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

Phase 1/2 Safety, Pharmacokinetic, and Pharmacodynamic Study of Sargramostim (Leukine®) in Pediatric Patients With Active Crohn's Disease, With One-year Surveillance and Retreatment Extension

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.

On 29 May 2009, Bayer began transitioning the sponsorship of this trial to Genzyme. NOTE: This study was originally posted by sponsor Berlex, Inc. Berlex, Inc. was renamed to Bayer HealthCare, Inc.

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