MedTrace Logo

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

NCT01034592 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2017-08-02

Study results available
· View outcomes & findings →

Summary

This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA).

Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence \[MDS International Working Group (IWG) 2000 Criteria will be applied\].

Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression

Conditions

  • Anemia
  • Leukemia
  • Acute Myeloid Leukemia (AML)
  • Myelodysplastic Syndromes (MDS)

Interventions

DRUG

Lenalidomide

2.5 mg/wk up to 5 mg 3x/wk

Sponsors & Collaborators

  • Celgene Corporation

    collaborator INDUSTRY

  • Jason Robert Gotlib

    lead OTHER

Principal Investigators

  • Jason Robert Gotlib · Stanford University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-11-30
Primary Completion
2012-10-31
Completion
2012-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01034592 on ClinicalTrials.gov