ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome

Leigh Syndrome Clinical Trial

Official title:
A Phase 2a, Open-label Study to Evaluate the Safety, Tolerability, and Clinical Activity of ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome

Status Withdrawn

Clinical Trial Summary

A phase 2a, open-label study to evaluate the safety, tolerability, and clinical activity of ABI-009 (nab-sirolimus) in patients with genetically-confirmed Leigh or Leigh-like syndrome

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03747328
Study type	Interventional
Source	Aadi, LLC
Contact
Status	Withdrawn
Phase	Phase 2
Start date	April 30, 2022
Completion date	March 31, 2025

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04378075` - A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy	Phase 2/Phase 3
Completed	`NCT01721733` - Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome	Phase 2
Withdrawn	`NCT05277363` - A Study of the Natural Course of SURF1 Deficiency
Completed	`NCT02544217` - A Dose-escalating Clinical Trial With KH176	Phase 1
Recruiting	`NCT03137355` - The International Registry for Leigh Syndrome
Recruiting	`NCT01793168` - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed	`NCT02352896` - Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome	Phase 2
Recruiting	`NCT05554835` - Global Registry and Natural History Study for Mitochondrial Disorders