Clinical trial NCT00206661: Safety and Efficacy Study in the Treatment of Pediatric Crohn's Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Crohn Disease
Keywords: Crohn Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-09-21
Enrollment: 22
Sex: ALL
Minimum age (years): 6
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Detailed description: 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.

Eligibility 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

Primary outcomes:
measure: Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy
timeFrame: 12 weeks
Secondary outcomes:
measure: Characterize the pharmacokinetic, pharmacodynamics properties of sargramostim treatment with concomitant corticosteroid induction therapy
timeFrame: Week 0, 1, and 2
measure: 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
timeFrame: PCDAI, PGA: Week -1 to EOS; IMPACT-II: Week 0, 8 and EOS
Other outcomes:
Locations:
city: San Francisco
country: United States
state: California
city: Atlanta
country: United States
state: Georgia
city: New Orleans
country: United States
state: Louisiana
city: Boston
country: United States
state: Massachusetts
city: Morristown
country: United States
state: New Jersey
city: Philadelphia
country: United States
state: Pennsylvania
city: Houston
country: United States
state: Texas
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: