Mocravimod for Acute Myeloid Leukemia

(MO-TRANS Trial)

Recruiting at 125 trial locations

Overseen By Jens Hasskarl, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Priothera SAS

Must be taking: CsA-based, TAC-based

Must not be taking: Anti-thymocyte globulin, Alemtuzumab

Disqualifiers: Macular edema, Cardiac dysfunction, 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

Duration: 12 months

366 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called mocravimod for people with acute myeloid leukemia (AML), a type of blood cancer. The trial aims to determine if mocravimod helps patients whose AML is in remission after initial treatment. Participants will receive either 1 mg or 3 mg of mocravimod, or a placebo, taken orally every day for 12 months. This study suits individuals with AML who plan to undergo a specific type of stem cell transplant. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that certain medications for GvHD prophylaxis are not allowed. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Previous studies have shown that mocravimod is safe for humans. Research indicates that mocravimod, used to treat acute myeloid leukemia (AML), is generally well-tolerated. Some studies show that the drug, which affects certain parts of the body, was tested without major safety issues. Patients in earlier trials did not report serious side effects. The FDA has granted the drug Orphan Drug Designation, recognizing it for treating rare diseases like AML. This designation often follows some evidence of safety and effectiveness. While these studies appear promising, it's important to understand that every treatment can affect people differently, and participating in a trial helps gather more information about its safety. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for acute myeloid leukemia, which often involve chemotherapy and stem cell transplants, mocravimod offers a new approach. Mocravimod is unique because it modulates the immune system by enhancing the activity of certain immune cells, potentially improving the body's ability to fight leukemia cells. Researchers are excited about mocravimod because it is taken orally, making it more convenient compared to other treatments that require intravenous administration. Additionally, its novel mechanism of action might lead to fewer side effects and better patient outcomes.

What evidence suggests that this trial's treatments could be effective for acute myeloid leukemia?

Research shows that mocravimod could be a promising treatment for acute myeloid leukemia (AML). Studies have found that adding mocravimod to the usual treatments for patients needing stem cell transplants is safe. This addition might improve outcomes for people with AML. Early findings suggest that mocravimod may help prevent cancer recurrence after treatment. Researchers are actively studying its use as a follow-up treatment after a transplant, with some positive early results. Participants in this trial will receive either 1 mg or 3 mg of mocravimod, or a placebo, to evaluate its effectiveness and safety. ^{1 2 6 7 8}

Who Is on the Research Team?

Marcos DeLima, MD

Principal Investigator

The Ohio State University Comprehensive Cancer Center

Are You a Good Fit for This Trial?

Adults aged 18-75 with Acute Myeloid Leukemia (AML) who are in remission and planning a stem cell transplant from a closely matched donor. They should be relatively healthy, with good organ function and performance status. Those with certain heart, lung, liver or kidney problems, or using specific drugs for graft-versus-host disease prevention are excluded.

Inclusion Criteria

I am fully active or can carry out light work.

My treatment before a transplant was moderately intense or higher.

I will be using CsA or TAC for graft-versus-host disease prevention.

Exclusion Criteria

My liver tests are higher than normal.

My kidneys are not working well (creatinine clearance < 45 mL/min).

I am using ATG, alemtuzumab, or abatacept for graft-versus-host disease prevention.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mocravimod or placebo orally once per day for 12 months

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• Mocravimod

Trial Overview The trial is testing Mocravimod as an additional treatment alongside standard care for AML patients undergoing allogeneic hematopoietic stem cell transplantation (Allo-HCT). Participants will either receive Mocravimod or a placebo without knowing which one they're getting.

How Is the Trial Designed?

Group I: 3mg mocravimod arm Experimental Treatment 1 Intervention

3 mg of mocravimod orally once per day for 12 months

Group II: 1mg mocravimod arm Experimental Treatment 1 Intervention

1 mg of mocravimod orally once per day for 12 months

Group III: Placebo arm Placebo Group 1 Intervention

placebo orally once per day for 12 months

Who Is Running the Clinical Trial?

Priothera SAS

Lead Sponsor

Trials

1

Recruited

250+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05429632

Study Details | NCT05429632 | Mocravimod as Adjunctive ... The purpose of this study is to evaluate the efficacy and safety of mocravimod as an adjunctive and maintenance treatment in adult acute myeloid leukemia (AML) ...

2. priothera.com priothera.com/priothera-positive-mocravimod-phase-ib-clinical-data-published-in-transplantation-and-cellular-therapy/

Positive mocravimod Phase Ib clinical data published in ... The study found that mocravimod can safely be added to standard treatment regimens in patients with hematological malignancies requiring allo-HCT.

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/4952.1/529631/MO-TRANS-A-Randomized-Double-Blind-Placebo

MO-TRANS: A Randomized, Double-Blind, Placebo ... We aim to evaluate the efficacy and safety of MOC as maintenance treatment in the post-HCT setting in patients with acute myeloid leukemia (AML). Study ...

4. isrctn.com isrctn.com/ISRCTN12632431

A Phase III placebo-controlled efficacy and safety study of ... A Phase III placebo-controlled efficacy and safety study of mocravimod as an adjunctive and maintenance treatment in acute myeloid leukemia patients

5. targetedonc.com targetedonc.com/view/phase-2b-3-mo-trans-trial-of-mocravimod-enrolls-first-patients-with-aml

Phase 2b/3 MO-TRANS Trial of Mocravimod Enrolls First ... The first patients with acute myeloid leukemia (AML) have been enrolled in the pivotal phase 2b/3 MO-TRANS trial (NCT05429632) of mocravimod.

6. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5774/527246/Clinical-Assessment-of-Mocravimod-As-a-Victim-of

Clinical Assessment of Mocravimod As a Victim of Drug-Drug ... Mocravimod (MOC), a novel sphingosine-1-phosphate receptor (S1PR) modulator is predominantly metabolized via the CYP3A4 pathway based upon in vitro data.

7. aml-hub.com aml-hub.com/medical-information/mocravimod-granted-orphan-drug-designation-by-the-fda

Mocravimod granted Orphan Drug Designation by the FDA The safety and efficacy of mocravimod, both as an adjunctive and maintenance therapy, for patients with acute myeloid leukemia receiving allo- ...

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36343893/

Mocravimod, a Selective Sphingosine-1-Phosphate ... Our results indicate that mocravimod is safe and support a larger study to investigate its efficacy in a homogeneous acute myelogenous leukemia patient ...