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
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