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

Overview

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.

Full Title of Study: “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

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2006

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.

Interventions

  • Drug: Sargramostim (Leukine)
    • 4 mcg sargramostim with and without corticosteroids
  • Drug: Sargramostim (Leukine)
    • 6 mcg sargramostim with and without corticosteroids

Arms, Groups and Cohorts

  • Experimental: Arm 1
  • Experimental: Arm 2

Clinical Trial Outcome Measures

Primary Measures

  • Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy
    • Time Frame: 12 weeks

Secondary Measures

  • Characterize the pharmacokinetic, pharmacodynamics properties of sargramostim treatment with concomitant corticosteroid induction therapy
    • Time Frame: 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
    • Time Frame: PCDAI, PGA: Week -1 to EOS; IMPACT-II: Week 0, 8 and EOS

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 6 Years

Maximum Age: 16 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Genzyme, a Sanofi Company
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Medical Monitor, Study Director, Genzyme, a Sanofi Company

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