Found 10 matching trials
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
PHASE2Primary Objective:
To evaluate the safety (acute effects associated with i...
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
PHASE3The Study's Primary Objective is to evaluate the pharmacokinetics, safety (...
Location
RUSH University Medical Center
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
PHASE3To evaluate the safety (acute adverse effects associated with infusions, an...
Location
Multiple Locations
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
PHASE3Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficac...
Location
Centro Estadual de Hemopterapia e Hematologia do Espirito Santo
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
PHASE3A pivotal, randomized, double-blind, placebo-controlled, multi-center thera...
Status
Active Not Recruiting
Location
University of California Los Angeles
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
PHASE2This is a multinational, multicenter, open-label, rater-blinded prospective...
Location
University of California - Los Angeles
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
PHASE3A pivotal, randomized, double-blind, placebo controlled, multi-center thera...
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
PHASE2This is a multinational, multicenter, open-label, rater-blinded prospective...
Location
University of California - Los Angeles
Pivotal Study of N-acetyl-L-leucine for CACNA1A
PHASE3A pivotal, randomized, double-blind, placebo-controlled, multi-center thera...
Location
Johns Hopkins University
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
PHASE2This is a multinational, multicenter, open-label, rater-blinded prospective...