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Leukemia, Myeloid, AcutePhase 2NCT05565105

CD34+ Transplants for Myeloid Malignancies

A Phase 2 study testing Busulfan, Melphalan and Fludarabine in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Guenther Koehne at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

This study will evaluate whether processing blood stem cell transplants using an investigational device (the CliniMACS system) results in less complications for patients undergoing transplant for treatment of a blood malignancy (cancer) or blood disorder. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Busulfan, Melphalan and Fludarabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
1 siteUnited States
Study dates
Jan 2027to Jan 2034
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

  • Malignant conditions or other life-threatening disorders correctable by transplant for which CD34+ selected, T-cell depleted allogeneic hematopoietic stem cell transplantation is indicated such as:
  • AML in 1st remission-- for patients whose AML does not have "good risk" cytogenetic features (i.e., t(8;21), (q22; q22.1); RUNX1-RUNX1T1, inv16).
  • Secondary AML in 1st remission
  • AML in 1st relapse or 2nd remission
  • CML not responding adequately to TKI therapy, CML in accelerated phase or blast crisis, or CML in CR after accelerated phase or blast crisis
  • Chronic myelomonocyte leukemia: CMML-1 and CMML-2.
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  • Intermediate to high-risk primary or secondary myelofibrosis
  • Patients aged ≥18 and ≤70 years old. Patients 65-70 years old will be examined as a separate cohort, since myeloablative regimens have been shown to be associated with increased morbidity, mortality, and toxicity in older patients, specifically, patients above 65 years of age (49).
  • Patients may be of either gender or any ethnic background
  • Patients must have an appropriately matched donor (MRD 8/8, MUD 8/8, or MMUD 7/8 [mismatch at HLA-A, B or DR-B1 locus])
  • Patients may be of any sex, gender, or ethnic background.
  • Patients must have a Karnofsky (adult) Performance Status of at least 70%
  • Patients must have adequate organ function measured by:
  • Cardiac: asymptomatic; left ventricular ejection fraction (LVEF) at rest must be ≥50%
  • Hepatic: < 3 × ULN AST and: < 1.5 × ULN total serum bilirubin, unless there is congenital benign hyperbilirubinemia or if the hyperbilirubinemia is directly caused by the disease in which the patient is receiving a transplant (e.g., AML Chloroma obstructing the biliary tree). Patients with higher bilirubin levels due to causes other than active liver disease are also eligible with Pl approval e.g., patients with PNH, Gilbert's disease or other hemolytic disorders.
  • Renal: serum creatinine: within normal range 0.7-1.3 mg/dL or if serum creatinine is outside the normal range, then CrCl > 45 mL/min (measured or calculated/estimated via Cockcroft-Gault formula).
  • Pulmonary: asymptomatic [but sustained DLCO ≥ 50% of predicted (corrected for hemoglobin)].
  • Each patient must be willing to participate as a research subject and must sign an informed consent form.

Who cannot join

  • Individuals who are pregnant or breast-feeding
  • Uncontrolled bacterial, viral, or fungal infections; or known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection
  • Patients cured of Hepatitis C for ≥3 years prior to enrollment are permitted
  • Patients with detectable hepatitis B surface antigen (HBsAg) will be excluded. Patients with viral hepatitis B core antibody (HBcAb) positivity, but absence of HBsAg, will be eligible if HBV DNA is undetectable and if they are willing to be on hepatitis B antiviral prophylaxis and undergo monitoring for HBV reactivation (every 12 weeks while on immune suppression) per institutional protocols. Patients will be referred promptly to hepatology specialist with the first detectable viral load.
  • Presence of leukemia in the CNS
  • Symptomatic cardiac disease (angina, congestive heart failure) and/or LVEF <50%.
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  • History of cardiovascular disease including but not limited to myocardial infarction, unstable angina, stroke, or transient ischemic attack within the 6 months prior to the initiation of study or congestive heart failure (New York Heart Association [NYHA] class III-IV)
  • Patients who subsequently receive a non-T-cell-depleted transplant
  • Stem cell count <3.0 × 10^6/kg after CD34+ selection
  • Known sensitivity to components of CliniMACS® Prodigy CD34 System
  • Have a history of other active malignancy(ies) unless the patient has been disease-free for at least 2 years and is deemed by the treating Investigator to be at low risk for recurrence of that malignancy; or the only prior cancer is curatively treated cervical cancer in situ, or basal cell or squamous cell carcinoma of the skin.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Guenther Koehne, MD, PhD contact

786-596-2000

GuentherK@baptisthealth.net

United States1 site

  • Baptist Health Herbert Wertheim Cancer Institute

    Miami, Florida, 33176

    Guenther Koehne, MD, Ph.D., 786-596-2000, GuentherK@baptisthealth.net

Other trials in United States

About this study

Official title: A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Myeloid Malignancies

This study will evaluate whether processing blood stem cell transplants using an investigational device (the CliniMACS system) results in less complications for patients undergoing transplant for treatment of a blood malignancy (cancer) or blood disorder.

Being tested
Busulfan, Melphalan, Fludarabine, CD34+ Allogeneic Peripheral Blood Stem Cell Transplant
Keywords
Stem Cell Transplant
Medical subject headings
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.

  • BusulfanDrug

    Also called Busulfex

    Busulfan: 0.8 mg/kg every 6 hours × 12 doses with dose modified according to pharmacokinetics

  • MelphalanDrug

    Also called Alkeran

    Melphalan: 70 mg/m²/day × 2

  • FludarabineDrug

    Also called Fludara

    Fludarabine: 25 mg/m²/day × 5

  • CD34+ Allogeneic Peripheral Blood Stem Cell TransplantProcedure or surgery

    Allogeneic human leukocyte antigen (HLA)-compatible related or unrelated transplants of peripheral blood stem cells (PBSC) depleted of T-cells by positive selection of CD34+ progenitor cells using the CliniMACS system

Study groups in NCT05565105
Study groupRoleWhat they receive
Busulfan/Melphalan/Fludarabine ConditioningExperimentalConditioning: * Busulfan, IV 0.8 mg/kg q6hours × 12 doses over 3 days * Melphalan,70 mg/m²/day × 2 days (or 50 mg/m²/day × 2 days for patients ≥65 years with a comorbidity index score of ≥3) via IV infusion over 30 minutes daily * Fludarabine, 25 mg/m²/day × 5 days via IV infusion over 30 minutes daily CD34+ Allogenic Peripheral Blood Stem Cell Transplant
Receives: Drug: Busulfan; Drug: Melphalan; Drug: Fludarabine; Procedure: CD34+ Allogeneic Peripheral Blood Stem Cell Transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall survival (OS)

    OS is defined as time from transplant to death due to any cause

    Measured: 2 years

Secondary outcomes (5)

  1. Disease-Free Survival (DFS)

    DFS is defined as the time from study treatment (transplant) to relapse, progression, or death from any cause, whichever occurs first.

    Measured: 2 years

  2. Transplant-Related Mortality (TRM)

    TRM is defined as the percentage of participants whose cause of death was related to the transplant.

    Measured: 2 years

  3. Acute Graft versus Host Disease (GvHD)

    Acute GvHD outcome is defined as the percentage of participants who develop acute GvHD

    Measured: 2 years

  4. Chronic GvHD

    Chronic GvHD outcome is defined as the percentage of participants who develop chronic GvHD

    Measured: 2 years

  5. Proportion of patients optimal and suboptimal doses

    The proportion of patients receiving optimal cell doses (CD34+: >5 × 10^6/kg and CD3+: < 5 × 10^4/kg) and suboptimal doses (CD34+: <3 x 10^6/kg and CD3+: >5 × 10^4/kg).

    Measured: Day 0

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Oct 2022
  2. Study startJan 2027 (estimated)
  3. Primary completionJan 2034 (estimated)
  4. Study completionJan 2034 (estimated)

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05565105
Sponsor's protocol ID
2021-KOE-001
Status last verified
Sep 2026
First submitted
28 Sep 2022
Last updated
13 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes
Individual data sharing
No

Publications

  • Aversa F, Terenzi A, Tabilio A, Falzetti F, Carotti A, Ballanti S, Felicini R, Falcinelli F, Velardi A, Ruggeri L, Aloisi T, Saab JP, Santucci A, Perruccio K, Martelli MP, Mecucci C, Reisner Y, Martelli MF. Full haplotype-mismatched hematopoietic stem-cell transplantation: a phase II study in patients with acute leukemia at high risk of relapse. J Clin Oncol. 2005 May 20;23(15):3447-54. doi: 10.1200/JCO.2005.09.117. Epub 2005 Mar 7.

    PubMed 15753458

  • Handgretinger R, Klingebiel T, Lang P, Schumm M, Neu S, Geiselhart A, Bader P, Schlegel PG, Greil J, Stachel D, Herzog RJ, Niethammer D. Megadose transplantation of purified peripheral blood CD34(+) progenitor cells from HLA-mismatched parental donors in children. Bone Marrow Transplant. 2001 Apr;27(8):777-83. doi: 10.1038/sj.bmt.1702996.

    PubMed 11477433

  • Urbano-Ispizua A, Rozman C, Pimentel P, Solano C, de la Rubia J, Brunet S, Perez-Oteyza J, Ferra C, Zuazu J, Caballero D, Bargay J, Carvalhais A, Diez JL, Espigado I, Alegre A, Rovira M, Campilho F, Odriozola J, Sanz MA, Sierra J, Garcia-Conde J, Montserrat E; Spanish Group for Allogeneic Peripheral Blood Transplantation (Grupo Espanol de Trasplante Hemopoyetico) and Instituto Portugues de Oncologia-Porto. Risk factors for acute graft-versus-host disease in patients undergoing transplantation with CD34+ selected blood cells from HLA-identical siblings. Blood. 2002 Jul 15;100(2):724-7. doi: 10.1182/blood-2001-11-0057.

    PubMed 12091376

  • Jakubowski AA, Small TN, Young JW, Kernan NA, Castro-Malaspina H, Hsu KC, Perales MA, Collins N, Cisek C, Chiu M, van den Brink MR, O'Reilly RJ, Papadopoulos EB. T cell depleted stem-cell transplantation for adults with hematologic malignancies: sustained engraftment of HLA-matched related donor grafts without the use of antithymocyte globulin. Blood. 2007 Dec 15;110(13):4552-9. doi: 10.1182/blood-2007-06-093880. Epub 2007 Aug 23.

    PubMed 17717135

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Busulfan, Melphalan and Fludarabine in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Guenther Koehne at 1 site in United States.

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 18 inclusion and 11 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: opening soon, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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?

Contact Guenther Koehne, MD, PhD (786-596-2000, GuentherK@baptisthealth.net), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall survival (OS) (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 NCT05565105, which also lists 4 linked publications.

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 13 Oct 2026.

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