A Phase 2 interventional study of MBM-01 in Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children and Ataxia Telangiectasia, sponsored by Matrix Biomed, Inc.. Status unknown at 1 site in United States. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2021-05-17.
Sponsored by Matrix Biomed, Inc. · Phase 2, Interventional, and Treatment
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.
Cells lacking ATM are left defenseless and unable to repair cellular damaged DNA, to exhibit normal cell cycle control, to effectively respond to oxidative damage, ionizing radiation, and, alkylating agents, and to maintain a healthy immune response among others. A-T patients have increased oxidative stress and significantly reduced total antioxidant levels. In an early study directed to oxidative stress in A-T patients, a decrease in the levels of total antioxidant capacity has been observed.
There is currently no cure for A-T, and current treatments are limited to palliative care. Therapies include rehabilitative care, infection prevention and treatment, and screening for pulmonary dysfunction and malignancies. Symptomatic treatments generally fall short and leave A-T patients debilitated and in a progressively wasting state. Patients suffering from A-T are in dire need of a treatment to alleviate the conditions of this disease. A drug product that can substitute for the loss of ATM has the potential to provide these patients with this critically unmet need. The active compound in MBM-01 has been shown to supplant the overall role of ATM by reducing oxidative stress, reducing DNA double strand breaks, and decreasing programmed cell death (in healthy cells). Accordingly, MBM-01 represents a potential breakthrough therapy for patients afflicted with A-T providing a multifactorial approached as evidenced by the following:
The study is a multi-center open label study to assess the efficacy of MBM-01 to treat ataxia telangiectasia. Patients will be assessed during three study phases: a baseline period, a 9-month treatment period, and a 3-month follow-up period. Patients will visit sites day 0, month 3, month 6, and month 9 for safety labs and efficacy assessments.
Dosing will follow a weight-tiered dosing schedule administered orally QD via premarked medicine cups. Patients will be administered study drug daily, 7-days a week. The patients will be placed into one of four dosing groups.
42 studies on the registry are indexed under Ataxia Telangiectasia; 7 are open to participants now.
This study's planned enrollment of 20 is below the median of 30 across 28 interventional studies indexed under Ataxia Telangiectasia.
Browse Ataxia Telangiectasia studies →Matrix Biomed, Inc. is the lead sponsor of 6 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Have a confirmed diagnosis of A-T.
a) Patients will either have a prior molecular confirmation or will be investigated;
Exclusion Criteria:
Females that are
a) pregnant, or are breast-feeding;
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Patients will be administered study drug daily for 9 months QD via premarked medicine cups.
Also known as: Tempol oral solution
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.
Time frame: ICARS evaluations will be 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)
The patient is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time, in seconds, is calculated from the initiation of the instruction to start and ends when the patient has reached the 25-foot mark. Baseline values are recorded twice.
Time frame: T25FW will be assessed at baseline, month 3, month 6, and month 9.
Change in total antioxidant capacity
The plasma antioxidant capacity will be determined by the trolox-equivalent antioxidant capacity test (TEAC)
Time frame: (TEAC) taken at baseline, month 3, month 6, and month 9
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 GSH/glutathione Disulfide (GSSG) Concentration Ratio
GSH concentrations will be compared to glutathione disulfide.
Time frame: 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 (CD4)
Time frame: Change in serum lymphocyte counts (CD4) 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 lymphocyte counts (CD19)
Time frame: Change in serum lymphocyte counts (CD19) taken at baseline, month 3, month 6, and month 9
Change 8-hydroxy-2- Deoxyguanosine (8-OHdG)
Evaluation of 8-hydroxy-2'-deoxyguanosine (8-OHdG) adduct levels in human peripheral blood lymphocytes taken at baseline, month 3, month 6, and month 9.
Time frame: Taken at baseline, month 3, month 6, and month 9.
Plan to share: No
Start is estimated for Jul 2021; primary completion for Jun 2022, 11 months later.
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This study is status unknown, as verified in May 2021. You cannot join it, but the record below documents what was studied.
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Matrix Biomed, Inc.