Safety and Efficacy Study in the Treatment of Pediatric Crohn's Disease

• Status: Terminated
• Trial end date: 2006-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to observe pediatric Crohn's Disease response to sargramostim in patients with or without steroid therapy at two possible dosage levels.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company
• Treatments: Molgramostim; Sargramostim

Criteria

Inclusion Criteria:

• Have a parent or guardian able to provide written informed consent

• Be able to provide verbal or written assent depending on age

• Age 6 to 16 years

• Have confirmed diagnosis of Crohn's Disease at study time of study entry based on radiologic, endoscopic, or histologic evaluations

• Have a PCDAI score >/= 30 points

• Have a negative serum pregnancy test within 7 days prior to receiving the first dose of sargramostim in female patients who, in the opinion of the investigator, are sexually active and of childbearing potential

• Be able to self-inject sargramostim or have a designee who can do so

• Available documentations of weight from 4 to 6 months prior to study entry

Exclusion Criteria:

• Existing colostomy or ileostomy

• Immediate need of GI surgery for active GI bleeding, peritonitis, intestinal obstruction, or intra-abdominal or perianal abscess requiring surgical drainage

• GI surgery within 3 month prior to receiving the first dose of sargramostim

• Symptoms of bowel obstruction or confirmed evidence of a clinically significant stricture within the last 6 month that has not been surgically corrected

• Use of any of the following medications within 4 weeks prior to receiving the first dose of study drug: 6 mercaptopurine, azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil, tacrolimus, cyclosporine, or thalidomide

• Use of licensed/registered and/or experimental anti-tumor necrosis factor (TNF) therapy such as infliximab or adalimumab within 8 weeks prior to first dose of sargramostim

• Use of any investigational drug within 4 weeks or 5 half-lives (whichever is greater) prior to receiving the first dose of sargramostim

• Concurrent use of corticosteroid therapy for Crohn's Disease, which exceeds a dose of 60 mg/day (or equivalent) of prednisone

• Inability to comply with protocol requirements or provide informed consent

• Presence of clinically important comorbid condition(s) unrelated to Crohn's Disease

• Prior use of recombinant human GM-CSF (sargramostim or molgramostim) or granulocyte colony-stimulating factor (G-CSF; filgrastim or pegfilgrastim)

• Current use of nutritional therapy (i.e., tube feeding or elemental/polymeric diet) which provides > 50% of daily caloric intake