MBM-01 for 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called MBM-01, an experimental therapy, to determine its effectiveness for individuals with Ataxia Telangiectasia (A-T), a genetic disorder affecting movement coordination and the immune system. The treatment aims to protect cells from damage and support both the brain and immune system. The trial seeks participants with a confirmed diagnosis of A-T who can commit to the study duration. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that MBM-01 is likely to be safe for humans?

In a previous study, MBM-01, also known as Tempol, was examined for its potential to replace the missing ATM protein in people with Ataxia Telangiectasia (A-T). Research has shown that this compound can help protect cells from DNA damage and reduce harm caused by harmful molecules in the body.

While detailed safety data from earlier studies on MBM-01 is not available here, it is important to note that this trial is in Phase 2. This indicates that the treatment has already undergone initial safety testing in humans. Generally, treatments reaching this phase have demonstrated some level of safety and are being tested further to understand their effects and any side effects better.

For those considering joining the trial, it may be reassuring to know that MBM-01 has been studied enough to reach this stage. However, since each person can react differently to a treatment, discussing any concerns with the trial team or a doctor is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for A-T?

Unlike the standard treatments for ataxia telangiectasia, which often focus on managing symptoms like infections and neurological issues, MBM-01 (Tempol) is unique because it targets oxidative stress directly. This is exciting because oxidative stress plays a key role in the progression of ataxia telangiectasia, and by addressing it, Tempol has the potential to slow disease progression rather than just alleviate symptoms. Researchers are optimistic because Tempol's mechanism offers a new way to tackle the underlying issues of the condition, potentially leading to more effective management and improved quality of life for patients.

What evidence suggests that MBM-01 might be an effective treatment for Ataxia Telangiectasia?

Research has shown that MBM-01 (Tempol), the investigational treatment in this trial, might help treat Ataxia Telangiectasia (A-T) by protecting cells from DNA damage. This protection may occur by reducing harm from free radicals and boosting proteins that aid cell survival. These effects could help preserve both the brain and the immune system. Early findings suggest that Tempol could benefit individuals with A-T who have lost ATM protein due to genetic mutations. Although further research is necessary, these actions show promise for improving outcomes in people with A-T. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

Interventions

• MBM-01

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04887311

MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia The study is a multi-center open label study to assess the efficacy of MBM-01 to treat ataxia telangiectasia.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9773194/

Systematic literature review and meta-analysis of clinical ... This study summarizes pertinent information regarding clinical outcomes and the association between patient characteristics and MBM prognosis.

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.e14001

Real-world outcomes in patients with brain metastases ... The combination of nivo-rela showed intracranial activity in patients with MBM, particularly in the 1L. Prospective trials are needed to confirm these findings.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04887311?term=AREA%5BBasicSearch%5D(tempol)&rank=5

MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia Members are chosen based on the scientific skills and knowledge needed to monitor the particular trial. Also called a data safety and monitoring board, or DSMB.

5. bmjopen.bmj.com bmjopen.bmj.com/content/15/1/e091098

Real-world outcomes in patients with melanoma brain ... Objective This study examined real-world treatment patterns and outcomes in patients with melanoma brain metastasis (MBM) treated with first-line ...

6. minew.com minew.com/product/mbm01-ultra-long-range-beacon/

MBM01 Long Bluetooth Beacon: Long Range, Large Battery The MBM01 has two versions: one with 210m range and 10 year battery, and another with 600m range and 10 year battery, both IP67 waterproof.

7. researchgate.net researchgate.net/figure/SDS-PAGE-of-thio-trastuzumab-conjugation-with-MBM-1_fig1_316833007

SDS-PAGE of thio-trastuzumab conjugation with MBM 1 The maleimide carbon-carbon double bond is strongly electrophilic and can react with nucleophilic reagents such as amines and thiols, and hence they are ...

8. neogen.com neogen.com/49f807/globalassets/pim/assets/original/10015/8105_sds_en-gb.pdf

Reveal Ruminant in MBM, Kit, Safety Data Sheet, English This is a test kit that is comprised of several individual components, listed below, each of which may have its own Safety Data Sheet (SDS).

9. matrixbiomed.com matrixbiomed.com/drug-product/

Drug Product Development MBM-01 is a potent inhibitor of the master genes responsible for cancer growth and progression, HIF-1 and HIF-2. Additionally, MBM-01 inhibits inflammatory ...