-inferiority Study

summary: The purpose of this study is to prove the non-inferiority of the intra-articular injection of hyaluronic acid (Sinovial®) in the symptomatic treatment of knee OA in comparison to Synvisc®.

ReferenceCitation: Theiler R, Brühlmann P. Overall tolerability and analgesic activity of intra-articular sodium hyaluronate in the treatment of knee osteoarthritis. Curr Med Res Opin. 2005 Nov;21(11):1727-33.

ReferenceCitation: Castellacci E, Polieri T. Antalgic effect and clinical tolerability of hyaluronic acid in patients with degenerative diseases of knee cartilage: an outpatient treatment survey. Drugs Exp Clin Res. 2004;30(2):67-73.

ReferenceCitation: Jordan KM, Arden NK, Doherty M, Bannwarth B, Bijlsma JW, Dieppe P, Gunther K, Hauselmann H, Herrero-Beaumont G, Kaklamanis P, Lohmander S, Leeb B, Lequesne M, Mazieres B, Martin-Mola E, Pavelka K, Pendleton A, Punzi L, Serni U, Swoboda B, Verbruggen G, Zimmerman-Gorska I, Dougados M; Standing Committee for International Clinical Studies Including Therapeutic Trials ESCISIT. EULAR Recommendations 2003: an evidence based approach to the management of knee osteoarthritis: Report of a Task Force of the Standing Committee for International Clinical Studies Including Therapeutic Trials (ESCISIT). Ann Rheum Dis. 2003 Dec;62(12):1145-55. Review.

ReferenceCitation: Recommendations for the medical management of osteoarthritis of the hip and knee: 2000 update. American College of Rheumatology Subcommittee on Osteoarthritis Guidelines. Arthritis Rheum. 2000 Sep;43(9):1905-15.

ReferenceCitation: Altman RD. Status of hyaluronan supplementation therapy in osteoarthritis. Curr Rheumatol Rep. 2003 Feb;5(1):7-14. Review.

ReferenceCitation: Lo GH, LaValley M, McAlindon T, Felson DT. Intra-articular hyaluronic acid in treatment of knee osteoarthritis: a meta-analysis. JAMA. 2003 Dec 17;290(23):3115-21.

ReferenceCitation: Wang CT, Lin J, Chang CJ, Lin YT, Hou SM. Therapeutic effects of hyaluronic acid on osteoarthritis of the knee. A meta-analysis of randomized controlled trials. J Bone Joint Surg Am. 2004 Mar;86(3):538-45.

ReferenceCitation: Bellamy N, Campbell J, Robinson V, Gee T, Bourne R, Wells G. Viscosupplementation for the treatment of osteoarthritis of the knee. Cochrane Database Syst Rev. 2006 Apr 19;(2):CD005321. Review.

ReferenceCitation: Recommendations for the registration of drugs used in the treatment of osteoarthritis. Group for the respect of ethics and excellence in science (GREES): osteoarthritis section. Ann Rheum Dis. 1996 Aug;55(8):552-7.

ReferenceCitation: Altman R, Brandt K, Hochberg M, Moskowitz R, Bellamy N, Bloch DA, Buckwalter J, Dougados M, Ehrlich G, Lequesne M, Lohmander S, Murphy WA Jr, Rosario-Jansen T, Schwartz B, Trippel S. Design and conduct of clinical trials in patients with osteoarthritis: recommendations from a task force of the Osteoarthritis Research Society. Results from a workshop. Osteoarthritis Cartilage. 1996 Dec;4(4):217-43.

ReferenceCitation: Strand V, Kelman A. Outcome measures in osteoarthritis: randomized controlled trials. Curr Rheumatol Rep. 2004 Feb;6(1):20-30. Review.

ReferenceCitation: Kirchner M, Marshall D. A double-blind randomized controlled trial comparing alternate forms of high molecular weight hyaluronan for the treatment of osteoarthritis of the knee. Osteoarthritis Cartilage. 2006 Feb;14(2):154-62. Epub 2005 Oct 19.

ReferenceCitation: Bellamy N, Buchanan WW, Goldsmith CH, Campbell J, Stitt LW. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. J Rheumatol. 1988 Dec;15(12):1833-40.

ReferenceCitation: Bellamy N. WOMAC: a 20-year experiential review of a patient-centered self-reported health status questionnaire. J Rheumatol. 2002 Dec;29(12):2473-6. Review.

ReferenceCitation: KELLGREN JH, LAWRENCE JS. Radiological assessment of osteo-arthrosis. Ann Rheum Dis. 1957 Dec;16(4):494-502.

ReferenceCitation: Altman R, Asch E, Bloch D, Bole G, Borenstein D, Brandt K, Christy W, Cooke TD, Greenwald R, Hochberg M, et al. Development of criteria for the classification and reporting of osteoarthritis. Classification of osteoarthritis of the knee. Diagnostic and Therapeutic Criteria Committee of the American Rheumatism Association. Arthritis Rheum. 1986 Aug;29(8):1039-49.

ReferenceCitation: Lockman LE. Practice tips. Knee joint injections and aspirations: the triangle technique. Can Fam Physician. 2006 Nov;52(11):1403-4.

ReferenceCitation: Lequesne MG, Mery C, Samson M, Gerard P. Indexes of severity for osteoarthritis of the hip and knee. Validation--value in comparison with other assessment tests. Scand J Rheumatol Suppl. 1987;65:85-9. Erratum in: Scand J Rheumatol 1988;17(3):following 241. Scand J Rheumatol Suppl 1988;73:1.

ReferenceCitation: Lequesne M, Samson M, Gérard P, Mery C. [Pain-function indices for the follow-up of osteoarthritis of the hip and the knee]. Rev Rhum Mal Osteoartic. 1990 Oct 30;57(9 ( Pt 2)):32S-36S. French.

ReferenceCitation: Ali Y, Weinstein M, Jokl P. Acute pseudogout following intra-articular injection of high molecular weight hyaluronic acid. Am J Med. 1999 Dec;107(6):641-2.

ReferenceCitation: Luzar MJ, Altawil B. Pseudogout following intraarticular injection of sodium hyaluronate. Arthritis Rheum. 1998 May;41(5):939-40.

ReferenceCitation: Maillefert JF, Hirschhorn P, Pascaud F, Piroth C, Tavernier C. Acute attack of chondrocalcinosis after an intraarticular injection of hyaluronan. Rev Rhum Engl Ed. 1997 Oct;64(10):593-4.

ReferenceCitation: Allen E, Krohn K. Adverse reaction to Hylan GF-20. J Rheumatol. 2000 Jun;27(6):1572.

ReferenceCitation: Chen AL, Desai P, Adler EM, Di Cesare PE. Granulomatous inflammation after Hylan G-F 20 viscosupplementation of the knee : a report of six cases. J Bone Joint Surg Am. 2002 Jul;84(7):1142-7.

ReferenceCitation: Zardawi IM, Chan I. Synvisc perisynovitis. Pathology. 2001 Nov;33(4):519-20.

ReferenceCitation: Hamburger MI, Lakhanpal S, Mooar PA, Oster D. Intra-articular hyaluronans: a review of product-specific safety profiles. Semin Arthritis Rheum. 2003 Apr;32(5):296-309. Review.

ReferenceCitation: Ehrich EW, Davies GM, Watson DJ, Bolognese JA, Seidenberg BC, Bellamy N. Minimal perceptible clinical improvement with the Western Ontario and McMaster Universities osteoarthritis index questionnaire and global assessments in patients with osteoarthritis. J Rheumatol. 2000 Nov;27(11):2635-41.

ReferenceCitation: Angst F, Aeschlimann A, Michel BA, Stucki G. Minimal clinically important rehabilitation effects in patients with osteoarthritis of the lower extremities. J Rheumatol. 2002 Jan;29(1):131-8.

ReferenceCitation: Bijlsma JW. Patient centred outcomes in osteoarthritis. Ann Rheum Dis. 2005 Jan;64(1):1-2.

ReferenceCitation: Tubach F, Ravaud P, Baron G, Falissard B, Logeart I, Bellamy N, Bombardier C, Felson D, Hochberg M, van der Heijde D, Dougados M. Evaluation of clinically relevant changes in patient reported outcomes in knee and hip osteoarthritis: the minimal clinically important improvement. Ann Rheum Dis. 2005 Jan;64(1):29-33. Epub 2004 Jun 18.

ReferenceCitation: Pavelka K, Uebelhart D. Efficacy evaluation of highly purified intra-articular hyaluronic acid (Sinovial(®)) vs hylan G-F20 (Synvisc(®)) in the treatment of symptomatic knee osteoarthritis. A double-blind, controlled, randomized, parallel-group non-inferiority study. Osteoarthritis Cartilage. 2011 Nov;19(11):1294-300. doi: 10.1016/j.joca.2011.07.016. Epub 2011 Aug 16.

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Italy
Czech Republic
Switzerland
France
Germany
Slovakia

location_facility: National Institute of Rheumatic Diseases

location_facility: Interni hemato-onkologická klinika, Fakultní nemocnice

location_facility: Unità Operativa di Reumatologia - Az. Ospedaliera Universitaria Senese - Policlinico "Le Scotte"

location_facility: Centrum léčby pohybového aparátu spol. s.r.o.

location_facility: University Hospital Centre Bois-Guillaume

location_facility: Orthopädische Universitätsklinik und Poliklinik der RWTH Aachen

location_facility: University Hospital Centre of Limoges - Rheumatology Service

location_facility: Department of Rheumatology and Institute of Physical Medicine - Zurich University

location_facility: Sezione Dipartimentale a valenza provinciale di Reumatologia - AzUSL 8

location_facility: Unità Operativa di Reumatologia - Istituto Ortopedico Gaetano Pini

location_facility: Medicina e Traumatologia dello Sport - AzUSL 2 Lucca


title: Clinical Study on Tacrolimus Ointment Over the Long-term. "CONTROL Study - Children"

summary: A long-term 0.03% tacrolimus ointment based regimen comprising of up to 6 weeks of initial twice daily treatment and subsequent twice weekly prophylactic application can effectively treat active lesions of atopic dermatitis, and prevent, delay, and reduce flares.

ReferenceCitation: Thaçi D, Reitamo S, Gonzalez Ensenat MA, Moss C, Boccaletti V, Cainelli T, van der Valk P, Buckova H, Sebastian M, Schuttelaar ML, Ruzicka T; European Tacrolimus Ointment Study Group. Proactive disease management with 0.03% tacrolimus ointment for children with atopic dermatitis: results of a randomized, multicentre, comparative study. Br J Dermatol. 2008 Dec;159(6):1348-56. doi: 10.1111/j.1365-2133.2008.08813.x. Epub 2008 Sep 6.

lead_sponsor: Astellas Pharma Inc

location_country:
Portugal
Spain
Italy
United Kingdom
Netherlands
Finland
Czech Republic
Hungary
France
Belgium
Germany


title: A Double-blind, Randomised Study Comparing the Safety and Tolerance of SMOFlipid 20% in Long-term Treatment With Parenteral Nutrition

summary: The aim of this study is to evaluate the safety and tolerance of SMOFlipid 20% in comparison to a standard lipid emulsion Intralipid 20% in patients requiring long-term parenteral nutrition. The safety and tolerance will be evaluated by biochemistry, hematology and coagulation variables, vital signs and adverse events. Further objectives to evaluate are the influence of SMOFlipid 20% on inflammation processes, the efficacy of anti-oxidative properties of vitamin E supplemented to SMOFlipid 20%, and the fatty acid pattern in red blood cells and serum.

lead_sponsor: Fresenius Kabi

location_country:
Denmark
Poland
United Kingdom
Australia
France
Israel
Netherlands

location_facility: Beilinson Medical Center, Department of Surgery "B"

location_facility: Hopital Edouard Herriot, Unite de Nutrition a Domicile Lyon

location_facility: University Hospital, Queen's Medical Centre

location_facility: Aalborg University Hospital, Department of Medical Gastroenterology

location_facility: Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego

location_facility: Rigshospitalet, Department of Medical Gastroenterology

location_facility: Wydzial Lekarski I Katedra Chirurgii Ogolnej


title: A Randomized, Double-Blind Study to Evaluate the Safety of Continued Treatment With CellCept in Patients With Well-Controlled Myasthenia Gravis Receiving a Stable Dose of Prednisone

summary: This 2 arm study will provide optional continuation of double-blind treatment with CellCept or placebo, in patients with myasthenia gravis who have achieved good symptom control in study WX17798. Patients who have completed 36 weeks of treatment in study WX17798, with stable prednisone dosing for the last 4 weeks, can continue on blinded treatment with CellCept (1g bid) or placebo until the database for WX17798 is locked and unblinded. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.

lead_sponsor: Hoffmann-La Roche

location_country:
United Kingdom
Italy
Ukraine
Former Serbia and Montenegro
United States
France
Germany


title: A Phase I Dose Escalation Study of BMS-690514 in Patients With Advanced or Metastatic Solid Tumors

summary: The purpose of this research study is to determine the highest dose of the drug (BMS-690514) that can be safely given to patients with advanced cancer.

ReferenceCitation: Soria JC, Baselga J, Hanna N, Laurie SA, Bahleda R, Felip E, Calvo E, Armand JP, Shepherd FA, Harbison CT, Berman D, Park JS, Zhang S, Vakkalagadda B, Kurland JF, Pathak AK, Herbst RS. Phase I-IIa study of BMS-690514, an EGFR, HER-2 and -4 and VEGFR-1 to -3 oral tyrosine kinase inhibitor, in patients with advanced or metastatic solid tumours. Eur J Cancer. 2013 May;49(8):1815-24. doi: 10.1016/j.ejca.2013.02.012. Epub 2013 Mar 13.

lead_sponsor: Bristol-Myers Squibb

location_country:
United States
Spain
Canada
France

location_facility: The University Of Texas Md Anderson Cancer Center


title: An Extension to a Phase II Study to Determine the Safety and Anti-Leukemic Effects of STI571 in Adult Patients With Philadelphia Chromosome Positive Leukemia Including Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Accelerated Phase Chronic Myeloid Leukemia

summary:
The objectives of Part 1 of the study were:

To determine the rate of hematologic response (HR) lasting ≥4 weeks in participants with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the accelerated phase (AP).
To evaluate duration of HR, overall survival, cytogenetic response (CyR), time to blast crisis in CML participants in the AP, improvement of symptomatic parameters, tolerability and safety of STI571 treatment.

The objective of the extension (Part 2) was:

-To enable participants to have access to study drug and continue study treatment and to decrease data collection to include only overall survival and serious adverse events.

location_country:
United Kingdom
Italy
United States
France
Germany

location_facility: MD Anderson Cancer Center, University of Texas


title: A Randomized Phase II-III Trial Evaluating the Efficacy of Capecitabine and Vinorelbine in Anthracycline and Taxane Pre-Treated Metastatic Breast Cancer

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known if capecitabine is more effective than vinorelbine in treating metastatic breast cancer.

PURPOSE: Randomized phase II/III trial to compare the effectiveness of capecitabine with that of vinorelbine in treating women who have metastatic breast cancer that has been previously treated with chemotherapy.

ReferenceCitation: Pajk B, Cufer T, Canney P, Ellis P, Cameron D, Blot E, Vermorken J, Coleman R, Marreaud S, Bogaerts J, Basaran G, Piccart M. Anti-tumor activity of capecitabine and vinorelbine in patients with anthracycline- and taxane-pretreated metastatic breast cancer: findings from the EORTC 10001 randomized phase II trial. Breast. 2008 Apr;17(2):180-5. Epub 2007 Oct 31.

ReferenceCitation: Hoon SN, Lau PK, White AM, Bulsara MK, Banks PD, Redfern AD. Capecitabine for hormone receptor-positive versus hormone receptor-negative breast cancer. Cochrane Database Syst Rev. 2021 May 26;5:CD011220. doi: 10.1002/14651858.CD011220.pub2.

location_country:
United Kingdom
Slovenia
France
Belgium
Germany

location_facility: Cancer Research Centre at Weston Park Hospital


title: Randomized Phase III Trial of Two Investigational Schedules of Ifosfamide vs. Standard Dose Doxorubicin in Patients With Advanced or Metastatic Soft Tissue Sarcoma

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether ifosfamide or doxorubicin is more effective for advanced or metastatic soft tissue sarcoma.

PURPOSE: Randomized phase III trial to compare the effectiveness of ifosfamide with that of doxorubicin in treating patients who have advanced or metastatic soft tissue sarcoma.

ReferenceCitation: Lorigan P, Verweij J, Papai Z, Rodenhuis S, Le Cesne A, Leahy MG, Radford JA, Van Glabbeke MM, Kirkpatrick A, Hogendoorn PC, Blay JY; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. Phase III trial of two investigational schedules of ifosfamide compared with standard-dose doxorubicin in advanced or metastatic soft tissue sarcoma: a European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. J Clin Oncol. 2007 Jul 20;25(21):3144-50.

location_country:
Denmark
Poland
Spain
United Kingdom
Netherlands
Hungary
France
Belgium
Germany
Slovakia

location_facility: Middlesex Hospital- Meyerstein Institute

location_facility: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology


title: Phase II Study of the Efficacy of rH Interleukin-2 in Patients With Slowly Progressing Acute Myelogenous Leukemia (AML) and With Limited Bone Marrow Blastosis After Autologous Stem Cell Transplantation or Chemotherapy

summary:
RATIONALE: Interleukin-2 may stimulate a person's white blood cells to kill acute myelogenous leukemia cells.

PURPOSE: Phase II trial to study the effectiveness of interleukin-2 in treating patients with acute myelogenous leukemia that has relapsed following previous treatment.

location_country: Croatia Italy Austria France Belgium Netherlands

location_facility:
Innsbruck Universitaetsklinik
Leiden University Medical Center
University Hospital Rebro
University Medical Center Nijmegen
Azienda Policlinico Umberto Primo
Institut Jules Bordet
Institut Gustave Roussy
Universitair Ziekenhuis Antwerpen
Ospedale San Eugenio
Hotel Dieu de Paris


title: A Randomized, Double-blind, Placebo Controlled, 3-arm Multicenter Phase 3 Study to Assess the Efficacy and Safety of Ianalumab in Patients With Active Sjogren's Syndrome (NEPTUNUS-2)

summary: A randomized, double-blind, placebo controlled, 3-arm multicenter phase 3 study to assess the efficacy and safety of ianalumab in patients with active Sjögren's syndrome (NEPTUNUS-2)

status: Recruiting

ipd_sharing: Yes

ipd_sharing_description:
Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
United Kingdom
Australia
Lebanon
Hungary
United States
France
Germany
Slovakia

status_simplified: Ongoing


title: An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia)

acronym: HELIA

summary: PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 18 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.

status: Enrolling by invitation

ipd_sharing: No

lead_sponsor: ProQR Therapeutics

location_country:
United States
Canada
France

location_facility: Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Center for Clinical Research Operations, Massachusetts Eye and Ear

location_facility: Centre de maladies rares CHNO des Quinze Vingts

location_facility: Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre

location_facility: Casey Eye Institute, Oregon Health & Science University


title: A Phase IV Multicenter, Open-Label Study Evaluating B Cell Levels In Infants Potentially Exposed To Ocrelizumab During Pregnancy

acronym: MINORE

summary: This study will evaluate the potential placental transfer of ocrelizumab in women with clinically isolated syndrome (CIS) or multiple sclerosis (MS) [in line with the locally approved indications] whose last dose of ocrelizumab was administered any time from 6 months before the last menstrual period (LMP) through to the first trimester (up to gestational week 13) of pregnancy, and the corresponding pharmacodynamic effects (B cell levels) in the infant.

primary_purpose: Other

location_country:
Spain
United Kingdom
Switzerland
United States
France
Germany

location_facility: Hopital Pierre Wertheimer - Hopital Neurologique

location_facility: MultipEL Studies - Institut für klinische Studien

location_facility: Hospital of the University of Pennsylvania

location_facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden


title: A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Spain
Italy
United Kingdom
Canada
United States
France

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

location_facility: University of Texas, Health Science Center of Houston

location_facility: Institut de Myologie - Hopital Antoine Béclère

summary: The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).


title: A Multicentre, Prospective, Open-label, Uncontrolled Phase 3 Study to Assess the Efficacy, Safety and Pharmacokinetics of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

summary: Assess the incidence of the composite of thrombotic events (TEs) and thromboembolic events (TEEs) in patients with congenital antithrombin deficiency under cover of Atenativ for surgical procedures or parturition.

primary_purpose: Prevention

lead_sponsor: Octapharma

location_country:
Spain
Italy
Austria
Hungary
United States
France
Germany

location_facility: Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Bleeding and Clotting Disorders Institute

location_facility: Universita degli Studi di Milano - IRCCS Ospedale Maggiore Policlinico - Centro Emofilia e Trombosi Angelo Bianchi Bonomi

location_facility: Universitätsklinik für Innere Medizin Klinische Abteilung für Hämatologie und Hämostaseologie

location_facility: University of Debrecen, Medical and Health Science Centre

location_facility: Centre Hospitalier Universitaire de Rouen (CHU de Rouen) - Centre de Traitement des Maladies Hemorragiques (CRTH) (Centre d'Hemophiles)


acronym: CROSSWALK-a

title: A Phase IB Randomized, Placebo-Controlled Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Patients With Sickle Cell Disease (SCD)

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

location_country:
Brazil
Spain
Italy
United States
France

location_facility: Hôpital Saint Eloi; Service de Médecine interne

location_facility: Hospital de Base de Sao Jose do Rio Preto

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Hematologia

location_facility: CHU Henri Mondor; Service de médecine interne

location_facility: Azienda Ospedaliera di Verona-Policlinico G.B. Rossi; Medicina Interna

location_facility: Hospital Universitari Vall d'Hebron; Servicio de Hematologia

location_facility: East Carolina University; Brody School of Medicine

summary: The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.


title: HighLife TSMVR Feasibility Study of the Open Cell CLARITY Valve in Patients With Moderate-severe or Severe MR, High Surgical Risk and With a High Risk for Left Ventricular Outflow Tract Obstruction (LVOTO)

acronym: HighFLO

summary: Feasibility, safety and performance of the HighLife CLARITY TSMV and its delivery system

lead_sponsor: HighLife SAS

location_country:
Poland
United Kingdom
Australia
France
Belgium
Germany

location_facility:
AZ St. Jan - Brugge
University Hospital Ulm
Medical University of Silesia
Macquarie University Hospital
Mount Hospital
UZ Leuven
Wesley Hospital
Cliniue Pasteur
Brighton and Sussex University Hospital
Deutsches Herzzentrum Munchen
Medical University of Warsaw
CHU de Rennes


title: BIOTRONIK Dynetic-35 for the Treatment of Peripheral Iliac Lesions, Using the Cobalt Chromium Balloon-expandable Dynetic-35 Stent in Association With Passeo-35 Xeo Peripheral Dilation Catheter

acronym: BIONETIC-I

summary: BIOTRONIK cobalt chromium balloon-expandable Dynetic-35 stent in association with Passeo-35 Xeo peripheral dilation catheter for the treatment of peripheral iliac lesions

lead_sponsor: Biotronik AG

location_country: Latvia Austria Hungary France Belgium Germany

location_facility: LKH Univ.-Klinikum Graz, Ambulanz für Angiologie

location_facility: Pauls Stradins Clinical University Hospital


title: An Open-Label Phase 2 Study of Surufatinib in Patients With Neuroendocrine Tumours in Europe

summary: This is a Phase 2, open-label, multi-centre study of surufatinib in patients with low to intermediate grade (Grade 1 or Grade 2), well-differentiated neuroendocrine tumours (NETs).

lead_sponsor: Hutchmed

location_country:
Spain
Italy
United Kingdom
United States
France
Norway
Germany

location_facility: University of California Irvine Medical Center UCIMC - H.H. Chao Comprehensive Digestive Disease Center CDDC

location_facility: Azienda Universitaria Ospedaliera Consorziale - Policlinico Bari

location_facility: Emory University, Winship Cancer Institute

location_facility: Institut Catala d'Oncologis (ICO) - Hospital Duran i Reynals

location_facility: Universita degli Studi di Firenze - Azienda Ospedaliero Universitaria Careggi (AOUC)

location_facility: Universitatsklinikum Essen, Klinik fur Endokrinologie


title: A Phase Ia/Ib, Open Label, Dose-escalation Study of the Combination of BI 907828 With BI 754091 (Ezabenlimab) and BI 754111 and the Combination of BI 907828 With BI 754091(Ezabenlimab) Followed by Expansion Cohorts, in Patients With Advanced Solid Tumors

summary:
This study has 2 parts. The first part is open to adults with different types of advanced cancer (solid tumors). The second part is open to people with specific types of soft tissue sarcoma, advanced lung cancer, and cancer in the stomach, bladder or bile ducts.

The participants get a combination of 2 medicines called BI 907828 and ezabenlimab (also called BI 754091). BI 907828 is a so-called MDM2 inhibitor that is being developed to treat cancer. Ezabenlimab is an antibody that may help the immune system fight cancer (immune checkpoint inhibitor). When the study started, some participants got a third medicine called BI 754111 in addition. Treatment with BI 754111 was stopped because data from another study showed no additional effect of BI 754111.

The purpose of the first part of the study is to find out the highest dose of BI 907828 that the participants can tolerate in combination with ezabenlimab. Once the best dose of BI 907828 for the combination with ezabenlimab is found, it will be used in the second part of the study.

The purpose of the second part is to see whether the combination of BI 907828 with ezabenlimab is able to make tumors shrink.

The participants are in the study as long as they benefit from and can tolerate treatment. During this time, they get infusions of ezabenlimab, and take tablets with BI 907828 every 3 weeks. The doctors check how many participants have health problems during the study. The doctors also monitor the size of the tumour.

ipd_sharing_description:
After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Also, Researchers can use the following link https://www.mystudywindow.com/msw/datasharing.

com/ to find information in order to request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website. The data shared are the raw clinical study data sets.

lead_sponsor: Boehringer Ingelheim

location_country:
Spain
United Kingdom
Australia
United States
France
Belgium
Japan
Singapore

location_facility:
INS Bergonie
National Cancer Center Hospital
Hospital Clínico San Carlos
UZ Leuven
Linear Clinical Research
Yale Cancer Center
Memorial Sloan-Kettering Cancer Center
The Royal Marsden Hospital
CTR Leon Berard
Hospital Vall d'Hebron
NEXT Oncology
National Cancer Centre


title: Follow-up in Early and Locally Advanced Breast Cancer Patients

summary: A cross-sectional follow-up study of Early and Locally Advanced Breast Cancer patients after primary treatment.

status: Active, not recruiting

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Poland
Jordan
Spain
Italy
United Kingdom
Netherlands
Switzerland
France
Belgium
Germany

location_facility: CHU Saint Pierre - CHU Saint-Pierre-Site Porte de Hal

location_facility: Leeds Teaching Hospitals NHS Trust - St. James's University Hospital

location_facility: Tumor and Breast Center ZeTuP St. Gallen

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Barts Health NHS Trust - St. Bartholomew'S Hospital

location_facility: Maria Sklodowska-Curie Memorial Cancer Centre - Maria Sklodowska-Curie National Research Institute of Oncology

location_facility: Universita Di Roma La Sapienza - Universita Degli Studi Di Roma La Sapienza - Ospedale Sant'Andrea

location_facility: HELIOS Kliniken - Helios Klinikum Wuppertal - Klin. Univ. Witten / Herdecke

location_facility: Universitaetsklinikum Leipzig-Klinik fuer Strahlentherapie und Radioonkologie

location_facility: Assistance Publique Hopitaux Paris - Assistance Publique - Hopitaux de Paris - Hopital Tenon

location_facility: Group Of Private Clinics Hirslanden - Hirslanden Klinik-Tumor Zentrum Aarau

location_facility: Groupe Radiopole Artois - Centre de radiotherapie Marie Curie

location_facility: CHU-UCL Namur - CHU Site Sainte-Elisabeth-UCL Namur


acronym: Stellar

title: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

ReferenceCitation: Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27. Review.

location_facility: Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Center for Clinical Research Operations, Massachusetts Eye and Ear

location_facility: Centre de maladies rares CHNO des Quinze Vingts

location_facility: Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre

location_facility: Casey Eye Institute, Oregon Health & Science University

summary: The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.


acronym: ILLUMINATE-A

title: ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

lead_sponsor: Alnylam Pharmaceuticals

ReferenceCitation: Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschenes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712.

location_country:
United Kingdom
Switzerland
United States
Israel
France
Netherlands
Germany
United Arab Emirates

summary: The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).


title: Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

location_country:
Taiwan
United Kingdom
Italy
Austria
Australia
New Zealand
Hungary
Korea, Republic of
France
Japan
Bulgaria

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD

location_facility: Haematology Service, Canterbury Health Laboratories

summary: The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. This antibody was intended to protect from bleeds by inhibiting a substance (Tissue Factor Pathway Inhibitor, TFPI) that reduces the ability of the body to form blood clots.


title: A Non-Interventional Observational Study of Pegvaliase-Naïve Adults With Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews

summary: The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.

time_perspective: Retrospective

lead_sponsor: BioMarin Pharmaceutical

location_country:
United Kingdom
Italy
Canada
United States
France
Turkey
Germany

location_facility: Ann and Robert H Lurie Children's Hospital

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Rheumatology Unit, Clinic for Inborn Errors of Metabolism

location_facility: University Klinik Jena, Klinik für Neuropädiatrie

location_facility: Bretonneau Hospital, Internal Medicine Department

location_facility: Emory University Department of Human Genetics

location_facility: Adult Metabolic Diseases Clinic, Vancouver General Hospital


title: A Phase 1/2 Open-label, Multi-center, Safety, Preliminary Efficacy and Pharmacokinetic (PK) Study of Isatuximab (SAR650984) in Combination With REGN2810, or Isatuximab Alone, in Patients With Advanced Malignancies

summary:
Primary Objectives:

To characterize the safety and tolerability of isatuximab in combination with REGN2810 in participants with metastatic, castration-resistant prostate cancer (mCRPC) who were naïve to anti-programmed cell death-1 (PD-1)/programmed cell death-ligand 1 (PD-L1)-containing therapy, or non-small cell lung cancer (NSCLC) who progressed on anti-PD-1/PD-L1-containing therapy, and to confirm the recommended Phase 2 dose (RP2D).
To assess the response rate of isatuximab in combination with REGN2810 in participants with either mCRPC who were anti-PD-1/PD-L1 therapy naive, or NSCLC who progressed on anti-PD-1/PD-L1 therapy, or of isatuximab as single agent in participants with mCRPC.

Secondary Objectives:

To evaluate the safety of the combination of isatuximab with REGN2810 or isatuximab monotherapy.
To evaluate the immunogenicity of isatuximab and REGN2810.
To characterize the pharmacokinetic (PK) profile of isatuximab single agent or in combination with REGN2810, and to characterize the PK of REGN2810 in combination with isatuximab.
To assess overall efficacy of isatuximab in combination with REGN2810 or as a single agent.

ReferenceCitation: Zucali PA, Lin CC, Carthon BC, Bauer TM, Tucci M, Italiano A, Iacovelli R, Su WC, Massard C, Saleh M, Daniele G, Greystoke A, Gutierrez M, Pant S, Shen YC, Perrino M, Meng R, Abbadessa G, Lee H, Dong Y, Chiron M, Wang R, Loumagne L, Lepine L, de Bono J. Targeting CD38 and PD-1 with isatuximab plus cemiplimab in patients with advanced solid malignancies: results from a phase I/II open-label, multicenter study. J Immunother Cancer. 2022 Jan;10(1):e003697. doi: 10.1136/jitc-2021-003697.

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

lead_sponsor: Sanofi

location_country:
Taiwan
United Kingdom
Italy
United States
France

location_facility:
Investigational Site Number 3800003
Investigational Site Number 2500002
Investigational Site Number 2500001
Investigational Site Number 3800005
Investigational Site Number 8400005
Investigational Site Number 3800006
Investigational Site Number 3800004
Investigational Site Number 8400003
Investigational Site Number 8260002
Investigational Site Number 8400007
Investigational Site Number 1580002
Investigational Site Number 8400004
Investigational Site Number 3800001
Investigational Site Number 8400002
Investigational Site Number 1580001
Investigational Site Number 8260001


title: Observational Registry Study of Leber Hereditary Optic Neuropathy (LHON) Affected Patients

acronym: REALITY

summary: This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported outcomes (PROs).

time_perspective: Cross-Sectional

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

lead_sponsor: GenSight Biologics

location_country:
United Kingdom
Italy
Spain
United States
France

location_facility:
Wills Eye Institute
Massachusetts Eye and Ear Infirmary
CHNO Les Quinze Vingts
Emory University Hospital
Ospedale Bellaria
CHU d'Angers
Ospedale San Raffaele
Alkek Eye Center
Moorfields Eye Hospital
Doheny Eye Center UCLA Pasadena
Institut Catala de Retina


title: An Open-Label, Multi-Center, Randomized, Dose-Escalation, Phase 1b Study to Evaluate Safety, Pharmacokinetics and Therapeutic Activity of RO6874281 in Combination With Atezolizumab ± Bevacizumab in Patients With Unresectable Advanced and/or Metastatic Renal Cell Carcinoma

summary: This is an open-label, multi-center, randomized, Phase 1b, adaptive, clinical study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary therapeutic activity of RO6874281 in combination with atezolizumab with/without bevacizumab in participants with unresectable advanced and/or metastatic RCC. The study will consist of a dose-escalation part and an extension part.

location_country:
Denmark
Spain
Italy
United Kingdom
Canada
Korea, Republic of
United States
France
Germany

location_facility: Herlev Hospital; Afdeling for Kræftbehandling

location_facility: Fondazione IRCCS Policlinico San Matteo, Oncologia

location_facility: Uniklinikum, Comprehensive Cancer Center Mainfranken; Interdisziplinäres Studienzentrum mit ECTU

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Hospital Ramon y Cajal; Servicio de Oncologia

location_facility: Institut Claudius Regaud; Departement Oncologie Medicale

location_facility: Hospital Clinico Universitario de Valencia; Servicio de Onco-hematologia

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Northwestern Center for Clinical Research; Cancer Center

location_facility: Southampton General Hospital; Medical Oncology

location_facility: Universita di Modena e Reggio Emilia;Dipartimento di Oncologia ed Ematologia

location_facility: Universitätsklinikum Tübingen; Klinik für Urologie

location_facility: Centre Léon Bérard - Centre régional de lutte contre le cancer Rhône-Alpes

location_facility: Barts & London School of Med; Medical Oncology

location_facility: University of Maryland Greenebaum Comprehensive Cancer Center

location_facility: Hospital Clínic i Provincial; Servicio de Oncología

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


title: Randomized Evaluation of BST-CarGel Versus Microfracture Alone On Recovery From Distal Femoral Cartilage Lesions

acronym: RECORD

summary: This multi-centre randomized, controlled trial will assess the impact of BST-CarGel scaffold with microfracture versus microfracture alone on short and long term clinical benefit in patients with cartilage lesions of the femoral condyle requiring operative management.

primary_purpose: Basic Science

ReferenceCitation: Shive MS, Stanish WD, McCormack R, Forriol F, Mohtadi N, Pelet S, Desnoyers J, Méthot S, Vehik K, Restrepo A. BST-CarGel® Treatment Maintains Cartilage Repair Superiority over Microfracture at 5 Years in a Multicenter Randomized Controlled Trial. Cartilage. 2015 Apr;6(2):62-72. doi: 10.1177/1947603514562064.

ReferenceCitation: Stanish WD, McCormack R, Forriol F, Mohtadi N, Pelet S, Desnoyers J, Restrepo A, Shive MS. Novel scaffold-based BST-CarGel treatment results in superior cartilage repair compared with microfracture in a randomized controlled trial. J Bone Joint Surg Am. 2013 Sep 18;95(18):1640-50. doi: 10.2106/JBJS.L.01345.

lead_sponsor: Piramal Healthcare Canada Ltd

location_country:
Spain
United Kingdom
Australia
Canada
Switzerland
France
Germany

location_facility:
Banff Sport Medicine
Hôpital Maisonneuve-Rosemont
Hospital Quironsalud Barcelona
University Hospital Southampton
CHRU Nancy - Hospital Central
Hospital Universitario Miguel Servet
Murdoch Orthopaedic Clinic
Calvary Wakefield Hospital
University Medical Centre Regensburg
Hospital Universitari del Mar
Hopital de La Croix-Rousse
SportClinic Zurich / Hirslanden Clinic
Fowler Kennedy Sport Medicine Clinic
The Royal Orthopaedic Hospital
Hospital Universitari Vall d'Hebron

intervention_type: Procedure


title: An Open-label, Multi-center Rollover Protocol for Patients Who Have Participated in a Novartis-sponsored Ribociclib (LEE011) Study and Are Continuing to Benefit From Ribociclib as Single Agent or in Combination With Other Investigational Treatments

summary: This study is to allow continued use of ribociclib (LEE011) as single agent or in combination with other investigational treatments in patients benefitting from treatment in an eligible Novartis-sponsored ribociclib (LEE011) study that has reached its primary objective(s) or has been halted for other reasons.

ipd_sharing: Undecided

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

lead_sponsor: Novartis Pharmaceuticals

location_country:
Taiwan
Spain
United States
France
Singapore

location_facility: The Regents of the University of Michigan


title: A Post-Market, Prospective, Multi-Center, Single-Arm Clinical Investigation of Phasix™ Mesh for VHWG Grade 3 Midline Hernia Repair

summary: The purpose of this study is to collect additional data on safety and performance of Phasix™ Mesh in subjects requiring Ventral Hernia Working Group (VHWG) Grade 3 midline hernia repair.

ReferenceCitation: Van Rooijen MMJ, Tollens T, Jørgensen LN, de Vries Reilingh TS, Piessen G, Köckerling F, Miserez M, Windsor ACJ, Berrevoet F, Fortelny RH, Dousset B, Woeste G, van Westreenen HL, Gossetti F, Lange JF, Tetteroo GWM, Koch A, Jeekel J. Slowly resorbable biosynthetic mesh: 2-year results in VHWG grade 3 hernia repair. Hernia. 2022 Feb;26(1):131-138. doi: 10.1007/s10029-021-02453-1. Epub 2021 Jul 19.

ReferenceCitation: van Rooijen MMJ, Jairam AP, Tollens T, Jørgensen LN, de Vries Reilingh TS, Piessen G, Köckerling F, Miserez M, Windsor ACJ, Berrevoet F, Fortelny RH, Dousset B, Woeste G, van Westreenen HL, Gossetti F, Lange JF, Tetteroo GWM, Koch A, Kroese LF, Jeekel J. A post-market, prospective, multi-center, single-arm clinical investigation of Phasix™ mesh for VHWG grade 3 midline incisional hernia repair: a research protocol. BMC Surg. 2018 Nov 20;18(1):104. doi: 10.1186/s12893-018-0439-7.

lead_sponsor: C. R. Bard

location_country:
Denmark
United Kingdom
Italy
Austria
Netherlands
France
Belgium
Germany

location_facility: Klinikum der Johann Wolfgang Goethe-Universität

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: University College London Hospitals NHS Trust


title: An Open-Label, Phase 2, Pilot Study Investigating the Safety, Clinical Activity, Pharmacokinetics, and Pharmacodynamics of Oral Treatment With the BTK Inhibitor PRN1008 in Patients With Newly Diagnosed or Relapsing Pemphigus Vulgaris

summary: Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy, and with conventional immunosuppressive "rescue treatment", if indicated. The duration of therapy in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase, Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.

lead_sponsor: Principia Biopharma, a Sanofi Company

location_country: Croatia Greece Australia France Israel

location_facility: Hospital of Venereal and Skin Diseases A.Syggros

location_facility: Papageorgiou General Hospital of Thessaloniki


title: A Phase II, Open-label, Randomized Controlled Study of PDR001 in Patients With Moderately Differentiated/Undifferentiated Locally Advanced Recurrent or Metastatic Nasopharyngeal Carcinoma Who Progressed on Standard Treatment

ReferenceCitation: Even C, Wang HM, Li SH, Ngan RK, Dechaphunkul A, Zhang L, Yen CJ, Chan PC, Chakrabandhu S, Ma BBY, Tanasanvimon S, Lee VHF, Lou PJ, Li Z, Spira AI, Sukari A, Guigay J, McCune S, Gonzalez-Maffe J, Szpakowski S, Yao Y, Liang H, Mataraza J, Séchaud R, Manenti L, Lim DW. Phase II, Randomized Study of Spartalizumab (PDR001), an Anti-PD-1 Antibody, versus Chemotherapy in Patients with Recurrent/Metastatic Nasopharyngeal Cancer. Clin Cancer Res. 2021 Dec 1;27(23):6413-6423. doi: 10.1158/1078-0432.CCR-21-0822. Epub 2021 Aug 25.

location_country:
Taiwan
Hong Kong
Thailand
United States
France
China
Singapore

location_facility: NYU Laura and Isaac Perlmutter Cancer Center Laura & Isaac Perlmutter Ctr

location_facility: Northwest Georgia Oncology Center NWGA Onc - Carrollton

summary:
The purpose of this randomized controlled Phase II study is to assess the efficacy of PDR001 versus investigator's choice of chemotherapy in patients with advanced nasopharyngeal carcinoma (NPC).

By blocking the interaction between PD-1 and its ligands PD-L1 and PD-L2, PDR001 leads to the activation of a T-cell mediated antitumor immune response.


title: An Open-label, Multicenter, Dose Escalation Phase 1b Study to Assess the Safety and Pharmacokinetics of Subcutaneous Delivery of Daratumumab With the Addition of Recombinant Human Hyaluronidase (rHuPH20) for the Treatment of Subjects With Relapsed or Refractory Multiple Myeloma

summary: The purpose of the study is to evaluate the pharmacokinetics and safety from the mixture of daratumumab and rHuPH20 prepared immediately before administration via Subcutaneous (SC) delivery (Part 1) and CF (co-formulated daratumumab and rHuPH20 preparation) administration via SC delivery of daratumumab (Part 2) and to evaluate the safety of Dara-CF 1800 milligram (mg) SC delivery without pre-dose and post-dose corticosteroids (Part 3).

ReferenceCitation: Usmani SZ, Nahi H, Mateos MV, van de Donk NWCJ, Chari A, Kaufman JL, Moreau P, Oriol A, Plesner T, Benboubker L, Hellemans P, Masterson T, Clemens PL, Luo M, Liu K, San-Miguel J. Subcutaneous delivery of daratumumab in relapsed or refractory multiple myeloma. Blood. 2019 Aug 22;134(8):668-677. doi: 10.1182/blood.2019000667. Epub 2019 Jul 3.

lead_sponsor: Janssen Research & Development, LLC

location_country:
Denmark
Spain
Sweden
United States
France
Netherlands


title: A Phase 1/2 Study of Cabiralizumab, an Anti-CSF1 Receptor Antibody, in Patients With Pigmented Villonodular Synovitis (PVNS)/ Diffuse Type Tenosynovial Giant Cell Tumor (Dt-TGCT)

lead_sponsor: Five Prime Therapeutics, Inc.

location_country:
Poland
United Kingdom
Korea, Republic of
United States
France
Netherlands

location_facility: Institut Bergonie- CRLCC de Bordeaux et du Sud-Ouest

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Klinika Nowotworow Tkanek Miekkich, Kosci i Czerniakow, Centrum Onkologii-Instytut im. M. Sklodowskiej-Curie

location_facility: The University of Texas, MD Anderson Cancer Center

summary: This is a phase 1/2 single arm, open-label, safety, tolerability, and PK study of cabiralizumab in PVNS/dt-TGCT patients.


type: Other Grant/Funding Number

title: Swiss Trial of Decompressive Craniectomy Versus Best Medical Treatment of Spontaneous Supratentorial Intracerebral Hemorrhage (SWITCH): a Randomized Controlled Trial

acronym: SWITCH

summary:
Spontaneous intracerebral hemorrhage (ICH) remains a devastating disease with mortality rates up to 52% at 30 days. It is a major public health problem with an annual incidence of 10-30 per 100'000 population, accounting for 2 million (10-15%) of about 15 million strokes worldwide each year. The strategy of decompressive craniectomy (DC) is beneficial in patients with malignant middle cerebral artery (MCA) infarction. Based on the common pathophysiological mechanisms of these two conditions, this procedure is also frequently performed in patients with ICH, but is has not yet been investigated in a randomized trial.

The primary objective of this randomized controlled trial is to determine whether decompressive surgery and best medical treatment in patients with spontaneous ICH will improve outcome compared to best medical treatment only.

Secondary objectives are to analyze mortality, dependency and quality of life. Safety endpoints are to determine cause of any mortality and the rate of medical and surgical complications after DC compared with best medical treatment alone.

lead_sponsor: University Hospital Inselspital, Berne

location_country: Spain Austria Netherlands Finland Switzerland Sweden France Belgium Germany

location_facility: Universitätsklinik für Neurochirurgie, Kepler Universitätsklinikum Linz

location_facility: Dep. of Clinical Neuroscience, Service of Neurosurgery

location_facility: Klinik für Neurochirurgie Uniklinik RWTH Aachen

location_facility: Neurochirurgische Klinik, Universitätsklinikum Mannheim

location_facility: Neurochirurgische Klinik, Universitätsmedizin Mainz

location_facility: Servicio de Neurocirurgía Hospital de la Santa Creu i Sant Pau

location_facility: Klinik für Neurochirurgie, Klinikum Kassel

location_facility: Dep. of Neurosurgery, University Hospital Zürich

location_facility: Universitätsklinik für Neurochirurgie, Medizinische Universität Innsbruck

location_facility: Klinik für Allgemeine Neurologie, Universitätsklinikum Münster

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Würzburg

location_facility: Dep. Neurosurgery, Ospedale Regionale di Lugano

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Düsseldorf

location_facility: Zentrum der Neurologie und Neurochirurgie, Universitätsklinikum Frankfurt

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Schleswig Holstein

location_facility: Academic Medical Center Amsterdam, Department of Neurology

location_facility: Servicios de Neurología, Neurocirugía y Cuidados Intensivos del Hospital Virgen del Rocío

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Freiburg

location_facility: Neurologische Klinik, Universitätsklinikum Erlangen

location_facility: University Medical Center Utrecht, Department of Neurology, Department of Neurosurgery

location_facility: Centre Hospitalier Universitaire de Caen

location_facility: Servicio de Neurología, Hospital Universitario La Paz

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Essen (AöR)

location_facility: Dep. of Neurosurgery, Kantonsspital Luzern

location_facility: Neurochirurgische Klinik, Universitätsklinikum Gießen und Marburg UKGM

location_facility: Department of Neurosurgery, Charité - Universitätsmedizin Berlin

location_facility: Klinik und Poliklinik für Neurochirurgie, Universitätsklinikum Bonn

location_facility: Klinik für Neurochirurgie, Universitätsmedizin Göttingen

location_facility: Department of Neurology, Helsinki University Central Hospital

location_facility: Dep. of Neurology / Dep. of Neurosurgery, Bern University Hospital

location_facility: Klinik für Neurochirugie, Helios Klinikum Erfurt

location_facility: Servicio de Neurocirurgía Bellvitge Hospital

location_facility: Dep. of Neurosurgery, Klinikum rechts der Isar der Technischen Universität München

lead_sponsor_type: academique


title: A Phase Ib, Open-label, Multicenter, Dose Escalation and Expansion Study, to Evaluate the Safety, Pharmacokinetics and Activity of INC280 in Combination With Cetuximab in c-MET Positive CRC and HNSCC Patients Who Have Progressed After Anti-EGFR Monoclonal Antibody Therapy.

summary:
This was an open-label, phase Ib, multicenter clinical trial to determine the MTD/RDE of the orally administered c-MET inhibitor INC280 in combination with cetuximab. This combination was to be explored in c-MET positive mCRC and HNSCC patients whose disease progressed on cetuximab or panitumumab treatment. The dose escalation part was to be guided by a Bayesian Logistic Regression Model with overdose control. At MTD/RDE, additional mCRC and HNSCC patients who progressed on cetuximab or panitumumab treatment were to be enrolled in two expansion groups to further assess the anti-tumor activity and the safety and tolerability of the combination of INC280 and cetuximab. Patients were to receive INC280 on a continuous bid dosing regimen and cetuximab every week. A treatment cycle was defined as 28 days with no scheduled break between cycles.

The trial was terminated because of difficulties in identifying patients who met the eligibility criteria.

ReferenceCitation: Delord JP, Argilés G, Fayette J, Wirth L, Kasper S, Siena S, Mesia R, Berardi R, Cervantes A, Dekervel J, Zhao S, Sun Y, Hao HX, Tiedt R, Vicente S, Myers A, Siu LL. A phase 1b study of the MET inhibitor capmatinib combined with cetuximab in patients with MET-positive colorectal cancer who had progressed following anti-EGFR monoclonal antibody treatment. Invest New Drugs. 2020 Dec;38(6):1774-1783. doi: 10.1007/s10637-020-00928-z. Epub 2020 May 14.

location_country:
Spain
Italy
Canada
United States
France
Belgium
Germany

location_facility: University of Utah / Huntsman Cancer Institute Onc Dept

location_facility: Massachusetts General Hospital Head & Neck


status: No longer available

acronym: RIALTO

title: An Open-Label, Multi-center, Expanded Access Protocol of Blinatumomab for the Treatment of Pediatric and Adolescent Subjects With Relapsed and/or Refractory B-precursor Acute Lymphoblastic Leukemia (ALL)

lead_sponsor: Amgen

study_type: Expanded Access

ReferenceCitation: Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rössig C, Kormany WN, Viswagnachar P, Chen-Santel C. Blinatumomab in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia: RIALTO expanded access study final analysis. Blood Adv. 2022 Feb 8;6(3):1004-1014. doi: 10.1182/bloodadvances.2021005579.

ReferenceCitation: Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Chen-Santel C. Blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia: results of the RIALTO trial, an expanded access study. Blood Cancer J. 2020 Jul 24;10(7):77. doi: 10.1038/s41408-020-00342-x. Erratum in: Blood Cancer J. 2021 Feb 1;11(2):28. Blood Cancer J. 2021 Oct 27;11(10):173.

location_country:
United Kingdom
Italy
Austria
Switzerland
United States
France
Germany

summary:
Primary Objective:

To estimate the incidence of treatment-emergent and treatment-related adverse events during treatment with blinatumomab in pediatric and adolescent subjects with B-precursor ALL in second or later bone marrow relapse, in any marrow relapse after alloHSCT, or refractory to other treatments

Secondary Objective(s):

To describe key efficacy outcomes, including incidence of complete response (CR) within 2 cycles of blinatumomab, minimal residual disease (MRD) remission within 2 cycles of blinatumomab, relapse free survival (RFS), overall survival (OS), incidence of alloHSCT, and 100-day mortality after alloHSCT.

Hypotheses:

A formal statistical hypothesis will not be tested. The incidence of treatment-emergent and treatment-related adverse events will be estimated.

Study Endpoints:

Incidence of treatment-emergent and treatment-related adverse events
Incidence of CR within 2 cycles of blinatumomab
MRD remission within 2 cycles of blinatumomab
RFS
OS
Incidence of alloHSCT
100-day mortality after alloHSCT

Study Design:

Multi-center, open-label, single-arm expanded access protocol


title: Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

lead_sponsor: Octapharma

location_country:
Poland
United Kingdom
Georgia
Canada
Ukraine
India
United States
France
Moldova, Republic of

location_facility: The National Children Specialized Hospital "OHMATDET"

location_facility: Great Ormond Street Hospital for Children

location_facility: Institute of Hematology and Transfusiology

location_facility: Danylo Halytsky Lviv National Medical University

summary: The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.


title: A Randomised, Open-labelled, Active-controlled, Multinational, Dose-escalation Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of Long-acting Growth Hormone (NNC0195-0092, Somapacitan) Compared to Daily Dosing of Norditropin® SimpleXx® in Children With Growth Hormone Deficiency

lead_sponsor: Novo Nordisk A/S

ReferenceCitation: Juul Kildemoes R, Hojby Rasmussen M, Agerso H, Overgaard RV. Optimal Monitoring of Weekly IGF-I Levels During Growth Hormone Therapy With Once-Weekly Somapacitan. J Clin Endocrinol Metab. 2021 Jan 23;106(2):567-576. doi: 10.1210/clinem/dgaa775.

ReferenceCitation: Papathanasiou T, Agersø H, Damholt BB, Højby Rasmussen M, Kildemoes RJ. Population Pharmacokinetics and Pharmacodynamics of Once-Daily Growth Hormone Norditropin(®) in Children and Adults. Clin Pharmacokinet. 2021 Sep;60(9):1217-1226. doi: 10.1007/s40262-021-01011-3. Epub 2021 Apr 17.

ReferenceCitation: Juul RV, Rasmussen MH, Agersø H, Overgaard RV. Pharmacokinetics and Pharmacodynamics of Once-Weekly Somapacitan in Children and Adults: Supporting Dosing Rationales with a Model-Based Analysis of Three Phase I Trials. Clin Pharmacokinet. 2019 Jan;58(1):63-75. doi: 10.1007/s40262-018-0662-5.

ReferenceCitation: Battelino T, Rasmussen MH, De Schepper J, Zuckerman-Levin N, Gucev Z, Sävendahl L; NN8640-4042 Study Group. Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: A randomized dose-escalation trial. Clin Endocrinol (Oxf). 2017 Oct;87(4):350-358. doi: 10.1111/cen.13409. Epub 2017 Aug 8.

location_country:
Slovenia
Spain
Austria
Switzerland
North Macedonia
France
Israel
Belgium
Norway
Sweden

summary: This trial is conducted in Europe and Asia. The aim of the trial is to investigate safety, tolerability, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of a single dose of long-acting growth hormone (NNC0195-0092, somapacitan) compared to daily dosing of Norditropin® SimpleXx® (somatropin) in children with growth hormone deficiency.


title: A Phase 2 Single Arm Study to Investigate the Efficacy of Autologous EBV-specific T-cells for the Treatment of Patients With Aggressive EBV Positive Extranodal NK/T-cell Lymphoma (ENKTCL)

acronym: CITADEL

summary: To investigate the efficacy of autologous EBV-specific T-cells for the treatment of patients with aggressive EBV positive extranodal NK/T-cell lymphoma

lead_sponsor: Cell Medica Ltd

location_country:
Korea, Republic of
United States
United Kingdom
France

location_facility: The Ohio State University Comprehensive Cancer Center


title: Phase Ib Dose Finding Study of Abiraterone Acetate Plus BEZ235 or BKM120 in Patients With Castration-resistant Prostate Cancer.

summary: This is an open label study of abiraterone acetate in combination with BEZ235 and abiraterone acetate in combination with BKM120 in CRPC patients with abiraterone acetate failure.

location_country:
Spain
United Kingdom
Canada
United States
France
Belgium

location_facility: Hackensack University Medical Center Hackensack Univ


summary: This study will evaluate the safety and efficacy of OnabotulinumtoxinA (BOTOX®) for the treatment of urinary incontinence due to NDO in non-catheterizing patients with MS.

ReferenceCitation: Tullman M, Chartier-Kastler E, Kohan A, Keppenne V, Brucker BM, Egerdie B, Mandle M, Nicandro JP, Jenkins B, Denys P. Low-dose onabotulinumtoxinA improves urinary symptoms in noncatheterizing patients with MS. Neurology. 2018 Aug 14;91(7):e657-e665. doi: 10.1212/WNL.0000000000005991. Epub 2018 Jul 20.

lead_sponsor: Allergan

location_country:
Poland
Portugal
Russian Federation
Canada
France
United States
Belgium
Czechia


summary: This non-drug, longitudinal, multi-center, multi-national study will evaluate the suitability of neurocognitive tests and functioning scales for the measurement of cognitive and functioning changes in individuals with Down Syndrome. Tests will be administered at clinic visits in Weeks 1, (4) and 24. The duration of the study for each individual will be between 24 and 27 weeks.

ReferenceCitation: Spiridigliozzi GA, Goeldner C, Edgin J, Hart SJ, Noeldeke J, Squassante L, Visootsak J, Heller JH, Khwaja O, Kishnani PS, Liogier d'Ardhuy X. Adaptive behavior in adolescents and adults with Down syndrome: Results from a 6-month longitudinal study. Am J Med Genet A. 2019 Jan;179(1):85-93. doi: 10.1002/ajmg.a.60685. Epub 2018 Dec 20.

lead_sponsor: Hoffmann-La Roche

location_country:
Spain
Italy
United Kingdom
Argentina
Canada
United States
France


title: An Open-label, Phase I, Dose Escalation Study Evaluating the Safety, Tolerability, and Pharmacokinetics of GDC-0575 Administered Alone and in Combination With Gemcitabine in Patients With Refractory Solid Tumors or Lymphoma

summary: This open-label, multicenter, Phase I, dose-escalation study will evaluate the safety, tolerability, and pharmacokinetics (PK) of GDC-0575 administered alone or in combination with gemcitabine in participants with refractory solid tumors or lymphoma. In Stage 1, cohorts of participants will receive multiple ascending oral doses of GDC-0575 alone or in combination with intravenous gemcitabine. In Stage 2, participants will receive GDC-0575 orally in combination with intravenous gemcitabine at or below the maximum tolerated dose determined in Stage 1. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs, up to approximately 5 years.

ReferenceCitation: Italiano A, Infante JR, Shapiro GI, Moore KN, LoRusso PM, Hamilton E, Cousin S, Toulmonde M, Postel-Vinay S, Tolaney S, Blackwood EM, Mahrus S, Peale FV, Lu X, Moein A, Epler J, DuPree K, Tagen M, Murray ER, Schutzman JL, Lauchle JO, Hollebecque A, Soria JC. Phase I study of the checkpoint kinase 1 inhibitor GDC-0575 in combination with gemcitabine in patients with refractory solid tumors. Ann Oncol. 2018 May 1;29(5):1304-1311. doi: 10.1093/annonc/mdy076.

lead_sponsor: Genentech, Inc.

location_country:
United States
France

location_facility: University of Oklahoma Health Sciences Center

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale


title: A Multicenter Pharmacogenomic Biomarker Study in Matched Patients With Severe Sepsis Treated With or Without Recombinant Human Activated Protein C [Xigris®, Drotrecogin Alfa (Activated)]

summary: The overall purpose of the study is to determine whether either of the Improved Response Polymorphisms (IRPs) individually predicts a differential DrotAA treatment effect in patients with severe sepsis and high risk of death. This will be an international, multicenter, "prospective-retrospective", nonrandomized, controlled, outcome-blinded, genotype-blinded, matched-patients study. No prospective enrollment or treatment of patients will occur under this protocol. Retrospectively collected clinical data and DNA samples will be analyzed for existing cohorts of patients with severe sepsis who were previously treated with DrotAA (treatment group) or not (control group) as part of their standard care in an ICU.

ReferenceCitation: Annane D, Mira JP, Ware LB, Gordon AC, Hinds CJ, Christiani DC, Sevransky J, Barnes K, Buchman TG, Heagerty PJ, Balshaw R, Lesnikova N, de Nobrega K, Wellman HF, Neira M, Mancini ADJ, Walley KR, Russell JA. Pharmacogenomic biomarkers do not predict response to drotrecogin alfa in patients with severe sepsis. Ann Intensive Care. 2018 Jan 31;8(1):16. doi: 10.1186/s13613-018-0353-2.

lead_sponsor: Sirius Genomics Inc.

location_country:
United Kingdom
United States
Canada
France

location_facility: University of Versailles, Hospital Raymond Poincaré (AP-HP)

location_facility: Imperial College London, Charing Cross Hospital

location_facility: Harvard University School of Public Health

location_facility: Johns Hopkins University, Bayview Medical Center

location_facility: University of British Columbia and Providence Health Care, St. Paul's Hospital

location_facility: Université Paris Descartes, Sorbonne Paris Cité, Cochin Hotel-Dieu University Hospital


title: Ingenio Device Algorithm Study

acronym: IVORY

summary: The purpose of the IVORY Study is to gather data to support global submissions/approvals for some models of the Ingenio device family.

lead_sponsor: Boston Scientific Corporation

location_country:
Denmark
Hong Kong
Spain
Italy
Austria
Netherlands
United Kingdom
Sweden
France
Malaysia
Belgium
Germany

location_facility: Azienda Ospedaliera Mater Domini Policlinico Universitario


title: An Open Label, Multicentre, Single Arm Study to Characterise the Efficacy, Safety and Tolerability of Gefitinib 250 mg (IRESSA™) as First Line Treatment in Caucasian Patients, Who Have Epidermal Growth Factor Receptor (EGFR) Mutation Positive Locally Advanced or Metastatic Non-Small Cell Lung Cancer

acronym: IFUM

summary: This study is carried out to see how Caucasian patients with lung cancer which has EGFR mutation will respond to gefitinib (IRESSA™) as a first line treatment. Safety data will also be collected and analysed to confirm that treatment with gefitinib is safe and well tolerated.

ReferenceCitation: Douillard JY, Ostoros G, Cobo M, Ciuleanu T, McCormack R, Webster A, Milenkova T. First-line gefitinib in Caucasian EGFR mutation-positive NSCLC patients: a phase-IV, open-label, single-arm study. Br J Cancer. 2014 Jan 7;110(1):55-62. doi: 10.1038/bjc.2013.721. Epub 2013 Nov 21.

lead_sponsor: AstraZeneca

location_country:
Poland
Portugal
Spain
Italy
Greece
United Kingdom
Switzerland
Hungary
France
Turkey
Romania
Norway
Bulgaria


title: An Evaluation of the Effectiveness and Tolerability of Tapentadol Hydrochloride Prolonged Release, and Tapentadol Hydrochloride Immediate Release on Demand, in Subjects With Severe Chronic Nociceptive, Mixed or Neuropathic Low Back Pain Taking WHO Step III Analgesics But Showing a Lack of Tolerability

lead_sponsor: Grünenthal GmbH

ReferenceCitation: Gálvez R, Schäfer M, Hans G, Falke D, Steigerwald I. Tapentadol prolonged release versus strong opioids for severe, chronic low back pain: results of an open-label, phase 3b study. Adv Ther. 2013 Mar;30(3):229-59. doi: 10.1007/s12325-013-0015-6. Epub 2013 Mar 7.

ipd_sharing_description: Information available on the Grünenthal Web Site.

location_country: Poland Spain Netherlands Switzerland France Belgium Germany Czechia

summary:
The main objective of the study is to evaluate the effectiveness, tolerability, and safety of tapentadol hydrochloride prolonged release in subjects suffering from severe chronic low back pain (LBP) who are taking WHO Step III analgesics and show lack of tolerability. This is a clinical effectiveness trial designed to establish a link between anticipated clinical outcomes and the clinical practice by means of selected measures of clinical and subject-reported outcome.

The trial will compare the effectiveness of previous analgesic treatment (WHO Step III) with that of tapentadol hydrochloride prolonged release treatment during defined periods of evaluation.


title: A PHASE 3, RANDOMIZED, DOUBLE BLIND, CONTROLLED, MULTI CENTER STUDY OF THE ANALGESIC EFFICACY AND SAFETY OF TANEZUMAB ADDED ON TO DICLOFENAC SR IN PATIENTS WITH OSTEOARTHRITIS OF THE KNEE OR HIP

lead_sponsor: Pfizer

ReferenceCitation: Tive L, Bello AE, Radin D, Schnitzer TJ, Nguyen H, Brown MT, West CR. Pooled analysis of tanezumab efficacy and safety with subgroup analyses of phase III clinical trials in patients with osteoarthritis pain of the knee or hip. J Pain Res. 2019 Mar 19;12:975-995. doi: 10.2147/JPR.S191297. eCollection 2019. Erratum in: J Pain Res. 2020 Sep 14;13:2267-2268.

ReferenceCitation: Hochberg MC, Tive LA, Abramson SB, Vignon E, Verburg KM, West CR, Smith MD, Hungerford DS. When Is Osteonecrosis Not Osteonecrosis?: Adjudication of Reported Serious Adverse Joint Events in the Tanezumab Clinical Development Program. Arthritis Rheumatol. 2016 Feb;68(2):382-91. doi: 10.1002/art.39492.

ReferenceCitation: Balanescu AR, Feist E, Wolfram G, Davignon I, Smith MD, Brown MT, West CR. Efficacy and safety of tanezumab added on to diclofenac sustained release in patients with knee or hip osteoarthritis: a double-blind, placebo-controlled, parallel-group, multicentre phase III randomised clinical trial. Ann Rheum Dis. 2014 Sep;73(9):1665-72. doi: 10.1136/annrheumdis-2012-203164. Epub 2013 Jul 12.

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Poland
Spain
United Kingdom
Austria
Russian Federation
Ukraine
Sweden
France
Romania
Germany

location_facility: County Emergency Clinic Hospital "Sf. Apostol Andrei"

location_facility: Road Clinical Hospital at Dnipropetrovsk station, Department of Rheumatology

location_facility: Communal Institution Ternopil regional council, "Ternopil Regional Clinical Hospital"

location_facility: Center polyclinic of Federal State Institution

location_facility: Clinical Emergency Military Hospital "Dr. Carol Davila"

location_facility: Clinical Emergency County Hospital Constanta

location_facility: Kyiv Central Basin Clinical Hospital, Department of cardiology,

location_facility: State Healthcare Institution of Moscow city "City Clinical Hospital #15 n.a. O. M. Filatov"

location_facility: Klinische Forschung Hannover - Mitte GmbH

location_facility: Schmerz- und Palliativzentrum Goeppingen

location_facility: Chernivtsi Regional Clinical Hospital,Department of Rheumatology

location_facility: Chair of Hospital Therapy of Ryazan State Medical University

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej "POLIMEDICA"

location_facility: 4th City Communal Clinical Hospital, Department of Rheumatology,

location_facility: Communal institution "City Hospital #7", Department of Therapy,

location_facility: "Centrum Medyczne MEDENS S.C. Niepubliczny Zaklad

location_facility: St. Petersburg State Healthcare Institution "City Pokrovskaya Hospital"

location_facility: City Clinical Hospital #8, Department of reumatology,

location_facility: Apotheke des Ernst von Bergmann Klinikums

location_facility: Institution of Russian Academy of Sciences "St. Petersburg Clinical Hospital of RAS"

location_facility: State Institution "Institute of Gerontology AMS of Ukraine"

location_facility: Ivano-Frankivsk Regional Clinical Hospital

location_facility: St. Petersburg State Healthcare Institution "City Outpatient Clinical #51"

location_facility: State Educational Institution of Additional Professional Education

location_facility: Institute of Urgent and Recovery Surgery named after V.K. Gusaka AMS Ukraine

location_facility: City communal clinical hospital #5, Dept. of Therapy, Danylo Galytskiy Lviv National Med. University

location_facility: Central City Clinical Hospital#1, Department of Therapy,

location_facility: Oleksandrivska Clinical Hospital in Kyiv-Department of Rheumatology # 1

location_facility: St. Petersburg State Healthcare Institution "City Hospital #25 City Rheumatology Center"

location_facility: Vinnytsya regional clinical hospital named after M.I. Pyrogova

summary: The purpose of this study is to investigate the analgesic efficacy and safety of tanezumab added on to diclofenac SR in patients with osteoarthritis of the knee or hip currently experiencing partial benefit from, and are tolerating, diclofenac 150 mg/day therapy.


title: A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

summary: This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
South Africa
France

location_facility: New York University [NYU] School of Medicine

location_facility: Institute for Genetic Medicine, Saint Peter's University Hospital