MBM-01 for Ataxia Telangiectasia

Phase 2

Waitlist Available

Research Sponsored by Matrix Biomed, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up t25fw will be assessed at baseline, month 3, month 6, and month 9.

Awards & highlights

No Placebo-Only Group

Summary

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.

Eligible Conditions

Ataxia Telangiectasia

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ t25fw will be assessed at baseline, month 3, month 6, and month 9.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~t25fw will be assessed at baseline, month 3, month 6, and month 9.

This trial's timeline: 3 weeks for screening, Varies for treatment, and t25fw will be assessed at baseline, month 3, month 6, and month 9. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in International Cooperative Ataxia Rating Scale (ICARS)

Secondary study objectives

Change 8-hydroxy-2- Deoxyguanosine (8-OHdG)

Change in GSH/glutathione Disulfide (GSSG) Concentration Ratio

Change in Timed 25 Feet Walking Test (T25FW)

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Cohort 1Experimental Treatment1 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.

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