Anitocabtagene Autoleucel for Myasthenia Gravis

Recruiting at 8 trial locations

Overseen By Clinical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Arcellx, Inc.

Must not be taking: Anti-CD20, Calcineurin, FcRN, others

Disqualifiers: Pregnant, Breastfeeding, Gene therapy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called anito-cel (anitocabtagene autoleucel) for individuals with generalized myasthenia gravis (GMG), a condition that causes muscle weakness. The trial aims to determine the safety and effectiveness of anito-cel in easing symptoms. Participants will receive a single dose of this experimental therapy through an IV. Eligible individuals should have GMG requiring ongoing treatment and significant muscle weakness affecting daily activities. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial requires stopping certain medications like Anti-CD20 agents, calcineurin inhibitors, FcRN inhibitors, azathioprine, mycophenolate mofetil, methotrexate, or cyclophosphamide before participating. The protocol does not specify other medications, so it's best to discuss with the trial team.

Is there any evidence suggesting that anito-cel is likely to be safe for humans?

Research shows that anitocabtagene autoleucel (anito-cel), a type of CAR-T cell therapy targeting BCMA, is under investigation for safety and tolerability. This Phase 1 trial primarily aims to determine the treatment's safety for participants. Limited information is available because the treatment remains in early research stages.

Phase 1 trials mark the first testing of a new treatment in humans following lab studies. They assess safety and potential side effects. Although specific safety data for anito-cel is not yet available, researchers closely monitor for any unwanted side effects. As the study progresses and more participants receive the treatment, further insights into its safety will emerge. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for myasthenia gravis, which typically include medications like acetylcholinesterase inhibitors and immunosuppressants, anito-cel is a cell therapy that offers a novel approach. This experimental treatment involves infusing a single dose of specially engineered cells directly into the bloodstream. Researchers are excited about anito-cel because it targets the condition at a cellular level, potentially providing a more precise and effective intervention with fewer side effects. This innovative approach could lead to more rapid and sustained symptom relief compared to current options.

What evidence suggests that anito-cel might be an effective treatment for myasthenia gravis?

Research has shown that anitocabtagene autoleucel (anito-cel) is a promising treatment. This therapy uses modified immune cells to target a specific protein, enhancing the body's ability to fight disease. In patients with relapsed/refractory multiple myeloma (RRMM), anito-cel achieved a 97% success rate, with almost all patients showing improvement. Of these, 62% experienced a complete response, with no signs of the disease. Although this data pertains to a different condition, it suggests that anito-cel could effectively alter immune responses. Participants in this trial will receive a single dose of anito-cel cells infused intravenously to evaluate its potential in treating Myasthenia Gravis. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with active generalized myasthenia gravis (GMG), a muscle weakness disease. Participants must be over 18, have moderate to severe symptoms, and show specific GMG autoantibodies above normal levels.

Inclusion Criteria

My GMG specific autoantibodies are above the normal range.

My MG symptoms moderately impact my daily life and my muscle strength is significantly affected.

I need ongoing treatment for my active GMG.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Enrollment (Leukapheresis)

Participants undergo leukapheresis to collect cells for CAR-T cell manufacturing

1 week

Pretreatment with Lymphodepletion Chemotherapy

Participants receive lymphodepletion chemotherapy prior to CAR-T cell infusion

1 week

Treatment

Participants receive a single infusion of anito-cel and are monitored for safety and efficacy

4 weeks

Weekly visits for safety monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Regular visits as per study protocol

Optional Bridging Therapy

Participants may receive bridging therapy while anito-cel is being manufactured

What Are the Treatments Tested in This Trial?

Interventions

• anito-cel

Trial Overview The study tests anito-cel, a CAR-T cell therapy targeting BCMA in patients with GMG. It's a Phase 1 trial that gradually increases doses to assess safety and early signs of effectiveness alongside standard lymphodepletion treatment.

How Is the Trial Designed?

Group I: anito-cel Experimental Treatment 2 Interventions

Single dose of anito-cel cells infused intravenously

Who Is Running the Clinical Trial?

Arcellx, Inc.

Lead Sponsor

Trials

5

Recruited

650+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06626919

A Phase 1 Study of Anitocabtagene Autoleucel for the ... A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia ...

2. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/546976

A Phase 1 Study of Anitocabtagene Autoleucel for the ... A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia ...

3. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/546976

A Phase 1 Study of Anitocabtagene Autoleucel for the ... A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia ...

4. clinicaltrials.icts.uci.edu clinicaltrials.icts.uci.edu/myasthenia-gravis

UC Irvine Myasthenia Gravis Clinical Trials for 2025 A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia ...

5. pharmacytimes.com pharmacytimes.com/view/anitocabtagene-autoleucel-demonstrated-deep-durable-responses-and-improved-mrd-negativity-in-rrmm

Anitocabtagene Autoleucel Demonstrates Deep, Durable ... Anito-cel targets BCMA and achieved a 97% overall response rate in RRMM patients, with 62% achieving complete or stringent complete responses.

6. arcellx.com arcellx.com/pipeline-focus-area/

Our Pipeline, Focus Areas, and Clinical Trials Arcellx has initiated a Phase 1, open-label study evaluating the efficacy and safety of anitocabtagene autoleucel (formerly CART-ddBCMA) in patients with ...

7. ucihealth.org ucihealth.org/clinical-trials/ascc-24-18

ARC-311 for Patients with Non-Oncology Plasma Cell ... Following a single infusion of anito-cel both safety and efficacy data will be assessed. The DLTs will be assessed in the first 28 days ...