Crohn DiseasePhase 1/2NCT00206661
Safety and Efficacy Study in the Treatment of Pediatric Crohn's Disease
A Phase 1/2 study testing Sargramostim (Leukine) in people with Crohn Disease, run by Genzyme, a Sanofi Company at 7 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Crohn Disease
- What is being tested?
- Sargramostim (Leukine)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 7 sitesUnited States
- Study dates
- Dec 2004to Dec 2006
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Study site
Atlanta, Georgia, 30342
Study site
Boston, Massachusetts, 02114-2696
Study site
Houston, Texas, 77030
Study site
Morristown, New Jersey, 07962-1956
Study site
New Orleans, Louisiana, 70118-5799
Study site
Philadelphia, Pennsylvania, 19104-4399
Study site
San Francisco, California, 94117
About this study
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
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.
Read the 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.
- Conditions
- Crohn Disease
- Being tested
- Sargramostim (Leukine)
- Keywords
- Crohn Disease
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sargramostim (Leukine)Drug
Also called BAY86-5326
4 mcg sargramostim with and without corticosteroids
Sargramostim (Leukine)Drug
Also called BAY86-5326
6 mcg sargramostim with and without corticosteroids
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Not described Receives: Drug: Sargramostim (Leukine) |
| Arm 2 | Experimental | Not described Receives: Drug: Sargramostim (Leukine) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy
Measured: 12 weeks
Secondary outcomes (2)
Characterize the pharmacokinetic, pharmacodynamics properties of sargramostim treatment with concomitant corticosteroid induction therapy
Measured: 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
Measured: PCDAI, PGA: Week -1 to EOS; IMPACT-II: Week 0, 8 and EOS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2005 (estimated)
- Study startDec 2004
- Primary completionDec 2006
- Study completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crohn DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00206661
- Sponsor's protocol ID
- 308001
- Other IDs
- 91409, Novel 6
- Status last verified
- Dec 2013
- First submitted
- 12 Sep 2005
- Last updated
- 4 Dec 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00206661
- Acronym / sponsor ID
- 308001
- Conditions
- Crohn Disease
- Keywords
- Crohn Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Dec 2004
- Primary completion
- Dec 2006 (actual)
- Study completion
- Dec 2006 (actual)
- First posted
- 21 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Sargramostim (Leukine) |
| Arm 2 | Experimental | Drug: Sargramostim (Leukine) |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sargramostim (Leukine)Also: BAY86-53264 mcg sargramostim with and without corticosteroids | Drug | Arm 1 |
| Sargramostim (Leukine)Also: BAY86-53266 mcg sargramostim with and without corticosteroids | Drug | Arm 2 |
Primary outcome
Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy
Time frame: 12 weeks
2 secondary outcomes
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
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2013.
Related trials for Crohn Disease
Not yet recruitingPhase 1
An Open-label, DDI Study to Investigate the Effects of Duvakitug on the PK of Selected Cytochrome P450 Substrates
Ulcerative Colitis, Crohn's Disease
RecruitingObservational study
Longitudinal Immunophenotyping of Patients With Inflammatory Bowel Disease
Inflammatory Bowel Disease, Crohn's Disease
RecruitingObservational study
Ibd CAncer and seRious Infections in France (I-CARE 2)
IBD, Ulcerative Colitis
RecruitingObservational study
Characterization of Colorectal Dysplasic Lesions and Stratification of Their Evolving Risk in Inflammatory Bowel Disease Patients: a Prospective, Observational and Multicenter Study
Crohn Disease (CD), Colitis Ulcerative
Enrolling by invitationPhase 4
MOdel-Informed Precision Dosing of Ustekinumab and VEdolizumab in Inflammatory Bowel Disease
Inflammatory Bowel Diseases, Crohn Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sargramostim (Leukine) in people with Crohn Disease, run by Genzyme, a Sanofi Company at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 4 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize the safety profile of sargramostim treatment with concomitant corticosteroid induction therapy (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2013.