Phase I, Dose Escalation Study of Decitabine

NCT02264873 | Completed Phase 1 DMC FDA Drug

• 2 other identifiers: IRB201400612PEDS004
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

Decitabine is a hypomethylating agent that has shown significant anti-leukemic effect in Myelodysplastic Syndrome (MDS) and Acute Myeloblastic Leukemia (AML). This study is based on the hypothesis that Decitabine delivered after allo-hematopoietic stem cell transplant (HSCT) in patients with leukemia will enhance disease control by the allogeneic immune system and lead to a longer disease free survival. The study is designed to provide safety data of low-dosing in the post-transplant setting.

Conditions

Leukemia, Lymphoblastic, Acute; Leukemia, Myeloid Acute; Hematopoetic Myelodysplasia

Keywords

Relapse; Autologous stem cell transplant

Outcome Measures

Primary Outcomes (1)

• Maximum Tolerated Dose as a Measure of Safety and Tolerability

  This is a dose escalation study of decitabine maintenance therapy after allo-HSCT for the maximally tolerated dose in pediatric patients without adverse events.

  3 months

Secondary Outcomes (2)

• Adverse Event Profile After Single Cycle Decitabine Post Transplant

  6 months

• Grade 3 Adverse Events after Decitabine

  6 months

Study Arms (1)

Decitabine

OTHER

This is a 3+3 design of dose escalation

Drug: Decitabine

Interventions

Decitabine DRUG

Dose escalation starting at 5 mg, and increasing by 2.5 mg to a Dose Level of 12.5 mg qd x 3 days.

Also known as: Decitabine, Dacogen

Decitabine

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age: greater than 1 and less than 31 years of age;
• Diagnosis: history of ALL, AML or MDS, currently in a complete remission (CR) following allo-HSCT (bone marrow leukemic blasts less than 5% by morphology), with high risk features including:
• Status post allogeneic HSCT
• GVHD prophylaxis:
• Karnofsky or Lansky performance scores more than 50%. Karnofsky scores will be used for patients \> 16 years of age and Lansky scores for patients ≤ 16 years of age;
• Platelet count ≥ 50,000 (untransfused);
• Absolute neutrophil count ≥ 1000; and;
• Hemoglobin ≥ 8 g/dL (un-transfused);

You may not qualify if:

• Progressive disease;
• Philadelphia chromosome positive ALL (these patients receive tyrosine kinase inhibitor posttransplant);
• Known hypersensitivity to any components of decitabine;
• Uncontrolled grade 3-4 graft versus host disease;
• Uncontrolled infection;
• Serum creatinine \> 2 mg/dL or glomerular filtration rate (GFR) less than 60 mL/min/1.73m2 ;
• Alanine Aminotransferase (ALT) greater than 3 times normal or serum total bilirubin greater than 2 mg/dL;

Sponsors & Collaborators

• University of Florida: lead
• Hyundai Hope On Wheels: collaborator

Study Sites (1)

UF Health Shands Hospital

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Recurrence

Interventions

Decitabine

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Paul Castillo, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2014
• First Posted: October 15, 2014
• Study Start: October 1, 2014
• Primary Completion: October 1, 2016
• Study Completion: September 18, 2018
• Last Updated: October 15, 2018

Record last verified: 2018-10

Locations

US (1)