CD34+ Transplants for Leukemia and Lymphoma

NCT ID: NCT05565105

Last Updated: 2025-12-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-01-31

Study Completion Date

2032-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Leukemia, Myeloid, Acute Leukemia, Lymphocytic, Acute

Keywords

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Stem Cell Transplant

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Regimen A: TBI/Thiotepa/Cyclophosphamide

Patients in enrolled in Regimen A will receive the following:

* Total Body Irradiation (TBI), hyper-fractionated to a dose of 1320 cGy depending on age, stage of disease and requirement of general anesthesia with lung shielding
* Thiotepa, 5 mg/kg/day x 2 days via IV infusion over 4 hours daily or 10 mg/kg on one day
* Cyclophosphamide, 60 mg/kg/day x 2 days via IV infusion over 2 hours daily (or if cyclophosphamide is contraindicated, fludarabine at 25 mg/m\^2 x 5 days may be substituted)

Group Type EXPERIMENTAL

Total Body Irradiation (TBI)

Intervention Type RADIATION

Hyper-fractioned TBI is administered by a linear accelerator at a dose rate of \< 15 cGy/minute.

Thiotepa

Intervention Type DRUG

Thiotepa: 5 mg/kg/day IV over approximately 4 hours daily x 2 (Day -5 and Day -4).

Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide: 60 mg/kg/day x 2 or Fludarabine 25 mg/m\^2 x 5 if cyclophosphamide is contraindicated

Regimen B: Busulfan/Melphalan/Fludarabine

Patients in enrolled in Regimen B will receive the following:

* Busulfan, IV 0.8 mg/kg q6hours x 10 or 12 doses over 3 days, depending on the disease
* Melphalan, 70 mg/m\^2/day x 2 days via IV infusion over 30 minutes daily
* Fludarabine, 25 mg/m\^2/day x 5 days via IV infusion over 30 minutes daily

Group Type EXPERIMENTAL

Busulfan

Intervention Type DRUG

Busulfan: 0.8 mg/kg every 6 hours x 10 or 12 doses (depending on disease) with dose modified according to pharmacokinetics

Melphalan

Intervention Type DRUG

Melphalan: 70 mg/m\^2/day x 2

Fludarabine

Intervention Type DRUG

Fludarabine: 25 mg/m\^2/day x 5

Interventions

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Total Body Irradiation (TBI)

Hyper-fractioned TBI is administered by a linear accelerator at a dose rate of \< 15 cGy/minute.

Intervention Type RADIATION

Thiotepa

Thiotepa: 5 mg/kg/day IV over approximately 4 hours daily x 2 (Day -5 and Day -4).

Intervention Type DRUG

Cyclophosphamide

Cyclophosphamide: 60 mg/kg/day x 2 or Fludarabine 25 mg/m\^2 x 5 if cyclophosphamide is contraindicated

Intervention Type DRUG

Busulfan

Busulfan: 0.8 mg/kg every 6 hours x 10 or 12 doses (depending on disease) with dose modified according to pharmacokinetics

Intervention Type DRUG

Melphalan

Melphalan: 70 mg/m\^2/day x 2

Intervention Type DRUG

Fludarabine

Fludarabine: 25 mg/m\^2/day x 5

Intervention Type DRUG

Other Intervention Names

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Thioplex Cytoxan Busulfex Alkeran Fludara

Eligibility Criteria

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Inclusion Criteria

* 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:

1. AML in 1st remission - for patients who is AML does not have 'good risk' cytogenetic features (i.e. t8:21, t 15: 17, inv16).
2. Secondary AML in 1st remission
3. AML in 1st relapse or 2nd remission
4. ALL/CLL in patient remission clinical or molecular features indicating a high risk for relapse; or ALL/CLL 2nd remission
5. CML failing to respond to or not tolerating imatinib or dasatinib in first chronic phase of disease; CML in accelerated phase, second chronic phase, or in CR after accelerated phase or blast crisis.
6. Non-Hodgkin's lymphoma with chemo responsive disease in any of the following categories:

1. Intermediate or high-grade lymphomas who have failed to achieve a first CR or have relapsed following a 1st remission who are not candidates for autologous transplants.
2. Any NHL in remission which is considered not curable with chemotherapy alone and not eligible/appropriate for autologous transplant.
7. Chronic myelomonocyte leukemia: CMML-1 and CMML-2.


* Patient's age includes from ≥18 to ≤74 years old.
* Patients may be of either gender or any ethnic background.
* Patients must have a Karnofsky (adult) Performance Status of at least 70%
* Patients must have adequate organ function measured by:

Cardiac: asymptomatic or if symptomatic then LVEF at rest must be 50% and must improve with exercise.

Hepatic: \< 3x ULN AST and: s 1.5 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 is also eligible with Pl approval e.g. patients with PNH, Gilbert's disease or other hemolytic disorders.

Renal: serum creatinine: s; 1.2 mg/dL or if serum creatinine is outside the normal range, then CrCl \> 30 ml/min (measured or calculated/estimated).

Pulmonary: asymptomatic or if symptomatic, 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.

Exclusion Criteria

* Female patients who are pregnant or breast-feeding
* Active viral, bacterial or fungal infection
* Patient seropositive for HIV-I /II; HTLV -I /II
* Presence of leukemia in the CNS
Minimum Eligible Age

18 Years

Maximum Eligible Age

74 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Guenther Koehne

OTHER

Sponsor Role lead

Responsible Party

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Guenther Koehne

Deputy Director and Chief of Blood and Marrow Transplant, Hematologic Oncology and Benign Hematology

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Guenther Koehne, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Baptist Health South Florida/Miami Cancer Institute

Locations

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Baptist Health South Florida/Miami Cancer Institute

Miami, Florida, United States

Site Status

Countries

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United States

Central Contacts

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Guenther Koehne, MD, PhD

Role: CONTACT

Phone: 786-596-2000

Email: [email protected]

Claudia Torralbas, RN

Role: CONTACT

Phone: 786-596-2000

Email: [email protected]

Facility Contacts

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Guenther Koehne, MD, Ph.D.

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 15753458 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 11477433 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12091376 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17717135 (View on PubMed)

Related Links

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Other Identifiers

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2021-KOE-001

Identifier Type: -

Identifier Source: org_study_id