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MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia

NCT04887311 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2021-05-17

No results posted yet for this study

Summary

Ataxia Telangiectasia (A-T) is an autosomal recessively inherited neurodegenerative disorder that also has dramatic effects on the immune and endocrine systems. The disorder results from mutations in the A-T mutated gene (ATM) leading to a loss in the production of the ATM protein.

The active compound in MBM-01 (4-hydroxy-2,2,6,6-tetramethylpiperidine-1-oxyl) may substitute for the loss of ATM by protecting cells from DNA damage, preventing and reducing oxidative damage, triggering an increase in cellular survival proteins, and preserving the brain and peripheral immune system.

Conditions

  • Ataxia Telangiectasia Louis-Bar
  • Ataxia Telangiectasia in Children
  • Ataxia Telangiectasia

Interventions

DRUG

MBM-01

Patients will be administered study drug daily for 9 months QD via premarked medicine cups.

Sponsors & Collaborators

  • The University of Texas Health Science Center, Houston

    collaborator OTHER

  • Matrix Biomed, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-31
Primary Completion
2022-06-30
Completion
2022-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT04887311 on ClinicalTrials.gov