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

• Register: ClinicalTrials.gov
• Last refreshed on: 24 May 2021
• Main ID: NCT04887311
• Date of registration: 05/05/2021
• Prospective Registration: Yes
• Primary sponsor: Matrix Biomed, Inc.
• Public title: MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
• Scientific title: An Open Label Study to Assess the Safety and Efficacy of MBM-01 for the Treatment of Ataxia Telangiectasia
• Date of first enrolment: July 2021
• Target sample size: 20
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT04887311
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Nick Russo, MD

• Name: Benji Crane

Key inclusion & exclusion 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.



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Ataxia Telangiectasia in Children

Ataxia Telangiectasia

Ataxia Telangiectasia Louis-Bar

Intervention(s)

Drug: MBM-01

Primary Outcome(s)

Change in International Cooperative Ataxia Rating Scale (ICARS) [Time Frame: ICARS evaluations will be taken at baseline, month 3, month 6, and month 9.]

Secondary Outcome(s)

Change in Total Plasma Lipid Peroxides [Time Frame: Change in total plasma lipid peroxide levels taken at baseline, month 3, month 6, and month 9.]

Change in lymphocyte counts (CD3) [Time Frame: Change in serum lymphocyte counts (CD3) taken at baseline, month 3, month 6, and month 9]

Change in lymphocyte counts (CD8) [Time Frame: Change in serum lymphocyte counts (CD8) taken at baseline, month 3, month 6, and month 9]

Change in Nine Hole Peg Test (9HPT) Baseline (Day 1) to end of treatment with MBM-01 [Time Frame: The change in 9HPT will be taken at baseline, month 3, month 6, and month 9.]

Change in Timed 25 Feet Walking Test (T25FW) [Time Frame: T25FW will be assessed at baseline, month 3, month 6, and month 9.]

Change 8-hydroxy-2- Deoxyguanosine (8-OHdG) [Time Frame: Taken at baseline, month 3, month 6, and month 9.]

Change in GSH/glutathione Disulfide (GSSG) Concentration Ratio [Time Frame: taken at baseline, month 3, month 6, and month 9.]

Change in lymphocyte counts (CD4) [Time Frame: Change in serum lymphocyte counts (CD4) taken at baseline, month 3, month 6, and month 9]

Change in total antioxidant capacity [Time Frame: (TEAC) taken at baseline, month 3, month 6, and month 9]

Change in lymphocyte counts (CD19) [Time Frame: Change in serum lymphocyte counts (CD19) taken at baseline, month 3, month 6, and month 9]

Secondary ID(s)

MBI-09-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The University of Texas Health Science Center, Houston