A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance After Allo-HSCT

NCT07559695 | Recruiting

• 1 other identifier: menin inhibitors maintenance
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances. Patients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.

Conditions

Menin Inhibitors; Acute Leukemia; Maintenance Therapy; KMT2A Rearrangement; NPM1 Mutation; NUP98 Gene Rearrangement; Post Hematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• 2-year rate of RFS

  It is measured as the proportions of numbers of patients with hematologic relapse to the numbers of the overall enrolled patients at 2 years from the first day of taking menin inhibitor post allo-HSCT.

  2 years

Secondary Outcomes (5)

• OS

  2 years

• EFS

  2 years

• CIR

  2 years

• NRM

  2 years

• Incidence and severity of GVHD

  2 years

Other Outcomes (1)

• Pre-/post-transplant MRD dynamics

  2 years

Study Arms (1)

Post-alloHSCT menin maintenance

Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) and received menin inhibitor maintenance therapy after transplantation. Menin inhibitors include but are not limited to Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors.

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with acute leukemia who underwent allogeneic hematopoietic stem cell transplantation and received post-transplant menin inhibitor maintenance therapy.

You may qualify if:

• Age ≥ 15 years.
• Patients diagnosed with acute leukemia (including AML, ALL, and MPAL) according to the World Health Organization (WHO 2022) criteria.
• Must meet one of the following characteristics: a. Harboring an NPM1 gene mutation (without concurrent FLT3-ITD or FLT3-TKD mutation); b. Harboring a KMT2A gene rearrangement or KMT2A-PTD; c. Harboring a NUP98 gene rearrangement; d. Other acute leukemia subtypes dependent on the menin-KMT2A interaction, if evidenced, may be enrolled upon discussion with and approval from the principal research team.
• Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), with ≥ 30 days elapsed since the date of graft infusion.
• Received menin inhibitor maintenance therapy after allo-HSCT and meets the following conditions: a. Received at least ≥ 2 complete cycles (7 days per cycle) of menin inhibitor therapy, or cumulative medication duration ≥ 14 days; b. Patient was in a state of CR/CRh/CRi at the initiation of maintenance therapy.
• No evidence of leukemia relapse during menin inhibitor maintenance therapy, defined as: a. Bone marrow blasts \< 5%, and blasts do not exhibit morphological features of acute leukemia (e.g.Auer rods); b. No evidence of extramedullary leukemia (e.g. CNS leukemia or myeloid sarcoma).
• The menin inhibitors used include but are not limited to: Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors.
• Capable of understanding and voluntarily signing the informed consent form.
• Complete clinical data.

You may not qualify if:

• Presence of any of the following at the initiation of menin inhibitor maintenance therapy (including within 28 days prior to starting treatment): a. Morphologic relapse in bone marrow (bone marrow blasts ≥ 5%); b. Presence of leukemic cells in peripheral blood.
• Active infection that is deemed uncontrolled by the investigator.
• Severe organ dysfunction, including: a. Hepatic impairment: ALT or AST ≥ 5 × ULN (Upper Limit of Normal), or total bilirubin ≥ 3 × ULN; b. Severe renal impairment: eGFR \< 30 ml/min/1.73 m²; c. Cardiac dysfunction: NYHA (New York Heart Association) Class III-IV.
• Concurrent acute graft-versus-host disease (aGVHD) ≥ Grade 2 or chronic graft-versus-host disease (cGVHD) ≥ Grade 3, requiring corticosteroids ≥ 1 mg/kg and ≥ 3 types of immunosuppressive therapy (including CNI, ruxolitinib, belumosudil, etc.).
• History of other malignancies requiring ongoing treatment (except for malignancies that have undergone curative treatment or are assessed to be in complete remission and require no systemic maintenance therapy or radiotherapy).
• Any gastrointestinal disorder that may affect the intake or absorption of oral medications (e.g.dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal GVHD, etc).
• Severely missing clinical data, precluding efficacy or safety assessment.
• Receipt of other maintenance therapies, including but not limited to hypomethylating agents, donor lymphocyte infusion (DLI), or other specific small-molecule targeted drugs.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

RECRUITING

Study Officials

• Su-ning Chen, M.D.

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Su-ning Chen, M.D.

CONTACT

(0086)13814881746; chensuning@sina.com

Hai-ping Dai, M.D.

CONTACT

(0086)13914086271; daihaiping8@126.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: April 24, 2026
• First Posted: April 30, 2026
• Study Start: November 1, 2025
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: April 30, 2026

Record last verified: 2026-04

Locations

CN (1)