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)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Priothera S.A.S.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

9 Sep 2025 → ongoing

Decision date (initial)

2024-08-08

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Priothera S.A.S.

External identifiers

EU CT number

2024-512752-38-00

EudraCT number

2021-002864-36

ClinicalTrials.gov

NCT05429632

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Efficacy, Others

1. To demonstrate the superior efficacy of mocravimod 3 mg to that of placebo in the TAC GvHD prophylaxis stratum
2. To demonstrate the superior efficacy of mocravimod 1 mg to that of placebo in the TAC GvHD prophylaxis stratum

Secondary objectives 3

1. 1. To demonstrate mocravimod’s 3 mg superior efficacy on overall survival (OS) to that of placebo in the TAC GvHD prophylaxis stratum.
2. 2. To demonstrate mocravimod’s 1 mg superior efficacy on overall survival (OS) to that of placebo in the TAC GvHD prophylaxis stratum
3. 3. To assess mocravimod’s effect on the occurrence of GvHD in comparison to placebo

Conditions and MedDRA coding

Adjunctive and maintenance treatment to HCT

Regulatory references

Scientific advice from competent authorities

Federal Institute For Drugs And Medical Devices, European Medicines Agency, Swedish Medical Products Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

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.

Exclusion criteria 14

1. Planned use of CsA, anti-thymocyte globulin (ATG), alemtuzumab, abatacept for GvHD prophylaxis.
2. Cardiac dysfunction
3. Pulmonary dysfunction.
4. 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
5. Renal dysfunction with creatinine clearance < 45 mL/min by the Cockcroft-Gault formula.
6. History of stroke or intracranial hemorrhage within 1 year prior to screening.
7. 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.
8. Planned use of serotherapy during conditioning, including ATG and alemtuzumab.
9. Planned ex vivo major graft manipulation, including T-cell depletion or CD34+ selection.
10. Subjects having received prior allogeneic HCT or recipients of a solid organ transplant.
11. 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.
12. Require treatments for cardiac dysfunction
13. Subjects with acute promyelocytic leukemia.
14. Blast crisis of chronic myeloid leukemia

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Relapse-free survival (RFS)

Secondary endpoints 5

1. Overall Survival (OS) - mocravimod's effect on OS to that to placebo
2. RFS at EOS (End-of-study) - to assess the sustained efficacy of moc in comparison to placebo
3. Survival free from Grade III/IV aGvHD at 12 mo & time to acute GvHD
4. Survival free from moderate /severe cGvHD at EOS & time to cGvHD
5. GRFS (GvHD-free, Relapse-free survival at 12 mo and at EOS)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Priothera S.A.S.

Sponsor organisation

Priothera S.A.S.

Address

57 Avenue Du General De Gaulle

City

St Louis

Postcode

68300

Country

France

Scientific contact point

Organisation

Priothera S.A.S.

Contact name

Stephan Oehen

Public contact point

Organisation

Priothera S.A.S.

Contact name

Karen von Graevenitz

Third parties 11

Locations

6 EU/EEA countries · 48 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 6 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 37 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

9 submissions — initial application plus substantial / non-substantial modifications