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

Overseen By Benji Crane

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Matrix Biomed, Inc.

Disqualifiers: Pregnancy, Severe vision impairment, Uncontrolled diabetes, Neoplastic disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 9 months

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications, but it mentions that some medications might be prohibited. It's best to discuss your current medications with the trial team to see if any changes are needed.

What is the purpose of this trial?

This trial tests MBM-01, a drug designed to protect and repair cells, in patients with Ataxia Telangiectasia (A-T). These patients lack a protein needed for cell repair, leading to severe health issues. MBM-01 helps reduce cell damage and boosts the body's repair mechanisms.

Eligibility Criteria

Inclusion Criteria

Have a confirmed diagnosis of A-T.

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

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline

Baseline assessments including safety labs and efficacy assessments

Baseline period

1 visit (in-person)

Treatment

Participants receive MBM-01 treatment for 9 months with assessments at months 3, 6, and 9

9 months

4 visits (in-person) at day 0, month 3, month 6, and month 9

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

1 visit (in-person)

Treatment Details

Interventions

• MBM-01

Participant Groups

Group I: Cohort 1 Experimental Treatment 1 Intervention

Group 1 Patients ≥13 years old will receive a total daily dose of 1200 mg/day. Group 2 - Group 4 Patients 4-12 years old will receive group weight-tiered doses at 17 mg/kg: Group 2 * Patients aged 4-12 years weighing 15kg to \<25 kg will take 340 mg/day. Group 3 * Patients aged 4-12 years weighing 25kg to \<35 kg will take 510 mg/day. Group 4 * Patients aged 4-12 years weighing ≥35 kg will take 850 mg/day.

Who Is Running the Clinical Trial?

Matrix Biomed, Inc.

Lead Sponsor

Trials

6

Recruited

250+

The University of Texas Health Science Center, Houston

Collaborator

Trials

974

Recruited

361,000+