NCT04870866

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_2

Timeline
14mo left

Started Jun 2019

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress86%
Jun 2019Jun 2027

Study Start

First participant enrolled

June 5, 2019

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

April 23, 2021

Completed
11 days until next milestone

First Posted

Study publicly available on registry

May 4, 2021

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 3, 2024

Completed
2.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 16, 2027

Expected
Last Updated

August 17, 2022

Status Verified

August 1, 2022

Enrollment Period

5.3 years

First QC Date

April 23, 2021

Last Update Submit

August 16, 2022

Conditions

Ataxia Telangiectasia

Keywords

ataxia telangiectasianicotinamide ribonucleosideLouis-Bar syndrome

Outcome Measures

Primary Outcomes (1)

  • NAD metabolome

    Increase of NAD+ and other stable NAD+ metabolites (referred to as the NAD metabolome) in blood

    2 years

Secondary Outcomes (9)

  • Patient well being

    2 years

  • Motoric function - The Scale for the Assessment and Rating of Ataxia (SARA)

    2 years

  • Motoric function - The International Cooperative Ataxia Rating Scale (ICARS)

    2 years

  • Motoric function - Customized gait scale (GS)

    2 years

  • Motoric function - AT Neuro Examination Scale Toolkit, updated version (AT-NEST)

    2 years

  • +4 more secondary outcomes

Study Arms (1)

NR treated

EXPERIMENTAL

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

Drug: Nicotinamide ribonucleoside

Interventions

Nicotinamide ribonucleosideDRUG

Two year intervention

Also known as: Niagen
NR treated

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • clinically and molecular verified classical A-T disease

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hilde Loge Nilsen

Lørenskog, Norway

Location

Oslo University Hospital

Oslo, Norway

Location

Related Publications (1)

  • Fang EF, Kassahun H, Croteau DL, Scheibye-Knudsen M, Marosi K, Lu H, Shamanna RA, Kalyanasundaram S, Bollineni RC, Wilson MA, Iser WB, Wollman BN, Morevati M, Li J, Kerr JS, Lu Q, Waltz TB, Tian J, Sinclair DA, Mattson MP, Nilsen H, Bohr VA. NAD+ Replenishment Improves Lifespan and Healthspan in Ataxia Telangiectasia Models via Mitophagy and DNA Repair. Cell Metab. 2016 Oct 11;24(4):566-581. doi: 10.1016/j.cmet.2016.09.004.

    PMID: 27732836BACKGROUND

MeSH Terms

Conditions

Ataxia Telangiectasia

Interventions

nicotinamide-beta-riboside

Condition Hierarchy (Ancestors)

Spinocerebellar AtaxiasCerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurocutaneous SyndromesAtaxiaDyskinesiasNeurologic ManifestationsTelangiectasisVascular DiseasesCardiovascular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPrimary Immunodeficiency DiseasesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Study Officials

  • Hilde L Nilsen

    University Hospital, Akershus

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Open label proof of concept
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

April 23, 2021

First Posted

May 4, 2021

Study Start

June 5, 2019

Primary Completion

September 3, 2024

Study Completion (Estimated)

June 16, 2027

Last Updated

August 17, 2022

Record last verified: 2022-08

Data Sharing

IPD Sharing
Will not share

Locations

NO(2)