Acute Myeloid Leukemia in RemissionPhase 2NCT04232241
Matched Unrelated vs. Haploidentical Donor for Allogeneic Stem Cell Transplantation in Patients With Acute Leukemia
A Phase 2 study testing Allogeneic Stem Cell Transplantation in people with Acute Myeloid Leukemia in Remission and Acute Lymphoblastic Leukemia in Remission, run by Universitätsklinikum Hamburg-Eppendorf at 24 sites in 7 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia in Remission, Acute Lymphoblastic Leukemia in Remission, Myelodysplastic Syndromes
- What is being tested?
- Allogeneic Stem Cell Transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 167 participants
- Where
- 24 sitesin 7 countries
- Study dates
- 14 Nov 2019to 26 Nov 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Patients age: 18 - 70 years at time of inclusion (female and male).
- Patients understand and voluntarily sign an informed consent form.
- ECOG ≤ 2.
- 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.
- Females/Males who agree to comply with the applicable contraceptive requirements of the protocol.
Who cannot join
- 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
- Positive serology for HIV.
Show all 13Show fewer
- Pregnant or lactating women (positive serum pregnancy test).
- Age < 18 and ≥ 71 years.
- Uncontrolled invasive fungal infection at time of screening (baseline).
- Serious psychiatric or psychological disorders.
- Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment.
- Uncontrolled severe autoimmune disease or uncontrolled other malignancy.
- Availability of an HLA-identical sibling as donor source.
Trial locations and contacts
24 sites in 7 countries.
24 sites in 7 countries
Germany9 sites
University Hospital Düsseldorf
Düsseldorf, 40225
Universitätsklinikum Essen
Essen, 45122
Universitätsklinikum Frankfurt am Main | Medizinische Klinik II
Frankfurt, 60590
Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106
Universitätsklinikum Hamburg-Eppendorf
Hamburg, 20246
Medizinische Hochschule Hannover
Hanover, 30625
Universitätsklinikum Leipzig Dep. Innere Medizin, Neurologie und Dermatologie Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, Hämostaseologie
Leipzig, 04103
Universitätsklinikum Münster
Münster, 48149
Universitätsklinikum Tübingen
Tübingen, 72076
Spain8 sites
Hospital Universitari Germans Trias I Pujol
Badalona, 08916
Hospital Clínico y Provincial de Barcelona
Barcelona, 08036
Hospital de la Santa Creu I Sant Pau
Barcelona, 08041
Hospital General Universitario Gregorio Marañón
Madrid, 28007
Hospital Universitario de Salamanca
Salamanca, 37007
Hospital Universitario Virgen del Rocío
Seville, 41013
Hospital Clínico Universitario de Valencia
Valencia, 46010
Hospital Universitario y Politécnico de La Fe
Valencia, 46026
Austria3 sites
LKH-Univ. Klinikum Graz
Graz, 8036
Medizinische Universität Innsbruck
Innsbruck, A-6020
Medizinische Universität Wien, Universitätsklinik für Innere Medizin I Einrichtung für Stammzelltransplantation KMT
Vienna, A-1090
Czechia1 site
Institute of Hematology and Blood Transfusion
Prague, 128 20 Praha 2
Russia1 site
Pavlov First Saint Petersburg State Medical University
Saint Petersburg, 197022
About this study
Official title: Matched Unrelated vs. Haploidentical Donor for Allogeneic Stem Cell Transplantation in Patients With Acute Leukemia With Identical GVHD Prophylaxis - A Randomized Prospective European Trial.
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%.
Read the detailed 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.
- Conditions
- Acute Myeloid Leukemia in Remission, Acute Lymphoblastic Leukemia in Remission, Myelodysplastic Syndromes
- Being tested
- Allogeneic Stem Cell Transplantation
- Keywords
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Allogeneic Stem Cell Transplantation, Matched Unrelated Allogeneic Stem Cell Transplantation, Haploidentical Allogeneic Stem Cell Transplantation, anti-leukemic activity, Cyclophosphamide as GVHD prophylaxis
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Stem Cell TransplantationDrug
Allogeneic Stem Cell Transplantation
| Study group | Role | What they receive |
|---|---|---|
| Treatment A | Active comparator | Allogeneic stem cell transplantation from 10/10 HLA matched unrelated donor Receives: Drug: Allogeneic Stem Cell Transplantation |
| Treatment B | Experimental | Allogeneic stem cell transplantation from haploidentical donor Receives: Drug: Allogeneic Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse incidence at two years between both arms
The primary efficacy endpoint will be analyzed using cumulative incidence estimation to assess the subdistribution hazard rates for both treatment groups at two years after accounting for competing risk events.
Measured: 2 years
Secondary outcomes (11)
Overall survival at two years between both arms
The overall survival at two years between both arms will be presented with Kaplan-Meier's estimates of survival.
Measured: 2 years
Overall survival for all patients assigned to one of the two treatment arms as time to event endpoint
The overall survival for all patients will be presented with Kaplan-Meier curve. To compare the survival distributions between two arms, log-rank test will be performed, and two-sided p-values will be presented.
If applicable, Cox regression model stratified by the types of leukemia, types of complete remission and conditioning will be performed as sensitivity analysis.
Measured: through study completion, an average of two yeras
Comparison of GVHD/relapse-free survival as Composite endpoint in both arms
The rate of composite endpoint in both arms will be analyzed with the same methods as for the overall survival at two years between both arms.
Measured: Starting at day +30 (+/- 3 d) to 24 months (+/- 1 mo) after allogenic stem cell transplantation (SCT)
Comparison of non-relapsed mortality (NRM) at 1 and 2 years after allogeneic SCT in both arms
Due to the existence of competing risk events (persisting disease and relapse), NRM of each arm at 1 and 2 years after allogeneic SCT will be analyzed with the same methods as for the primary endpoint
Measured: At 1 and 2 years after allogeneic SCT
Comparison of acute graft-versus-host disease (aGVHD) on day +100 and 1 year (max grade) after allogeneic SCT according to the Glucksberg scale revised by Przepiorka et al. between both arms
For each time point, the frequency and percentage of aGVHD (maximum grade) of each arm will be presented. To compare the difference between both arms, logistic regression adjusted for covariates and stratification factors will be performed.
Measured: On day +100 and 1 year (max grade) after allogeneic SCT
Comparison of chronic graft-versus-host disease (cGVHD) according to the NIH consensus criteria of Jagasia et al. at 1 and 2 years after allogeneic SCT between both arms
For each time point, the frequency and percentage of cGVHD of each arm will be presented. To compare the difference between both arms, logistic regression adjusted for the stratification factors will be performed.
Measured: At 1 and 2 years after allogeneic SCT
Comparison of toxicity of both regimens scored according to the current version of the NCI CTCAE between both arms
Safety will be analyzed with frequency of patients with AEs as described above.
Measured: through study completion, an average of two yeras
Comparison of immune reconstitution between both arms
Frequency and percentage of patients having immune reconstitution in two arms will be provided. For the comparison between two arms, logistic regression adjusted for the stratification factors will be performed.
Measured: At day 30, 100, 6 months, 1 year and 2 years after allogenic SCT
Comparison of full donor chimerism between both arms
Frequency and percentage of patients having full donor chimerism in two arms will be provided. For the comparison between two arms, logistic regression adjusted for the stratification factors will be performed.
Measured: At day 30, 100, 6 months, 1 year and 2 years after allogenic SCT
Evaluation of Sorror Risk Score on outcome after allogeneic SCT
Comorbidity score after Sorror will be assessed prior to randomization and outcome in both arms will be analyzed according the pre-transplant Sorror score.
Measured: At baseline
Comparison of QOL (FACT-BMT) before and after transplantation at + 100 days, 6 months, 1 year, 2 years between both arms
The means of change in scores at each time point (day 100, 6 months, 1 year and 2 years after transplantation respectively) from baseline and the confidence intervals of each arm will be presented. To compare the difference in QOL scores between both arms, logistic regression adjusted for covariates and stratification factors will be performed for each time point.
Measured: At day 100, 6 months, 1 year and 2 years after allogenic SCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2020
- Study start14 Nov 2019
- Primary completion22 Nov 2026 (estimated)
- Study completion26 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia in RemissionStudies of the same condition, open and under way.
- More trials run by Universitätsklinikum Hamburg-EppendorfThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 167 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universitätsklinikum Hamburg-Eppendorf
- Sponsor type
- University, hospital or other
- Collaborators
- Gesellschaft für Therapieforschung mbH, Staburo GmbH
- Responsible party
- Sponsor
- Investigators
- Nicolaus Kröger, Prof. Dr., Principal investigator, University Medical Center Hamburg-Eppendorf, Department of Stem Cell Transplantation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04232241
- Sponsor's protocol ID
- HaploMUDStudy
- Status last verified
- Apr 2025
- First submitted
- 2 Jan 2020
- Last updated
- 22 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sanz J, Galimard JE, Labopin M, Afanasyev B, Sergeevich MI, Angelucci E, Kroger N, Koc Y, Ciceri F, Diez-Martin JL, Arat M, Sica S, Rovira M, Aljurf M, Tischer J, Savani B, Ruggeri A, Nagler A, Mohty M. Post-transplant cyclophosphamide containing regimens after matched sibling, matched unrelated and haploidentical donor transplants in patients with acute lymphoblastic leukemia in first complete remission, a comparative study of the ALWP of the EBMT. J Hematol Oncol. 2021 May 28;14(1):84. doi: 10.1186/s13045-021-01094-2.
PubMed 34049582
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Matched Unrelated vs. Haploidentical Donor for Allogeneic Stem Cell Transplantation in Patients With Acute Leukemia With Identical GVHD Prophylaxis - A Randomized Prospective European Trial.
- ClinicalTrials.gov ID
- NCT04232241
- Acronym / sponsor ID
- HaploMUDStudy
- Conditions
- Acute Myeloid Leukemia in Remission, Acute Lymphoblastic Leukemia in Remission, Myelodysplastic Syndromes
- Keywords
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Allogeneic Stem Cell Transplantation, Matched Unrelated Allogeneic Stem Cell Transplantation, Haploidentical Allogeneic Stem Cell Transplantation, anti-leukemic activity, Cyclophosphamide as GVHD prophylaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 167 participants (actual)
- Lead sponsor
- Universitätsklinikum Hamburg-EppendorfUniversity, hospital or other
- Collaborators
- Gesellschaft für Therapieforschung mbH, Staburo GmbH
- Study start
- 14 Nov 2019 (actual)
- Primary completion
- 22 Nov 2026 (estimated)
- Study completion
- 26 Nov 2026 (estimated)
- First posted
- 18 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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%.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment A | Active comparator | Drug: Allogeneic Stem Cell TransplantationAllogeneic stem cell transplantation from 10/10 HLA matched unrelated donor |
| Treatment B | Experimental | Drug: Allogeneic Stem Cell TransplantationAllogeneic stem cell transplantation from haploidentical donor |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
- Patients age: 18 - 70 years at time of inclusion (female and male).
- Patients understand and voluntarily sign an informed consent form.
- ECOG ≤ 2.
- 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.
Key exclusion criteria (13 listed)
- Severe renal, hepatic, pulmonary or cardiac disease, such as:
- Positive serology for HIV.
- Pregnant or lactating women (positive serum pregnancy test).
- Age < 18 and ≥ 71 years.
- Uncontrolled invasive fungal infection at time of screening (baseline).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Stem Cell TransplantationAllogeneic Stem Cell Transplantation | Drug | Treatment A, Treatment B |
Primary outcome
Relapse incidence at two years between both arms
Time frame: 2 years
The primary efficacy endpoint will be analyzed using cumulative incidence estimation to assess the subdistribution hazard rates for both treatment groups at two years after accounting for competing risk events.
11 secondary outcomes
Overall survival at two years between both arms
Time frame: 2 years
Overall survival for all patients assigned to one of the two treatment arms as time to event endpoint
Time frame: through study completion, an average of two yeras
Comparison of GVHD/relapse-free survival as Composite endpoint in both arms
Time frame: Starting at day +30 (+/- 3 d) to 24 months (+/- 1 mo) after allogenic stem cell transplantation (SCT)
Comparison of non-relapsed mortality (NRM) at 1 and 2 years after allogeneic SCT in both arms
Time frame: At 1 and 2 years after allogeneic SCT
Comparison of acute graft-versus-host disease (aGVHD) on day +100 and 1 year (max grade) after allogeneic SCT according to the Glucksberg scale revised by Przepiorka et al. between both arms
Time frame: On day +100 and 1 year (max grade) after allogeneic SCT
Comparison of chronic graft-versus-host disease (cGVHD) according to the NIH consensus criteria of Jagasia et al. at 1 and 2 years after allogeneic SCT between both arms
Time frame: At 1 and 2 years after allogeneic SCT
Comparison of toxicity of both regimens scored according to the current version of the NCI CTCAE between both arms
Time frame: through study completion, an average of two yeras
Comparison of immune reconstitution between both arms
Time frame: At day 30, 100, 6 months, 1 year and 2 years after allogenic SCT
Comparison of full donor chimerism between both arms
Time frame: At day 30, 100, 6 months, 1 year and 2 years after allogenic SCT
Evaluation of Sorror Risk Score on outcome after allogeneic SCT
Time frame: At baseline
Comparison of QOL (FACT-BMT) before and after transplantation at + 100 days, 6 months, 1 year, 2 years between both arms
Time frame: At day 100, 6 months, 1 year and 2 years after allogenic SCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2025.
- Derived from this study: PubMed 34049582Sanz J, Galimard JE, Labopin M, Afanasyev B, Sergeevich MI, Angelucci E, Kroger N, Koc Y, Ciceri F, Diez-Martin JL, Arat M, Sica S, Rovira M, Aljurf M, Tischer J, Savani B, Ruggeri A, Nagler A, Mohty M. Post-transplant cyclophosphamide containing regimens after matched sibling, matched unrelated and haploidentical donor transplants in patients with acute lymphoblastic leukemia in first complete remission, a comparative study of the ALWP of the EBMT. J Hematol Oncol. 2021 May 28;14(1):84. doi: 10.1186/s13045-021-01094-2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nicolaus Kröger, Prof. Dr. principal investigator
University Medical Center Hamburg-Eppendorf, Department of Stem Cell Transplantation
24 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Stem Cell Transplantation in people with Acute Myeloid Leukemia in Remission and Acute Lymphoblastic Leukemia in Remission, run by Universitätsklinikum Hamburg-Eppendorf at 24 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Germany, Spain, Austria, Czechia, Finland and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relapse incidence at two years between both arms (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04232241, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2025.