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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Crohn Disease

Intervention / Treatment
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* Drug: Sargramostim (Leukine)
* Drug: Sargramostim (Leukine)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br> | Drug: Sargramostim (Leukine)<br>* 4 mcg sargramostim with and without corticosteroids<br>* Other names: BAY86-5326;|
| Experimental: Arm 2<br> | Drug: Sargramostim (Leukine)<br>* 6 mcg sargramostim with and without corticosteroids<br>* Other names: BAY86-5326;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Characterize the pharmacokinetic, pharmacodynamics properties of sargramostim treatment with concomitant corticosteroid induction therapy |  | Week 0, 1, and 2 | 
| Evaluate the efficacy of sargramostim treatment with concomitant corticosteroids as measured by the Pediatric Crohn's Disease Activity Index (PCDAI), Physician's Global Assessment (PGA), and IMPACT-III Questionnaire |  | PCDAI, PGA: Week -1 to EOS; IMPACT-II: Week 0, 8 and EOS | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Crohn Disease