Friedreich Ataxia Clinical Trial
Official title:
A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
Friedreich's ataxia (FA) is a rare, autosomal recessive disease caused by a mutation in the autosomal frataxin (FXN) gene. Progressive cardiomyopathy with cardiac hypertrophy and fibrosis is observed in most individuals with FA. The disease is more severe in those with earlier onset. Presently, there is no therapy that alters the progression of cardiomyopathy in FA, which is responsible for 59% of FA-related deaths. The primary objective of this dose escalation study is to assess the safety and tolerability of three ascending doses of LX2006 in patients with FA-associated cardiomyopathy. LX2006 is designed to restore hFXN levels in order to improve mitochondrial function. Assessments of cardiac function, biomarkers and other preliminary efficacy endpoints are also included in this study. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT05573698 -
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
|
Phase 1 | |
| Completed |
NCT00229632 -
Idebenone to Treat Friedreich's Ataxia
|
Phase 2 | |
| Completed |
NCT05579691 -
A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia
|
Phase 2 | |
| Completed |
NCT04273165 -
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
|
Phase 2 | |
| Not yet recruiting |
NCT05874388 -
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
|
N/A | |
| Completed |
NCT02594917 -
Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension
|
||
| Completed |
NCT01716221 -
An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia
|
Phase 4 | |
| Active, not recruiting |
NCT05485987 -
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
|
Phase 2 | |
| Completed |
NCT03214588 -
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
|
Phase 2 | |
| Completed |
NCT03418740 -
Neurology Measures in FA Children
|
||
| Not yet recruiting |
NCT06054893 -
A Study of Omaveloxolone in Children With Friedreich's Ataxia
|
Phase 1 | |
| Recruiting |
NCT04921930 -
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT03933163 -
Micronised Resveratrol as a Treatment for Friedreich Ataxia
|
Phase 2 | |
| Completed |
NCT02705547 -
Rosuvastatin (Crestor) in Friedreich Ataxia
|
Early Phase 1 | |
| Completed |
NCT02035020 -
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients
|
Phase 2 | |
| Completed |
NCT00015808 -
Safety Study of Idebenone to Treat Friedreich's Ataxia
|
Phase 1 | |
| Completed |
NCT04817111 -
NAD+ Precursor Supplementation in Friedreich's Ataxia
|
Phase 2 | |
| Active, not recruiting |
NCT05168774 -
FRDA Investigator Initiated Study (IIS) With Elamipretide
|
Phase 1/Phase 2 | |
| Completed |
NCT03917225 -
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
|
Phase 2 | |
| Completed |
NCT00224640 -
Iron-Chelating Therapy and Friedreich Ataxia
|
Phase 1/Phase 2 |