Phase 2 Study of Telintra® in Deletion 5q Myelodysplastic Syndrome

Phase 2TerminatedNCT01422486

Telik2 enrolled

Overview

Study TLK199.2107 is a multicenter, single arm, open-label Phase 2 study of oral ezatiostat (Telintra®) in patients with lenalidomide (Revlimid®) refractory or resistant, red blood cell (RBC) transfusion-dependent, Low to Intermediate-1 IPSS risk, del5q Myelodysplastic Syndrome (MDS). Independence from red blood cell transfusions, improvement in the levels of red blood cells, white blood cells, and platelets, and the response of the bone marrow were evaluated. Patients received a starting dose of 2000 mg total daily dose in divided doses (1000 mg orally twice daily for three weeks (21 days) on therapy followed by a one-week (7 days) off therapy rest period in four-week (28 days) treatment cycles. Patients continued treatment until documentation of lack of MDS response, MDS progression, unacceptable toxicity, or patient withdrawal from the study.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Syndrome (MDS)

Interventions

ezatiostat hydrochloride (Telintra®)DRUG

Three weeks of treatment with ezatiostat at 2000 mg per day in divided doses followed by a one week rest period in four-week treatment cycles.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Primary or de Novo MDS * Low or Intermediate-1 IPSS risk MDS * Deletion of the 5q chromosome \[del(5q) MDS\] * Refractory or resistant to lenalidomide (Revlimid) * ECOG performance score of 0 or 1 * Documentation of significant anemia with or without additional cytopenia * Adequate kidney and liver function * Patients must have discontinued hematopoietic growth factors at least 3 weeks prior to study entry Exclusion Criteria: * Prior allogenic bone marrow transplant for MDS * Known sensitivity to ezatiostat (injection or oral tablets) * Prior treatment with hypomethylating agent (HMA) (e.g., azacitadine, decitabine) * History of MDS IPSS risk score of greater than 1.0 * Pregnant or lactating women * Any severe concurrent disease, infection or comorbidity that, in the judgement of the investigator, would make the patient inappropriate for study entry * Oral steroids greater than 10 mg per day. Exceptions: those prescribed for other conditions (such as new adrenal failure, asthma, arthritis) or brief steroid use (such as tapered dosing for an acute non-MDS condition) * History of hepatitis B or C, or HIV

Locations (5)

Loyola University

Maywood, Illinois, United States

SIU School of Medicine, Simmons Cancer Center

Springfield, Illinois, United States

Center for Cancer and Blood Disorders

Bethesda, Maryland, United States

Columbia University

New York, New York, United States

Outcomes

Primary Outcomes

Hematologic Improvement-Erythroid (HI-E) rate

Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)

Time frame: At 8, 16, 24, and 32 weeks of treatment

Secondary Outcomes

RBC Transfusion independence (TI) rate

Time frame: At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment

Hematologic Improvement-Neutrophil (HI-N) rate

Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)

Time frame: At 8, 16, 24, & 32 weeks of treatment

Hematologic Improvement-Platelet (HI-P) rate

Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)

Time frame: At 8, 16, 24, & 32 weeks of treatment

Unilineage, bilineage, trilineage, and overall HI response rate

Time frame: 2 years

Cytogenetic response rate

Time frame: 16 weeks, 48 weeks and at the time of first HI response

Duration of response

Time frame: 2 years

Safety of ezatiostat in this MDS population

Recording and grading of AEs using NCI-CTCAE v4.03

Time frame: At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment

Evaluation of the relationship between HI-E response, gene expression profiling and response-related variables

Data from ClinicalTrials.gov

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