Matched Unrelated vs. Haploidentical Donor for Allogeneic Stem Cell Transplantation in Patients With Acute Leukemia

Universitätsklinikum Hamburg-Eppendorf

Status and phase: Active, not recruiting; Phase 2

Conditions: Acute Myeloid Leukemia in Remission; Acute Lymphoblastic Leukemia in Remission; Myelodysplastic Syndromes

Treatments: Drug: Allogeneic Stem Cell Transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT04232241

HaploMUDStudy

Details and patient eligibility

About

Primary objective of this open label, two-arm, multicenter, multinational, randomized trial is to compare anti-leukemic activity of allogeneic stem cell transplantation for patients with acute leukemia in complete remission between a 10/10 HLA matched unrelated donor and a haploidentical donor.

The hypothesis: Haploidentical stem cell transplantation with post cyclophosphamide induces a stronger anti-leukemic activity in comparison to 10/10 HLA matched unrelated donor and reduces the risk of relapse at 2 years after stem cell transplantation by 10%.

Full description

Secondary objectives are to assess and compare the safety and efficacy of study treatments therapy in both study arms on non-relapse mortality (NRM), relapse-free survival (RFS), Overall survival (OS), QOL, toxicity, development of acute and chronic GvDH as well as engraftment and chimerism and impact of measurable residual disease.

Enrollment: 167 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

1. Acute Myeloid Leukemia (AML) intermediate or high risk according to ELN or Acute Lymphoblastic Leukemia (ALL) high risk according to ESMO guidelines in 1. CR or AML/ALL in 2. CR, or high risk MDS (according to IPSS-R) in 1. CR or 2. CR.
2. Patients age: 18 - 70 years at time of inclusion (female and male).
3. Patients understand and voluntarily sign an informed consent form.
4. ECOG ≤ 2.
5. 10/10 HLA-matched unrelated donor and haploidentical (≥ 5/10 and ≤ 8/10 HLA) relative matched donor available at least 4 weeks after completion of induction and/or consolidation therapy.
6. Females/Males who agree to comply with the applicable contraceptive requirements of the protocol.

Exclusion Criteria

1. Severe renal, hepatic, pulmonary or cardiac disease, such as:

   • total bilirubin, SGPT or SGOT > 3 times upper the normal level
   • left ventricular ejection fraction < 30 %
   • creatinine clearance < 30 ml/min
   • DLCO < 35 % and/or receiving supplementary continuous oxygen

2. Positive serology for HIV.

3. Pregnant or lactating women (positive serum pregnancy test).

4. Age < 18 and ≥ 71 years.

5. Uncontrolled invasive fungal infection at time of screening (baseline).

6. Serious psychiatric or psychological disorders.

7. Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment.

8. Uncontrolled severe autoimmune disease or uncontrolled other malignancy.

9. Availability of an HLA-identical sibling as donor source.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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167 participants in 2 patient groups

Treatment A

Active Comparator group

Description:

Allogeneic stem cell transplantation from 10/10 HLA matched unrelated donor

Treatment:

Drug: Allogeneic Stem Cell Transplantation

Treatment B

Experimental group

Description:

Allogeneic stem cell transplantation from haploidentical donor

Treatment:

Drug: Allogeneic Stem Cell Transplantation