Clinical trial NCT00003894: Thalidomide in Treating Patients With Chronic Graft-Versus-Host Disease Following Bone Marrow Transplant
Official title: Thalidomide vs. Placebo for Steroid Dependent or Refractory Chronic Graft vs. Host Disease (cGVHD) IND #42782
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Graft Versus Host Disease
Unspecified Adult Solid Tumor, Protocol Specific
Unspecified Childhood Solid Tumor, Protocol Specific
Keywords: unspecified childhood solid tumor, protocol specific
unspecified adult solid tumor, protocol specific
graft versus host disease
Lead sponsor: Roswell Park Cancer Institute
Sponsor class: OTHER
First posted: 2004-06-17
Enrollment: 36
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: RATIONALE: Thalidomide may interfere with the body's ability to recognize transplanted bone marrow cells as foreign and may help treat patients with graft-versus-host disease.

PURPOSE: Randomized phase II trial to study the effectiveness of thalidomide in treating patients who have chronic graft-versus-host disease following bone marrow transplantation.

Detailed description: OBJECTIVES: I. Determine the efficacy of thalidomide in patients with steroid dependent or refractory chronic graft vs host disease following an allogeneic bone marrow transplant. II. Determine the toxicity of this regimen in these patients.

OUTLINE: This is a randomized study. Patients are stratified according to the type of bone marrow transplant received (allogeneic vs matched unrelated donor) and type of chronic graft vs host disease (steroid dependent vs steroid refractory). Patients receive either oral thalidomide or placebo 2-4 times a day for 6 months. The drug is then tapered until stopped. If disease recurs, the drug may be restarted for a second 6 month course. The maximum duration of treatment is 18 months. Patients continue on steroid therapy, which is tapered during study therapy. If no response is seen after 8 weeks, patients may cross over to the other alternate drug. Patients are followed weekly for 1 month, then every month for 1 year.

PROJECTED ACCRUAL: A total of 36 patients (18 per arm) will be accrued for this study.

Eligibility criteria: DISEASE CHARACTERISTICS: Histologically confirmed chronic graft vs host disease following an allogeneic bone marrow transplant (BMT) Greater than 100 days since BMT Must be receiving at least 1 mg/kg of prednisone or equivalent immunosuppression

PATIENT CHARACTERISTICS: Age: 3 and over Performance status: Not specified Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Not specified Renal: Not specified Other: Not pregnant Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY: Biologic therapy: See Disease Characteristics Chemotherapy: Not specified Endocrine therapy: See Disease Characteristics Radiotherapy: Not specified Surgery: Not specified

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: New Orleans
country: United States
facility: Louisiana State University School of Medicine
state: Louisiana
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: Dallas
country: United States
facility: Simmons Cancer Center - Dallas
state: Texas
city: Fort Sam Houston
country: United States
facility: Brooke Army Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center - Fort Worth
state: Texas
city: Houston
country: United States
facility: University of Texas - MD Anderson Cancer Center
state: Texas
city: Milwaukee
country: United States
facility: Cancer Center of the Medical College of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Roswell Park Cancer Institute
name: Barbara Jean Bambach, MD
role: STUDY_CHAIR
References: