MedTrace Logo

Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside

NCT06324877 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-04-18

No results posted yet for this study

Summary

Study design: Single arm open-label clinical trial in ataxia-telangiectasia to test the effects of nicotinamide riboside on ataxia scales, immune function, and neurofilament light chain. Study population: 6-10 patients with Ataxia-Telangiectasia. Dose: Nicotinamide riboside 25 mg/kg/day in 3 equal divided doses.

Primary endpoint: Scales for assessment and rating of ataxia (SARA), and International Cooperative Ataxia Rating Scale (ICARS). Improvement of at least ½ standard deviation in key clinical scales which includes either; a) significant improvement in total combined scores from the SARA and ICARS scales, and /or b) significant improvements any aspects of the SARA and ICARS scales individually, especially pertaining to; Postural and gait improvements, Improved syllable speed and articulation, Improved fine motor skills.

Secondary endpoints: Serum analysis of neurofilament light chain (Nfl), Type 1 Interferon (INFs) epigenetic signature

Conditions

  • Ataxia Telangiectasia

Interventions

DRUG

Nicotinamide riboside

Oral capsule Nicotinamide Riboside 25mg/kg/day divided into 3 equal doses

Sponsors & Collaborators

  • The University of Queensland

    lead OTHER

Principal Investigators

  • David Coman, MBBS FRACP · Queensland Children's Hospital

Study Design

Allocation
NA
Purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-05-01
Primary Completion
2025-10-30
Completion
2025-10-30

Countries

  • Australia

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06324877 on ClinicalTrials.gov