Mocravimod as Adjunctive and Maintenance Treatment in Patients with AML Undergoing Allo-HCT (MO-TRANS Study)
Status
Active
ClinicalTrials.gov ID
NCT05429632
Study Title
Randomized, double-blind, placebo-controlled, multicenter Phase III study to evaluate the efficacy and safety of mocravimod as adjunctive and maintenance treatment in adult AML patients undergoing allogeneic HCT.
Study Overview
Allogeneic HCT is a standard treatment of curative intent for AML. A major limitation for successful outcome of allogeneic HCT is disease relapse. The graft-versus-leukemia (GvL) effect is also critical to prevent disease relapse and is mediated by donor T cells contained in the HCT graft that trigger immune responses against leukemic cells.
Mocravimod is an oral S1P receptor (S1PR) modulator that blocks lymphocyte egress from lymphoid organs via its phosphorylated active metabolite. Mocravimod’s potential to preserve the desirable GvL effect while decreasing GvHD is a promising way to maintain the prospect of a cure while decreasing transplant-related mortality (TRM) and morbidity in AML patients undergoing allogeneic HCT.
Study Design
This is a multicenter, randomized, double-blinded, placebo-controlled trial.
Purpose
The purpose of this pivotal study is to assess the efficacy and safety of mocravimod compared to standard of care as an adjunctive and maintenance treatment for allogeneic hematopoietic cell transplantation (HCT). Mocravimod is administered in addition to standard of care GvHD prophylaxis in adult high-risk AML patients.
Who May Participate
Age & Sex
Males and females
Age 18-75
No healthy volunteers
Diagnosis
Adult AML undergoing first allogeneic HCT
Conditions
High or intermediate risk AML in CR1,
or any risk AML in CR2
Study Details
Status
Active
Locations
Study Type
Study Phase
Phase III
Product
Oral mocravimod
Sponsor
Eligibility
Inclusion
- AML (WHO 2022)
– ELN high-risk or intermediate-risk in CR1, or
– any ELN risk in CR2 - Planned to undergo 1st allo-HCT:
– Planned use of a related or unrelated donor or with no more than 1 antigen mismatch or planned use of a haploidentical donor
– Planned use of G-CSF mobilized peripheral blood stem cells
– Planned use of a conditioning regimen with a transplant conditioning score (TCI) of > 1.5
– Planned use of TAC-based GvHD prophylaxis - Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Male or female, age ≥ 18 years and ≤ 75 years.
- Body weight of ≥ 50 kg.
- Willingness to use highly effective contraception methods
Exclusion
- Planned CsA, ATG, alemtuzumab, or abatacept for GvHD prophylaxis
- Diabetes mellitus
- History or active macular edema and/or uveitis
- Significant cardiac dysfunction (EF <40%, NYHA II-IV, QTc >450/470 ms)
- Requirement of the following treatments for cardiac dysfunction without option of treatment pause at the start of IMP treatment:
– Beta blockers, verapamil-type and diltiazem-type calcium channel blockers, or cardiac glycosides), or treatment with quinidine. - Pulmonary dysfunction (SpO₂ <90%; DLCO or FEV1 <50%) Hepatic or renal dysfunction (ALT/AST >2.5×ULN; CrCl <45 mL/min)
- Active clinically significant infection (viral, bacterial, or fungal)
- History of or active infection with HIV, HBV, or HCV
- Breastfeeding or positive pregnancy test
- Participation in another interventional clinical trial
- All prior induction, consolidation, or maintenance treatment must be terminated one day before ICF signature at the latest
Study Locations
Clinical Trial
Expanded Access Policy – Access to investigational medicinal product
Priothera is late-stage clinical development biotech company and is developing mocravimod, an investigational medicinal product, in a large clinical study for the treatment of patients with acute myeloid leukemia undergoing allogeneic hematopoietic cell transplantation; a rare, serious and life-threatening disease.
Priothera’s primary objective is to generate robust clinical data to support regulatory decision-making and, if successful, to obtain marketing authorization from relevant health authorities, including the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA).
In line with the position of health authorities, investigational medicinal products should, whenever feasible, be accessed within the context of a clinical trial. Clinical trials are essential to establish the safety and efficacy of new therapies and to ensure that patients receive appropriate medical oversight.
Information on ongoing clinical trials, including eligibility criteria, is available through public registries such as www.clinicaltrials.gov.
Priothera recognizes that some patients may not be eligible for participation in clinical trials and may seek access to investigational therapies through expanded access (also referred to as compassionate use) programs. At the current stage of development, Priothera does not offer expanded access to mocravimod, as the available data are not sufficient to support such use outside of controlled clinical trials.
This policy will be reviewed periodically and may be updated as additional clinical data become available and the development program progresses.