Clinical Trials
40 trials registered for Perinatal-lethal Gaucher disease
A First-in-Human Study of EOS301984 as Monotherapy or Combination Therapy in Adult Participants With Advanced Solid Tumors
NCT06547957Phase 1iTeos Therapeutics
Active↗
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK), and Preliminary Anticancer Activity of GSK4524101 Alone or With Niraparib in Participants With Solid Tumors
NCT06077877Phase 1/2GlaxoSmithKline
Active↗
Pelvic Organs Prolapse Treatment Using Neodymium Laser
NCT05000957N/AMeLSyTech, Ltd
Active↗
Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA]
NCT05536973Phase 2Adverum Biotechnologies, Inc.
Active↗
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
NCT04370054Phase 3Pfizer
Active↗
Subclinical Cytomegalovirus Reactivation in Acute ANCA-associated Vasculitis
NCT04916704N/AUniversity Hospital Birmingham NHS Foundation Trust
Active↗
To Assess Efficacy and Safety of Batoclimab in Adult Participants With Active CIDP
NCT05581199Phase 2Immunovant Sciences GmbH
Active↗
A Study Investigating Treatment Factor X in People With Factor X Deficiency
NCT00930176Phase 3Bio Products Laboratory
Completed↗
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
NCT05101902N/AAdrenas Therapeutics Inc
Completed↗
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
NCT03587116Phase 3Pfizer
Completed↗
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT04560933N/ABioMarin Pharmaceutical
Completed↗
Clinical Trial on the Combination of Avelumab and Axitinib for the Treatment of Patients With Recurrent Glioblastoma
NCT03291314Phase 2Universitair Ziekenhuis Brussel
Completed↗
Effects of Kieser Resistance Training Plus Kieser Pelvic Floor Training vs. Kieser Resistance Training Plus Traditional Pelvic Floor Exercise on Urinary Incontinence in Prostate Cancer Patients After Radical Prostatectomy
NCT06206993N/AUniversity Hospital Heidelberg
Completed↗
EVALUATION OF THE TOLERABILITY AND BIOMEDICAL POTENTIAL OF A GARMENT CONTAINING PRINTED CERAMICS IN MUSCULAR PAIN
NCT07274293N/AnoiVita Srls
Completed↗
Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
NCT03185897N/ABaxalta now part of Shire
Completed↗
Immunity Against AAV in Crigler Najjar Patient
NCT02302690N/AGenethon
Completed↗
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
NCT00749957Phase 1/2Beacon Therapeutics
Completed↗
RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy
NCT05044598Phase 1/2University College, London
Completed↗
Regional Differences in Human Immunodeficiency Virus (HIV) Testing
NCT03158987N/AJena University Hospital
Completed↗
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
NCT02416622Phase 1/2Beacon Therapeutics
Completed↗