A phase III placebo-controlled efficacy and safety study of mocravimod as an adjunctive and maintenance treatment in AML patients undergoing allo-HCT (MO-TRANS Study)
- Conditions
- Adjunctive and maintenance treatment to HCTMedDRA version: 21.0Level: LLTClassification code 10000886Term: Acute myeloid leukemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2021-002864-36-DE
- Lead Sponsor
- Priothera S.A.S.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 249
1.Subjects with a diagnosis of AML (excluding acute promyelocytic
leukemia) according to the World Health Organization (WHO) 2022
classification of AML and related precursor neoplasms, including therapy
AML with myelodysplasia related gene mutations.
2.Subjects with European LeukemiaNet (ELN) high risk or intermediate
risk or AML in CR1, or AML of any risk in CR2. (Complete remission with
incomplete count recovery [CRi] is also allowable).
- Complete remission is defined as: < 5% marrow blasts by morphologic
examination and no circulating peripheral blasts ; absence of
extramedullary disease; absolute neutrophil count = 1.0x109/L
(1000/µL); platelet count = 100x109/L (100 000/µL)
- CRi is defined as meeting all complete remission criteria except for
residual absolute neutropenia < 1000/µL and/or thrombocytopenia <
100 000/µL
3.Planned use of a related or unrelated donor or with no more than 1
antigen mismatch or planned use of a haploidentical donor
4.Life expectancy = 6 months at screening.
5.Eastern Cooperative Oncology Group (ECOG) performance status of 0
or 1
6.Male or female, age = 18 years and = 75 years.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 200
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 49
1.Planned use of anti-thymocyte globulin (ATG), alemtuzumab,
abatacept for GvHD prophylaxis.
2.Planned use of serotherapy during conditioning, including ATG and
alemtuzumab.
3.Planned ex vivo major graft manipulation, including T-cell depletion or
CD34+ selection.
4.Subjects having received prior allogeneic HCT or recipients of a solid
organ transplant.
5.Immunosuppressive drugs for concomitant disease. Subjects must be
able to be off prednisone (> 10 mg/day) or other immunosuppressive
medications for at least 3 days prior to the start of treatment of the
study. Physiologic replacement dosing of hydrocortisone is permissible.
6.Require treatments for cardiac dysfunction
7.Subjects with acute promyelocytic leukemia.
8.Blast crisis of chronic myeloid leukemia
9.Cardiac dysfunction
10.Pulmonary dysfunction.
11.Significant liver disease or liver injury or known history of alcohol
abuse, chronic liver or biliary disease. Hepatic dysfunction as defined by
aspartate aminotransferase (AST) and/or alanine aminotransferase
(ALT) > 2.5 x upper limit of normal (ULN); or total bilirubin > 1.5 x ULN
12.Renal dysfunction with creatinine clearance < 45 mL/min by the
Cockcroft-Gault formula.
13.History of stroke or intracranial hemorrhage within 1 year prior to
screening.
14.Active clinically significant infection (viral, bacterial, or fungal) that
requires ongoing antimicrobial therapy and in the judgment of the
investigator represents a risk to proceeding with HCT.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To compare the efficacy of mocravimod to that of placebo;Secondary Objective: To compare mocravimod's effect on overall survival (OS) to that of placebo;Primary end point(s): Relapse-free survival (RFS);Timepoint(s) of evaluation of this end point: 1° EP will be assessed once 46 events (relapse or death of any cause)<br>have been reached or once all subjects have completed their EOT visit,<br>whichever comes last. (EOT – End-of-Treatment)
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Key 2° Endpoints:<br>-Overall Survival (OS) - mocravimod's effect on OS to that to placebo<br>- RFS at EOS (End-of-study) - to assess the sustained efficacy of moc in<br>comparison to placebo<br>-Survival free from Grade III/IV aGvHD at 12 mo & time to acute GvHD<br>-Survival free from moderate /severe cGvHD at EOS & time to cGvHD<br>-GRFS (GvHD-free, Relapse-free survival at 12 mo and at EOS);Timepoint(s) of evaluation of this end point: Event-driven trial