Descartes-08 for Myasthenia Gravis

Recruiting at 20 trial locations

Overseen By Dr., MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Cartesian Therapeutics

Must be taking: Immunosuppressive drugs

Must not be taking: Rituximab, Ocrelizumab, Calcineurin inhibitors

Disqualifiers: Chronic illness, Neuromuscular disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new mRNA CAR T-cell therapy called Descartes-08 for individuals with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. The study consists of two parts, lasting approximately 6 and 8 months, and includes both a treatment group and a placebo group. Participants should have had gMG for more than a year, frequently experience muscle weakness, and be on stable doses of certain medications. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not require you to stop taking your current medications if they are deemed necessary by the investigator. However, your medication doses must be stable for at least 8 weeks before starting the trial. Some specific treatments, like certain biologics and immunoglobulins, must be stopped a few weeks before the trial.

Is there any evidence suggesting that Descartes-08 is likely to be safe for humans?

Research has shown that Descartes-08, a new mRNA CAR T-cell therapy under testing, is generally well-tolerated. In studies with 36 participants, any side effects were manageable, indicating the treatment's safety for most people. Additionally, Descartes-08 is administered without chemotherapy, potentially reducing the risk of serious side effects. Overall, the safety information appears promising for those considering participation in a clinical trial for this therapy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about Descartes-08 for myasthenia gravis because it offers a novel approach compared to existing treatments like acetylcholinesterase inhibitors, corticosteroids, and immunosuppressants. Unlike these, which mainly aim to manage symptoms or suppress the immune system, Descartes-08 involves leukapheresis and uses a manufactured cellular therapy designed to target the underlying immune response more precisely. This approach could potentially offer more effective and longer-lasting relief with fewer side effects, marking a significant advancement in treating this autoimmune condition.

What evidence suggests that Descartes-08 might be an effective treatment for myasthenia gravis?

Research shows that Descartes-08, which participants in this trial may receive, may help treat myasthenia gravis, a condition that weakens muscles. Participants who received Descartes-08 experienced a noticeable improvement, with an average reduction of 4.8 points on a scale measuring the disease's impact on daily activities. Studies have also found a significant decrease in the severity of myasthenia gravis symptoms. In this trial, Descartes-08 will be compared to a placebo. Previous studies demonstrated a clear improvement in overall symptom scores by the third month. These findings suggest that Descartes-08 could effectively manage symptoms of myasthenia gravis. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

Adults with generalized myasthenia gravis who test positive for acetylcholine receptor autoantibodies can join this trial. Specific eligibility details are not provided, but typically participants must meet certain health standards and may be excluded based on factors that could interfere with the study or their safety.

Inclusion Criteria

My myasthenia gravis is moderate to severe.

Patient must be able to give written informed consent.

Women of childbearing potential must agree to use highly effective birth control from Screening until 14 days post last dose of Descartes-08.

Exclusion Criteria

History of significant recurrent infections or any active infection that in the opinion of the Investigator may interfere with the patient's participation in the opinion of the investigator.

Patients must be seronegative for hepatitis B surface antigen.

My kidney function is reduced with a creatinine clearance below 30 mL/min.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive the investigational mRNA CAR T-cell therapy Descartes-08 or placebo

6 months

Treatment Part 2

Eligible participants continue receiving the investigational therapy or placebo

8 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 months

What Are the Treatments Tested in This Trial?

Interventions

• Descartes-08

Trial Overview The AURORA Study is testing Descartes-08, an experimental mRNA CAR T-cell therapy against a placebo to see if it's safe and works for treating myasthenia gravis. The first part of the study lasts about 6 months, followed by an optional second part lasting approximately 8 months.

How Is the Trial Designed?

Group I: Decartes-08 Experimental Treatment 1 Intervention

This group will undergo leukapheresis and receive manufactured Decartes-08

Group II: Placebo Placebo Group 1 Intervention

This group will receive placebo

Who Is Running the Clinical Trial?

Cartesian Therapeutics

Lead Sponsor

Trials

10

Recruited

300+

Citations

1. ir.cartesiantherapeutics.com ir.cartesiantherapeutics.com/news-releases/news-release-details/cartesian-therapeutics-descartes-08-observed-provide-deep-and

Cartesian Therapeutics' Descartes-08 Observed to Provide ... Descartes-08-treated participants were observed to sustain deep responses through long-term follow-up, with an average 4.8-point reduction in MG-ADL at Month ...

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000212220

Efficacy and Safety of Autologous BCMA-directed mRNA ... Compare the effect of Descartes-08 versus placebo, as measured by the change in MG Composite (MGC) score from baseline to Month 3.

3. ir.cartesiantherapeutics.com ir.cartesiantherapeutics.com/news-releases/news-release-details/cartesian-therapeutics-announces-positive-updated-results-phase

Release Details Consistent with previously reported data, Descartes-08 was observed to be well-tolerated across the safety dataset (n=36), and adverse events ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10416207/

Safety and Efficacy of Autologous RNA Chimeric Antigen ... Descartes-08 infusions were followed by numerical decreases on MG severity scales equal to or above what is considered clinically meaningful and ...

5. ir.cartesiantherapeutics.com ir.cartesiantherapeutics.com/static-files/0c5c6dce-c19c-48cc-a204-70de5ef7b7ba

Updated Data from Phase 2b Trial of Descartes-08 in ... Average reduction (±SEM) in AChR antibody in participants with baseline levels above LLOQ randomized to Descartes-08 (n=12) vs. Placebo (n=9).

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04146051

Study Details | NCT04146051 | Descartes-08 CAR-T Cells ... This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis.