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

• Status: Not yet recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Matrix Biomed, Inc.
• Collaborator: The University of Texas Health Science Center, Houston
• Treatments: Tempol

Criteria

Inclusion Criteria:

1. Have a confirmed diagnosis of A-T.

a) Patients will either have a prior molecular confirmation or will be investigated;

2. If female and of childbearing potential, must be using an effective birth-control method with a history of reliability for the individual patient;

3. If a female with a male partner. If the male is of childbearing potential, adequate methods of contraception must be employed including use of condoms with spermicide. No sperm donation for 90 days until after the conclusion of the study;

4. Body weight > 15 kg;

5. Be able to participate for the full term of the clinical investigation;

6. The patient and his/her parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the patient must provide assent to participate in the study

Exclusion Criteria:

1. Females that are

a) pregnant, or are breast-feeding;

2. Females of childbearing potential who do not use adequate birth control, as determined by their Health Care Provider;

3. Patients with severe vision or hearing impairment (that is not corrected by glasses or hearing aids) that, at the investigator's discretion, interferes with their ability to perform study assessments;

4. Patients who have been diagnosed with arthritis or other musculoskeletal disorders affecting joints, muscles, ligaments, and/or nerves that by themselves affects patient's mobility and, at the investigator's discretion, interferes with their ability to perform study assessments;

5. A disability that may prevent the patient from completing all study requirements;

6. Severe or unstable pulmonary disease;

7. Uncontrolled diabetes. Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible;

8. Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years;

9. Has participated in any other trial with an investigational drug and received a dose within 30 days;

10. Requires any concomitant medication prohibited by the protocol;

11. Any other severe, unstable, or serious disease or condition that in the Investigator's opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality; and

12. Evidence of significant medical illness, or psychiatric illness/social situation that would, in the investigator's judgment, make the patient inappropriate for this study.