Clinical trial · Interventional
Phenylbutyrate and Tretinoin in Treating Patients With Hematologic Cancer
A Phase I, Dose-Finding Trial of Sodium Phenylbutrate (NSC 657802) in Combination With All Trans-retinoic Acid (ATRA, NSC 122758) in Patients With Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
- 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)
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I trial to study the effectiveness of combining phenylbutyrate and tretinoin in treating patients who have hematologic cancer.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic/Myeloproliferative Diseases | Myelodysplastic/Myeloproliferative Neoplasm | ALIAS | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| sodium phenylbutyrate | Drug | — | UNRESOLVED |
| tretinoin | Drug | Tretinoin | ALIAS |
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 120 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Histologically or cytologically confirmed myelodysplastic syndrome (MDS)
* Refractory anemia
* Primary refractory leukopenia or thrombocytopenia with morphologic features of MDS
* Refractory anemia with excess blasts (RAEB)
* Refractory anemia with ringed sideroblasts
* RAEB in transformation
* Must have excess blasts or be hematopoietically compromised, defined as one of the following:
* RBC transfusion dependent
* Granulocyte count less than 1,000/mm\^3
* Platelet count less than 50,000/mm\^3 OR
* Diagnosis of chronic myelomonocytic leukemia
* Hematopoietically compromised (as defined above) OR
* Excess blasts OR
* Evaluable disease related symptomatology (organomegaly or leukemia cutis) OR
* Diagnosis of acute myeloid leukemia
* WBC less than 20,000/mm\^3 and stable for at least 2 weeks
* Unlikely to require cytotoxic therapy during study
* No CNS or pulmonary leukostasis or CNS leukemia
PATIENT CHARACTERISTICS:
Age:
* 18 and over
Performance status:
* Zubrod 0-2
Life expectancy:
* Not specified
Hematopoietic:
* See Disease Characteristics
* Hemoglobin at least 8 g/dL (transfusion allowed)
* No disseminated intravascular coagulation
Hepatic:
* Bilirubin less than 2.0 mg/dL (unless due to hemolysis or Gilbert's syndrome)
Renal:
* Creatinine less than 2.0 mg/dL
Other:
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception for 2 weeks prior, during, and for 3 months after study
* No active infection
PRIOR CONCURRENT THERAPY:
Biologic therapy:
* See Disease Characteristics
* At least 3 weeks since prior biologic therapy, including hematopoietic growth factors, and recovered
Chemotherapy:
* See Disease Characteristics
* At least 3 weeks (1 month for MDS patients) since prior chemotherapy and recovered
Endocrine therapy:
* Not specified
Radiotherapy:
* At least 3 weeks since prior radiotherapy and recovered
Surgery:
* Not specifiedReferences
Publications (0)
Data not yet available