Clinical trial for AML
Official title Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
ClinicalTrials.gov ID: NCT04644016
What this study is testing
What is Clofarabine?
Clofarabine is an investigational injectable medicine being studied as a potential treatment for acute myeloid leukemia (AML). Clofarabine is designed to interfere with DNA synthesis by inhibiting DNA polymerase and ribonucleotide reductase, leading to cancer cell death.
Also referred to as Clolar.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study is looking at how many patients die from the transplant treatment within one year.
- Who it's for
- Children and young adults with serious blood cancers or non‑malignant blood disorders who do not have a matched related donor.
- What's involved
- Participants receive a cord blood stem cell transplant and have regular medical visits and tests to check safety and outcomes.
- Time commitment
- The study follows participants for about one year, with scheduled visits and assessments during that time.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 21
You may be able to join if
- Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).
- Therapy-related AML (t-AML).
- White cell count at presentation \> 100,000.
- Presence of extramedullary leukemia at diagnosis.
- Any unfavorable subtype by FAB or WHO classification.
You likely can't join if
- Inadequate performance status/ organ function.
- Advanced metabolic disease (EBMT handbook).
- Active CNS leukemic involvement.
- Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.
- Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.
- Autologous stem cell transplant within the preceding 6 months.
See the full eligibility criteria
- Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).
- Therapy-related AML (t-AML).
- White cell count at presentation \> 100,000.
- Presence of extramedullary leukemia at diagnosis.
- Any unfavorable subtype by FAB or WHO classification.
- High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.
- Requirement for 2 or more inductions to achieve CR1.
- Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.
- Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.
- Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.
- Other high-risk features not defined above.
- Complete second remission (CR2).
- White cell count at presentation \> 30,000 for B-cell lineage and \> 100,000 for T-cell lineage.
- Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.
- Failure to achieve complete remission (CR) after four weeks of induction therapy.
- Persistence or recurrence of MRD on therapy.
- Any patient unable to tolerate consolidation and/or maintenance chemotherapy as would have been deemed appropriate by the treating physician.
- Other high-risk features not defined above.
- Complete second remission (CR2).
- International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.
- Any IPSS risk category if life-threatening cytopenia(s) exists.
- Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.
- MDS/ myeloproliferative disorder overlap syndromes without myelofibrosis.
- Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.
- Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease/ CR/ PR with no single lesion equal to or...
- Hurler Syndrome
- Hunter (MPS 2 - early disease)
- Sly syndrome (MPSVIII)
- α-Mannosidosis
- X- ALD
- Osteopetrosis
- Metachromatic Leukodystrophy
- Globoid (GLD) VII. Non-Malignant disorders (other) [also see EBMT Handbook for criteria for transplant (46)]
- Hemoglobinopathies
- Bone Marrow Failure syndromes
- Karnofsky or Lansky score ≥ 70% (see Appendix)
- Bilirubin ≤ 1.5 mg/dL (unless benign congenital hyperbilirubinemia).
- ALT ≤ 3 x upper limit of normal.
- Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .
- Left ventricular ejection fraction ≥ 50%.
- Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.
- Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \> 50 mL/min/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2) \>30% of predicted normal for age.
- Each CB unit must be at least 3/8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.
- For malignant diseases follow MSKCC CBU selection algorithm
- For non-malignant diseases, CBU will be required to have \> 5 x 107 TNC/kg; high HLA allele level match is preferable Participant
- Inadequate performance status/ organ function.
- Advanced metabolic disease (EBMT handbook).
- Active CNS leukemic involvement.
- Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.
- Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.
- Autologous stem cell transplant within the preceding 6 months.
- Any prior allogeneic stem cell transplant.
- Active and uncontrolled infection (bacterial/fungal/viral) at time of transplantation.
- HIV infection.
- Seropositivity for HTLV-1.
- Pregnancy or breast feeding.
- Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.
The study team makes the final eligibility decision.
Where it is enrolling
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at New York, New York, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.