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

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00206661
First received: September 12, 2005
Last updated: December 2, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Crohn Disease
Drug: Sargramostim (Leukine)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Characterize the pharmacokinetic, pharmacodynamics properties of sargramostim treatment with concomitant corticosteroid induction therapy [ Time Frame: Week 0, 1, and 2 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 22
Study Start Date: December 2004
Study Completion Date: December 2006
Primary Completion Date: December 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1 Drug: Sargramostim (Leukine)
4 mcg sargramostim with and without corticosteroids
Other Name: BAY86-5326
Experimental: Arm 2 Drug: Sargramostim (Leukine)
6 mcg sargramostim with and without corticosteroids
Other Name: BAY86-5326

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

Ages Eligible for Study:   6 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00206661

Locations
United States, California
San Francisco, California, United States, 94117
United States, Georgia
Atlanta, Georgia, United States, 30342
United States, Louisiana
New Orleans, Louisiana, United States, 70118-5799
United States, Massachusetts
Boston, Massachusetts, United States, 02114-2696
United States, New Jersey
Morristown, New Jersey, United States, 07962-1956
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19104-4399
United States, Texas
Houston, Texas, United States, 77030
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00206661     History of Changes
Other Study ID Numbers: 308001, 91409, Novel 6
Study First Received: September 12, 2005
Last Updated: December 2, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Sanofi:
Crohn Disease

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases

ClinicalTrials.gov processed this record on August 21, 2014