Clinical trial · Interventional
Combination Chemotherapy Followed By Donor Bone Marrow or Umbilical Cord Blood Transplant in Treating Children With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Phase II Window Evaluation of the Farnesyl Transferase Inhibitor (R115777) Followed by 13-CIS Retinoic Acid, Cytosine Arabinoside and Fludarabine Plus Hematopoietic Stem Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Giving chemotherapy drugs, such as R115777, isotretinoin, cytarabine, and fludarabine, before a donor bone marrow transplant or an umbilical cord transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. This phase II trial is studying how well giving combination chemotherapy together with donor bone marrow or umbilical cord blood transplant works in treating children with newly diagnosed juvenile myelomonocytic leukemia
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (11)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| anti-thymocyte globulin | Biological | — | UNRESOLVED |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| cytarabine | Drug | Cytarabine | ALIAS |
| double-unit umbilical cord blood transplantation | Procedure | — | UNRESOLVED |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| isotretinoin | Drug | — | UNRESOLVED |
| laboratory biomarker analysis | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment (tipifarnib, bone marrow/umbilical cord transplant)
- description
- See detailed description.
- interventionNames
- Drug: tipifarnib
- Drug: isotretinoin
- Drug: fludarabine phosphate
- Drug: cytarabine
- Radiation: radiation therapy
- Drug: cyclophosphamide
- Biological: anti-thymocyte globulin
- Procedure: allogeneic bone marrow transplantation
- Procedure: double-unit umbilical cord blood transplantation
- Procedure: umbilical cord blood transplantation
- Other: laboratory biomarker analysis
Primary outcomes (5)
- measure
- Response rate (CR or PR)
- timeFrame
- Up to 6 years
- description
- The response rates in the up-front window with respect to whether or not patients had vas activating mutations will also be estimated by proportions.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Newly diagnosed, previously untreated juvenile myelomonocytic leukemia * Presenting with all of the following: * Absence of t(9;22) or bcr/abl by PCR * Absolute monocyte count greater than 1,000/mm\^3 * Less than 20% bone marrow blasts * Presenting with at least 2 of the following: * Elevated F hemoglobin * Myeloid precursors in peripheral blood * WBC greater than 10,000/mm\^3 * Sargramostim (GM-CSF) hypersensitivity * See Disease Characteristics * Bilirubin no greater than 2.0 mg/dL * ALT no greater than 3 times normal * Creatinine no greater than 2 times normal * No concurrent sargramostim (GM-CSF) * No concurrent proton pump inhibitors
References
Publications (1)
- DERIVEDStieglitz E, Gu CJ, Richardson M, Kita R, Santaguida MT, Ali KA, Strachan DC, Dhar A, Yam G, Anderson W, Anderson E, Hubner J, Tasian SK, Loh ML, Lacher MD. Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. JCO Precis Oncol. 2023 Sep;7:e2300302. doi: 10.1200/PO.23.00302. PMID 37944074