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

NCT04887311 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: MBI-09-01
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Ataxia Telangiectasia Louis-Bar; Ataxia Telangiectasia in Children; Ataxia Telangiectasia

Outcome Measures

Primary Outcomes (1)

• Change in International Cooperative Ataxia Rating Scale (ICARS)

  The ICARS is a 19 item rating scale of ataxia with the total score ranging from 0 to 100. A score of 0 means normal and higher scores represent worsened disease.

  ICARS evaluations will be taken at baseline, month 3, month 6, and month 9.

Secondary Outcomes (10)

• Change in Nine Hole Peg Test (9HPT) Baseline (Day 1) to end of treatment with MBM-01

  The change in 9HPT will be taken at baseline, month 3, month 6, and month 9.

• Change in Timed 25 Feet Walking Test (T25FW)

  T25FW will be assessed at baseline, month 3, month 6, and month 9.

• Change in total antioxidant capacity

  (TEAC) taken at baseline, month 3, month 6, and month 9

• Change in Total Plasma Lipid Peroxides

  Change in total plasma lipid peroxide levels taken at baseline, month 3, month 6, and month 9.

• Change in GSH/glutathione Disulfide (GSSG) Concentration Ratio

  taken at baseline, month 3, month 6, and month 9.

Study Arms (1)

Cohort 1

EXPERIMENTAL

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.

Drug: MBM-01

Interventions

MBM-01 DRUG

Patients will be administered study drug daily for 9 months QD via premarked medicine cups.

Also known as: Tempol oral solution

Cohort 1

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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;
• Body weight \> 15 kg;
• Be able to participate for the full term of the clinical investigation;
• 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

You may not qualify if:

• Females that are
• a) pregnant, or are breast-feeding;
• Females of childbearing potential who do not use adequate birth control, as determined by their Health Care Provider;
• 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;
• 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;
• A disability that may prevent the patient from completing all study requirements;
• Severe or unstable pulmonary disease;
• Uncontrolled diabetes. Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible;
• Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years;
• Has participated in any other trial with an investigational drug and received a dose within 30 days;
• Requires any concomitant medication prohibited by the protocol;
• 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
• Evidence of significant medical illness, or psychiatric illness/social situation that would, in the investigator's judgment, make the patient inappropriate for this study.

Sponsors & Collaborators

• Matrix Biomed, Inc.: lead
• The University of Texas Health Science Center, Houston: collaborator

Study Sites (1)

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Schubert R, Erker L, Barlow C, Yakushiji H, Larson D, Russo A, Mitchell JB, Wynshaw-Boris A. Cancer chemoprevention by the antioxidant tempol in Atm-deficient mice. Hum Mol Genet. 2004 Aug 15;13(16):1793-802. doi: 10.1093/hmg/ddh189. Epub 2004 Jun 22.

  PMID: 15213104 BACKGROUND

Related Links

• Related Info

MeSH Terms

Conditions

Ataxia Telangiectasia

Interventions

tempol

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocutaneous Syndromes; Ataxia; Dyskinesias; Neurologic Manifestations; Telangiectasis; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Primary Immunodeficiency Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Central Study Contacts

Benji Crane

CONTACT

6264376506; bjcrane@matrixbiomed.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Open label

Study Record Dates

• First Submitted: May 5, 2021
• First Posted: May 14, 2021
• Study Start: July 1, 2021
• Primary Completion: June 1, 2022
• Study Completion: December 1, 2022
• Last Updated: May 17, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)