Trials / Active Not Recruiting

Active Not RecruitingNCT04870866

NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia

Status: Active Not Recruiting
Phase: Phase 2
Study type: Interventional
Enrollment: 13 (actual)

Sponsor: University Hospital, Akershus · Academic / Other
Sex: All
Age: 3 Years
Healthy volunteers: Not accepted

Summary

The study investigates the effect of dietary supplementation of nicotinamide ribonucleoside (NR) in children with ataxia telangiectasia (AT), with main focus on neurological symptoms.

Detailed description

Ataxia Telangiectasia (AT) is a genetic disease, where patients are born with mutations in the Ataxia- Telangiectasia Mutated (ATM) gene. The gene codes for the ATM kinase, which is required for repair of DNA double-stranded breaks and DNA damage response signalling. There is no treatment available for the neurological manifestations of AT. The study investigates the effects of NR (300 mg/day) during 2 years.

Conditions

Ataxia Telangiectasia

Interventions

Type	Name	Description
DRUG	Nicotinamide ribonucleoside	Two year intervention

Timeline

Start date: 2019-06-05
Primary completion: 2024-09-03
Completion: 2027-06-16
First posted: 2021-05-04
Last updated: 2022-08-17

Locations

2 sites across 1 country: Norway

Regulatory

FDA-regulated drug study

Source: ClinicalTrials.gov record NCT04870866. Inclusion in this directory is not an endorsement.