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Can a gentler chemo prep make stem cell transplants safer for blood cancer patients?
NCT ID NCT01063647
First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time
Summary
This trial is testing whether a chemotherapy drug called treosulfan, given in different doses along with fludarabine, can safely prepare patients with advanced blood cancers for a stem cell transplant. The study focuses on patients who are not eligible for the standard, more intense conditioning regimens due to increased toxicity risk. The goal is to see if this approach is feasible and tolerable, and whether it can help keep the cancer in remission after the transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Treosulfan (a chemotherapy drug) combined with fludarabine, used as a conditioning regimen before stem cell transplantation
- What this could lead to
- If successful, this could provide a safer conditioning option for patients with blood cancers who cannot tolerate standard high-dose chemotherapy or radiation, potentially improving transplant outcomes.
- What could go wrong
- This is an early-phase trial with a small number of patients, so results may not be conclusive. The treatment carries risks of serious side effects, including transplant-related mortality and graft-versus-host disease.
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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56 people
The number who actually took part.
- Start date
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Nov 2001
- Finished
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Jun 2006
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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: 1. Patients with a haematological chemosensitive malignancy indicated for an allogeneic transplantation, but presenting an increased toxicity risk for classical (high-dose busulfan or standard-dose total body irradiation) conditioning therapies (remission criteria ref. to Appendix L): * CML in first or subsequent chronic phase * NHL in 2nd CR/PR, chemosensitive PR after autologous transplantation ; CLL in 2nd or subsequent CR/PR * Relapsed Morbus Hodgkin (MH) after autologous transplantation * Multiple Myeloma (MM) stage II and III according to Durie and Salmon * AML in 2nd CR/PR or high-risk AML in 1st CR/PR High-risk defined for example by the following: * Cytogenetics: -5/5q, -7/7q, del(5q), abnormalities of 3q, complex karyotype (\> 3 abnormalities), or * PR after 1 cycle of induction therapy * ALL in 2nd CR/PR or high-risk ALL in 1st CR/PR High-risk defined as follows: * Leukocytes \> 3000/µl (B-Linage) or \> 100000/µl (T-Linage); * Pro-B-ALL, pre-T-ALL * Cytogenetics: t(9;22)/BCR-ABL; t(4;11)/ALL1-AF * MDS (patients without prior chemotherapy may be included) 2. Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) or one mismatch (out of the 6 standard markers) sibling donor (1 misMRD): • HLA-identity defined by the following markers: A, B, DRB1. DQB1 must be recorded. 3. Age \> 18 years 4. Karnofsky Index \> 80 % 5. Adequate contraception in female patients of child-bearing potential 6. Co-operative behavior of individual patients 7. Written informed consent Exclusion Criteria: 1. Completely chemotherapy-resistant disease 2. Severe cardiac insufficiency, severe cardio-vascular or other severe concomitant diseases 3. Symptomatic malignant involvement of the CNS 4. Active infectious disease 5. HIV-positive or active hepatitis infection 6. Impaired liver function (Bilirubin \> 1.5 x upper normal limit; Transaminases \> 3.0 x upper normal limit) 7. Impaired renal function (Creatinine-clearance \< 60 ml/min; Serum Creatinine \> 1.5 x upper normal limit). 8. Pleural effusion or ascites \> 1.0 L 9. Pregnancy or lactation 10. Known hypersensitivity to fludarabine and/or treosulfan 11. Parallel participation in another experimental drug trial
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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
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5th Medical Clinic, Clinic North
Nuremberg, 90340, Germany
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Charité University Hospital Berlin
Berlin, 12203, Germany
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Helsinki University Central Hospital
Helsinki, FIN-00290, Finland
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Karolinska University Hospital & Karolinska Institute
Stockholm, 141 86, Sweden
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Silesian Medical University
Katowice, 40-029, Poland
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University Hospital Hamburg Eppendorf
Hamburg, 20246, Germany
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University Hospital Rostock
Rostock, 18057, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a stronger drug cocktail give older myeloma patients a better start?
- Can a p53-Targeting drug boost chemotherapy in Hard-to-Treat blood cancers?
- Cheap blood count ratios eyed as window into Myeloma's inflammatory grip
- Can a t-cell engager rescue myeloma that outsmarted CAR-T?
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?