Viewing Study NCT04870866


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Study NCT ID: NCT04870866
Status: ACTIVE_NOT_RECRUITING
Last Update Posted: 2022-08-17
First Post: 2021-04-23
Is NOT Gene Therapy: True
Has Adverse Events: False

Brief Title: NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia
Sponsor: University Hospital, Akershus
Organization:

Study Overview

Official Title: NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia
Status: ACTIVE_NOT_RECRUITING
Status Verified Date: 2022-08
Last Known Status: None
Delayed Posting: No
If Stopped, Why?: Not Stopped
Has Expanded Access: False
If Expanded Access, NCT#: N/A
Has Expanded Access, NCT# Status: N/A
Acronym: None
Brief 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.

Study Oversight

Has Oversight DMC: False
Is a FDA Regulated Drug?: True
Is a FDA Regulated Device?: False
Is an Unapproved Device?: None
Is a PPSD?: None
Is a US Export?: True
Is an FDA AA801 Violation?: