A Phase 1, Multicenter, Open-Label Study of REM-422, an MYB mRNA Degrader, in Patients With Relapsed/Refractory AML or Higher-Risk MDS

2023-510479-72-00 Protocol REM-422-102 Human pharmacology (Phase I) - Other Ongoing, recruitment ended

Start 5 Aug 2025 · Status Ongoing, recruitment ended · 1 EU/EEA countries · 4 sites · Protocol REM-422-102

Overview

Sponsor-declared trial summary

Phase Human pharmacology (Phase I) - Other
Status Ongoing, recruitment ended
Participants planned 80
Countries 1
Sites 4

Relapsed/Refractory AML or Higher-Risk MDS

Key facts

Sponsor
Remix Therapeutics Inc.
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Neoplasms [C04], Diseases [C] - Hemic and Lymphatic Diseases [C15]
Trial duration
5 Aug 2025 → ongoing
Decision date (initial)
2024-07-08
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Conditions and MedDRA coding

Relapsed/Refractory AML or Higher-Risk MDS

VersionLevelCodeTermSystem organ class
20.0 LLT 10068361 MDS 10029104
20.0 LLT 10066572 AML progression 10029104
20.0 LLT 10001941 AML 10029104

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Remix Therapeutics Inc.

Sponsor organisation
Remix Therapeutics Inc.
Address
100 Forge Road Suite 400
City
Watertown
Postcode
02472-2265
Country
United States

Scientific contact point

Organisation
Remix Therapeutics Inc.
Contact name
Jennifer Webster

Public contact point

Organisation
Remix Therapeutics Inc.
Contact name
Jennifer Webster

Locations

1 EU/EEA country · 4 investigational sites

By country

CountryMS statusPlanned subjectsSites
France Ongoing, recruitment ended 20 4
Rest of world
United States
60

Investigational sites

France

4 sites · Ongoing, recruitment ended
Institut Gustave Roussy
Hématologie, 114 Rue Edouard Vaillant, 94800, Villejuif
Centre Hospitalier Universitaire De Bordeaux
Hématologie Clinique et Thérapie Cellulaire, Avenue De Magellan, 33600, Pessac
Assistance Publique Hopitaux De Paris
Hématologie - Immunologie, 1 Avenue Claude Vellefaux, 75010, Paris
Institut Universitaire Du Cancer Toulouse-Oncopole
Oncologie médicale - Unité de phases précoces, 1 Avenue Irene Joliot Curie, 31059, Toulouse Cedex 9

Country notifications

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

Country Trial startTrial end Recruitment startRecruitment end Early termination
France 2024-11-24 2024-12-04 2025-04-02

Oversight and notifications

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

Temporary halts 1 · Art. 38 CTR

Temporary halt TH-78735

Halt date
2025-04-02
Member states concerned
France
Publication date
2025-04-10
Reason
Sponsor decision, Safety related (clinical or pre-clinical results)
Explanation
As per REM-422-102 Study Stopping Rules (Section 11.1.1):
In the event of a participant death assessed as possibly related to REM-422, enrollment in the study will be paused and the DRC will examine the entirety of available data, encompassing safety, PK, Pd, and preliminary antitumor activity. The objective is to make a well-informed recommendation regarding whether to resume enrollment in the study or not.
Follow-up measures
Please check further details on attached notification letters.
Benefit-risk balance changed
Yes
Treatment stopped
No

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-03-27 France Acceptable with conditions
2024-07-01
2024-07-08
2 SUBSTANTIAL MODIFICATION SM-1 2024-07-10 France Acceptable
2024-09-23
2024-09-23
3 SUBSTANTIAL MODIFICATION SM-2 2024-12-17 France Acceptable
2025-02-25
2025-02-25
4 SUBSTANTIAL MODIFICATION SM-3 2025-03-31 France Acceptable
2025-05-14
2025-05-14
5 SUBSTANTIAL MODIFICATION SM-5 2025-05-28 France Acceptable with conditions
2025-08-04
2025-08-04
6 SUBSTANTIAL MODIFICATION SM-6 2025-08-13 France Acceptable
2025-09-05
2025-09-15
7 NON SUBSTANTIAL MODIFICATION NSM-1 2025-11-26 France Acceptable
2025-09-05
2025-11-26