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
3 other identifiers
interventional
22
1 country
7
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Dec 2004
Typical duration for phase_1
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2004
CompletedFirst Submitted
Initial submission to the registry
September 12, 2005
CompletedFirst Posted
Study publicly available on registry
September 21, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2006
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2006
CompletedDecember 4, 2013
December 1, 2013
2 years
September 12, 2005
December 2, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy
12 weeks
Secondary Outcomes (2)
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
Study Arms (2)
Arm 1
EXPERIMENTALArm 2
EXPERIMENTALInterventions
4 mcg sargramostim with and without corticosteroids
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (7)
Unknown Facility
San Francisco, California, 94117, United States
Unknown Facility
Atlanta, Georgia, 30342, United States
Unknown Facility
New Orleans, Louisiana, 70118-5799, United States
Unknown Facility
Boston, Massachusetts, 02114-2696, United States
Unknown Facility
Morristown, New Jersey, 07962-1956, United States
Unknown Facility
Philadelphia, Pennsylvania, 19104-4399, United States
Unknown Facility
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Monitor
Genzyme, a Sanofi Company
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 12, 2005
First Posted
September 21, 2005
Study Start
December 1, 2004
Primary Completion
December 1, 2006
Study Completion
December 1, 2006
Last Updated
December 4, 2013
Record last verified: 2013-12