An Open-Label Extension Study of BPN14770 in Subjects with Fragile X Syndrome

Date Added
May 1st, 2023
PRO Number
Pro00125951
Researcher
Caroline Buchanan

List of Studies

Keywords
Genetics, Pediatrics, Rare Diseases
Summary

This is an OLE study for subjects completing from one of two double-blind clinical
trials. Subjects must have completed the Week 13 visit from one of these two parent
clinical trials to be eligible for this OLE.

Institution
Self Regional Healthcare
Recruitment Contact
Caleb Hinzman
864-672-6912
chinzman@ggc.org

A Phase 2, Randomized, Double-blind, Placebo-controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Date Added
March 14th, 2023
PRO Number
Pro00123493
Researcher
Charlie Strange

List of Studies


Keywords
Genetics, Liver, Lung
Summary

This study will evaluate the safety and effectiveness of the study drug belcesiran with placebo (an inactive drug treatment) in treating Alpha-1 Antitrypsin Deficiency-Associated Liver Disease AATLD. Belcesiran or placebo will be administered subcutaneously (via a shot) in the thigh or abdomen. About 46 people will take part in this study in about 30 hospitals and clinics worldwide. If deemed eligible, subjects will be randomized (assigned into a group by chance) into 1 of 3 study cohorts (groups). Subjects in Cohorts 1 will be dosed monthly to receive a total of 7 doses. Subjects in Cohort 2 will be dosed monthly to receive a total of 13 doses. Cohort 3 will be dosed monthly for the first 6 months and then quarterly thereafter for a total of 13 doses. Subjects will be given the option for extended treatment in an optional treatment period of an additional 4 to 6 doses of either belcesiran or placebo up to 72 weeks depending on group assignment. Subjects will then enter a follow up period for up to 96 weeks depending on group assignment. Study participation will last up to 192 weeks (3.5 years).

Institution
MUSC
Recruitment Contact
Mary Hayden
843-792-8438
blantonm@musc.edu

REAL Answers (Registry Expansion Analysis to Learn Answers)

Date Added
November 16th, 2022
PRO Number
Pro00124358
Researcher
Cathy Melvin

List of Studies