ABA-101 for Multiple Sclerosis

Not currently recruiting at 2 trial locations

Overseen By Leonard Dragone, MD PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Abata Therapeutics

Must not be taking: Disease-modifying therapies

Disqualifiers: Immunocompromised, MS relapse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety of a new treatment called ABA-101 for individuals with progressive multiple sclerosis (MS). The main goal is to observe the body's reaction to a single dose, starting with a low dose and then increasing it. It suits those with MS who have experienced worsening symptoms not due to recent flare-ups. Participants should not currently be on specific MS treatments. The trial aims to determine if this new treatment could become a future option for managing MS. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

The trial requires that participants are not currently on disease-modifying therapies (DMTs), so you would need to stop taking those medications to join.

Is there any evidence suggesting that ABA-101 is likely to be safe for humans?

Research has shown that ABA-101 is safe in animal studies. These studies found the treatment to be well-tolerated and effective in reducing inflammation. However, this trial marks the first time ABA-101 is being tested in humans.

As this is the initial study in humans, no human data is available yet. Early-phase trials like this primarily focus on safety by starting with a low dose. If the low dose proves safe, researchers will test a higher dose. This approach helps identify possible side effects while ensuring participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for multiple sclerosis, which often include medications like interferons and glatiramer acetate that modulate the immune system, ABA-101 targets the disease with a unique approach. Researchers are excited about ABA-101 because it potentially offers a new mechanism of action by directly modulating specific pathways involved in the disease, which could lead to better outcomes. Moreover, the study explores both low and high doses of ABA-101, providing valuable insights into its effectiveness and safety across different levels. This innovative approach could pave the way for more precise and effective treatments for multiple sclerosis.

What evidence suggests that ABA-101 might be an effective treatment for multiple sclerosis?

Research has shown that ABA-101 might help treat progressive multiple sclerosis. In animal studies, it reduced brain and spinal cord damage by lowering inflammation. These studies also suggest that ABA-101 decreases harmful proteins that cause inflammation and increases helpful proteins that reduce it. This process uses special cells called Tregs to calm inflammation where needed. While human studies are just beginning, these early results are promising for its potential effectiveness. Participants in this trial will receive either a low dose or a high dose of ABA-101 to evaluate its effects. ^{1 2 4 5 6}

Who Is on the Research Team?

Samantha Singer, President and CEO, Abata Therapeutics, MS MBA

Principal Investigator

Abata Therapeutics

Are You a Good Fit for This Trial?

This trial is for adults over 18 with progressive multiple sclerosis, confirmed by the McDonald criteria. Participants must show signs of brain inflammation on MRI, have worsening disability not linked to MS relapses, match a specific genetic marker (HLA), and meet certain physical function tests.

Inclusion Criteria

I can complete the nine-hole peg test as required.

My MS is getting worse without having relapses.

Has a diagnosis of MS per the 2017 revised McDonald criteria

Exclusion Criteria

Is considered by the Investigator to be immunocompromised

I am currently on medication to slow my disease's progression.

My condition worsened or returned within the last 24 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of ABA-101, starting with a low dose followed by a high dose after safety is demonstrated

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with adverse events tracked through Day 29

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ABA-101

Trial Overview The study is testing ABA-101's safety and effects in humans for the first time. It starts with a low dose to ensure safety before moving on to higher doses in participants with progressive MS.

How Is the Trial Designed?

Group I: ABA-101 Dose 2 Experimental Treatment 1 Intervention

High Dose ABA-101

Group II: ABA-101 Dose 1 Experimental Treatment 1 Intervention

Low Dose ABA-101

ABA-101 is already approved in United States for the following indications:

Approved in United States as ABA-101 for:

• Progressive Multiple Sclerosis (under investigation, not approved)

Who Is Running the Clinical Trial?

Abata Therapeutics

Lead Sponsor

Trials

1

Recruited

10+

Citations

1. neurologylive.com neurologylive.com/view/fda-fast-tracks-autologous-treg-therapy-aba-101-progressive-multiple-sclerosis

FDA Fast Tracks Autologous Treg Therapy ABA-101 for ... ABA-101 has shown promising safety and anti-inflammatory effects in animal models, and Abata Therapeutics plans to initiate a Phase 1 study ...

2. ctv.veeva.com ctv.veeva.com/study/a-phase-1-open-label-single-ascending-dose-study-of-aba-101-in-participants-with-progressive-multip

ABA-101 in Participants With Progressive Multiple Sclerosis This study will test the safety and effects of ABA-101 when given as a single dose to participants with progressive multiple sclerosis.

3. rarediseaseadvisor.com rarediseaseadvisor.com/news/aba-101-gets-fast-track-designation-progressive-ms/

After IND Approval, ABA-101 Gets Fast Track Designation ... Moreover, it showed effective suppression of inflammatory cytokines, anti-inflammatory cytokine production, antigen-dependent Treg functionality ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12052076/

Update on novel multiple sclerosis treatments - PubMed Central A phase 3 trial (NCT01433497) demonstrated clinical efficacy in slowing disease progression in primary progressive and clinically inactive ...

5. regmednet.com regmednet.com/world-multiple-sclerosis-day-insights-developments-and-current-regenerative-prospects/

World Multiple Sclerosis Day: Current Regenerative ... By directing Tregs to these fragments, ABA-101 aims to deliver localized anti-inflammatory effects at the site of the disease, potentially ...

6. multiplesclerosisnewstoday.com multiplesclerosisnewstoday.com/news-posts/2024/07/12/phase-1-trial-aba-101-progressive-ms-expected-late-2024-start/

Phase 1 trial of ABA-101 for progressive MS gets FDA OK “In preclinical studies, ABA-101 was shown to be safe and demonstrated tissue-specific trafficking, persistence, and robust suppression of ...