NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-06-05

Study Completion Date

2027-06-16

Brief Summary

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

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Detailed Description

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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

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Ataxia Telangiectasia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open label proof of concept

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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NR treated

Nicotinamide ribonuceloside (NR), sold under the trade name Niagen™

Group Type EXPERIMENTAL

Nicotinamide ribonucleoside

Intervention Type DRUG

Two year intervention

Interventions

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Nicotinamide ribonucleoside

Two year intervention

Intervention Type DRUG

Other Intervention Names

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Niagen

Eligibility Criteria

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Inclusion Criteria

* clinically and molecular verified classical A-T disease

Exclusion Criteria

* less than 2 years of age
* participation in other on-going study
* pregnancy
* liver failure
* other severe medical conditions considered to set patient at risk

Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No