Pentostatin for High Risk and Refractory Chronic Graft Versus Host Disease in Children
- Conditions
- Graft vs Host Disease
- Registration Number
- NCT00144430
- Brief Summary
This is a multicenter trial through the Pediatric Blood and Marrow Transplant Consortium.
The Primary hypothesis of this study is that because of its effect as a potent immunosuppressive agent targeting lymphocytes, pentostatin will show a sustained response in pediatric subjects with severe chronic GVHD. Secondary hypotheses include that the infection and toxicity rate of pentostatin in this setting will be acceptable given its lack of severe myelosuppression, and subjects with refractory chronic GVHD will have significant QOL impairment and symptomatology. These may change as subjects are being treated for their chronic GVHD with pentostatin.
- Detailed Description
To participate in this study, subjects must have diagnosed chronic Graft versus Host Disease that is refractory to therapy or that is considered high risk (i.e. low platelet count, progressive onset and greater than 50% of body surface area affected). Subjects must have not failed more than 2 immunosuppressive regimens in order to be considered for this trial. Eligible subjects will receive intravenous pentostatin every 2 weeks for 24 weeks. If the subject has had a complete response, the therapy will end at 24 weeks. If the subject has had a partial or mixed response or stable disease, they will continue on study receiving pentostatin for 52 weeks.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 60
Not provided
- More than two previous regimens to treat chronic GVHD. A regimen for the purpose of this study is defined as an adequate trial of an immunosuppressive or immunosuppressive combination (patient was given drug or drugs for 3 months unless there was clear progression during regimen).
- Calculated Cr. Clearance<30 ml/min/1.73m2
- Active infection not responding to oral or IV antibiotics.
- Subjects and families unwilling to firmly commit to return for follow-up doses and appointments.
- Use of any investigational agent in the past 30 days (this does not include the use of licensed agents for indications not listed in the package insert).
- Pregnancy or nursing mother; Females of child-bearing potential and sexually active males should use effective contraception while on study.
- Active veno-occlusive disease of the liver.
- Bronchiolitis obliterans (defined for the purpose of this study by respiratory symptoms such as shortness of breath or cough and FEV1<50%).
- Karnofsky performance score less than 40 (or Lansky performance less than 40 in subjects under 16 years of age).
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method To estimate the response rate (CR and PR) of pentostatin when used to treat refractory-chronic GVHD in children. To end of study
- Secondary Outcome Measures
Name Time Method To gather initial efficacy and safety data in high-risk untreated chronic GVHD in children. To end of study To evaluate toxicities of pentostatin when used to treat chronic GVHD in children. To end of study To evaluate quality of life (QOL) and symptoms at diagnosis and after therapy with pentostatin in pediatric patients with refractory chronic GVHD. To end of study
Trial Locations
- Locations (25)
Loma Linda University Medical Center
🇺🇸Loma Linda, California, United States
Columbus Children's Hospital
🇺🇸Columbus, Ohio, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Columbia Presbyterian Medical Center
🇺🇸New York, New York, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
St. Louis University
🇺🇸St. Louis, Missouri, United States
DeVos Children's Hospital
🇺🇸Grand Rapids, Michigan, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Southwest Texas Methodist Hospital
🇺🇸San Antonio, Texas, United States
Pediatric Medical Center for Northern California
🇺🇸Oakland, California, United States
University of Miami
🇺🇸Miami, Florida, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Nemour's Children's Clinic
🇺🇸Jacksonville, Florida, United States
John's Hopkins
🇺🇸Baltimore, Maryland, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Rainbow Babies and Children's
🇺🇸Cleveland, Ohio, United States
Victor Aquino, MD
🇺🇸Dallas, Texas, United States
All Children's Health System
🇺🇸St. Petersburg, Florida, United States
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
LSUHSC
🇺🇸New Orleans, Louisiana, United States
New York Medical College
🇺🇸Valhalla, New York, United States