Stem cell transplant study halted early: which prep works best?
NCT ID NCT01366612
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested two different drug combinations given before a stem cell transplant for people with myeloid blood cancers like leukemia. One group got fludarabine and busulfan, the other got those drugs plus low-dose total body irradiation. The goal was to see which regimen led to fewer cancer relapses within a year. The trial was stopped early after enrolling 53 adults, so the findings are limited.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Fludarabine and Busulfan, with or without low-dose total body irradiation
- What this could lead to
- If successful, this could help identify the best way to prepare patients for stem cell transplants, potentially reducing cancer relapse.
- What could go wrong
- The trial was terminated early with only 53 of 80 planned participants, so results are limited. Conditioning regimens carry serious risks like infection and organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
-
53 people
The number who actually took part.
- Started
-
Jun 2010
- Finished
-
Aug 2020
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 70 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Diagnosis of acute myelogenous leukemia, chronic myelogenous leukemia, other myeloproliferative disorder, or myelodysplastic syndrome * Any stage of disease will be considered for transplantation * Have a suitable related or unrelated donor (Section 3.3) * Age ≥18 but \<70 yrs * KPS of ≥70% * Recovery from all hematologic and non-hematology toxicities from previous therapies. Exclusion Criteria: * Diagnosis other than acute myelogenous leukemia, myeloproliferative disorder, or myelodysplastic syndrome * Chemotherapy or radiotherapy within 14 days of initiating treatment in this study with the exception of lenalidomide, decitabine, azacitidine, imatinib mesylate, dasatinib, nilotinib hydrochloride and hydroxyurea * Prior dose-intense therapy requiring HSC support within 56 days of initiating treatment in this study * Uncontrolled bacterial, viral, fungal or parasitic infections * Uncontrolled CNS metastases * Known amyloid deposition in heart * Organ dysfunction * LVEF \<40% or cardiac failure not responsive to therapy * FVC, FEV1, or DLCO \<50% of predicted and/or receiving supplementary continuous oxygen * Evidence of hepatic synthetic dysfunction, or total bilirubin \>2x or AST \>3x ULN * Measured creatinine clearance \<20 ml/min * Karnofsky score \<70% * Life expectancy limited by another co-morbid illness * Diagnosed or treated for another malignancy within 3 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy * Female subject is pregnant or breast-feeding (women) or unwilling to use acceptable birth control methods (men or women) for twelve months after treatment. Confirmation that the subject is not pregnant must be established by a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test result obtained during screening. Pregnancy testing is not required for post-menopausal or surgically sterilized women. * Documented hypersensitivity to fludarabine or melphalan or to bortezomib, boron or mannitol or any components of the formulation * Patients unable or unwilling to provide consent * Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure (see section 8.4), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening has to be documented by the investigator as not medically relevant * Patient has received other investigational drugs with 14 days before enrollment * Serious medical or psychiatric illness likely to interfere with participation in this clinical study Donor Inclusion and Exclusion Criteria Donor Inclusion Criteria: HLA 6/6 (HLA-A, B, DrB1) related donor or 7/8 (HLA-A, B, C, DrB1) unrelated donor Related donors will be evaluated in accordance with HUMC standard practice guidelines for the evaluation and management of allogeneic donors Unrelated donors will be identified, evaluated, and managed in accordance with National Marrow Donor Program standards Age ≥18 and \<70 yrs KPS of ≥70% Willing to donate bone marrow using standard techniques or peripheral blood HSC by leukapheresis Have adequate veins for apheresis or agree to placement of a central venous catheter (femoral, subclavian) if donating peripheral blood HSC Donor Exclusion Criteria Identical twin Female donors who are pregnant or breastfeeding Infection with HIV or viral hepatitis (B or C) Known allergy to filgrastim Current serious systemic illness Uncontrolled bacterial, viral, or fungal infection Receiving experimental therapy or investigational agents History of cancer other than treated basal cell cancer of the skin or carcinoma in situ of the cervix. Cancer treated with curative intent \>5 yrs before donation will be reviewed on a case-by-case basis by the principal investigator
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute myelogenous leukemia are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
John Theurer Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a p53-Targeting drug boost chemotherapy in Hard-to-Treat blood cancers?
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
- Can a calming drug make cord blood transplants safer?
- Can a Nine-Week group program help blood cancer patients grow through trauma?