MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Status:
Not yet recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Matrix Biomed, Inc.
Collaborator:
The University of Texas Health Science Center, Houston