Clinical trial NCT00280631: Study of TLK199 Tablets in Myelodysplastic Syndrome (MDS)
Official title: Phase 1-2a Dose-Ranging Study of TLK199 Tablets in Myelodysplastic Syndrome (MDS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Myelodysplastic Syndrome (MDS)
Lead sponsor: Telik
Sponsor class: INDUSTRY
First posted: 2006-01-23
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety and efficacy of TLK199 Tablets in patients with Myelodysplastic Syndrome (MDS)

Detailed description: This Phase 1-2a study is an open label, dose-ranging study of TLK199 Tablets in patients with all World Health Organization or French-American-British classification types of myelodysplastic syndrome (MDS). In Phase 1, the safety, pharmacokinetics, and hematologic response rate with TLK199 Tablets will be evaluated. The Phase 1 dose-ranging stage is designed to determine the maximum tolerated dose or optimal biologic dose of TLK199 Tablets. Evaluation of the pharmacokinetics of TLK199 Tablets will be conducted. In Phase 1, an additional 12 patients will undergo pharmacokinetic evaluation under fed conditions at the selected Phase 2a dose. In Phase 2a, further safety and hematologic responses by the modified International Working Group MDS response criteria will be studied.

Eligibility criteria: Inclusion Criteria:

* Histologically confirmed diagnosis of MDS
* Documented significant cytopenia for at least 2 months
* Adequate liver and kidney function
* Ineligible for stem cell bone marrow transplantation
* At least 18 years of age
* Discontinuation of growth factors (e.g., G-CSF) within 3 weeks of study entry

Exclusion Criteria:

* Prior bone marrow transplant
* Failure to recover from any prior surgery or any major surgery within 4 weeks of study entry
* Pregnant or lactating women
* Other investigational drugs within 14 days of study entry
* Chemotherapy, radiotherapy or immunotherapy within 14 days of study entry

Primary outcomes:
measure: Maximum Tolerated Dose or Optimal Biologic Dose
timeFrame: 6 months
Secondary outcomes:
measure: Safety Pharmacokinetic Parameters, Hematologic Response Parameters
timeFrame: 6 months
Other outcomes:
Locations:
city: Maywood
country: United States
facility: Loyola University Chicago-Cardinal Bernardin Cancer Center
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Worcester
country: United States
facility: University of Massachusetts (UMass) Memorial Medical Center
state: Massachusetts
Study officials:
affiliation: Telik
name: Gail Brown, MD
role: STUDY_DIRECTOR
References:
citation: Raza A, Galili N, Smith S, Godwin J, Lancet J, Melchert M, Jones M, Keck JG, Meng L, Brown GL, List A. Phase 1 multicenter dose-escalation study of ezatiostat hydrochloride (TLK199 tablets), a novel glutathione analog prodrug, in patients with myelodysplastic syndrome. Blood. 2009 Jun 25;113(26):6533-40. doi: 10.1182/blood-2009-01-176032. Epub 2009 Apr 27.