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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00295165




Registration number
NCT00295165
Ethics application status
Date submitted
21/02/2006
Date registered
23/02/2006
Date last updated
4/12/2013

Titles & IDs
Public title
Efficacy (Induction of Response/Remission) and Safety Study in Patients With Moderate to Severe Crohn's Disease
Scientific title
Randomized, Double-Blind, Placebo-Controlled, Phase 3 Induction Study to Assess the Efficacy and Safety of 6µg Sargramostim (Leukine) Administered Subcutaneously Once Daily for 8 Weeks in Patients With Active Crohn's Disease
Secondary ID [1] 0 0
91494
Secondary ID [2] 0 0
310187
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Crohn Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Crohn's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sargramostim (Leukine)
Treatment: Drugs - Placebo

Experimental: Arm 1 -

Placebo Comparator: Arm 2 -


Treatment: Drugs: Sargramostim (Leukine)
Sargramostim 6 mcg/kg subcutaneously once daily

Treatment: Drugs: Placebo
Placebo subcutaneously once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To induce clinical remission and/or clinical response following 8 weeks of treatment
Timepoint [1] 0 0
8 weeks
Secondary outcome [1] 0 0
To assess the safety profile of sargramostim (including development of antibodies against sargramostim)
Timepoint [1] 0 0
During study treatment
Secondary outcome [2] 0 0
To assess quality of life (QoL)
Timepoint [2] 0 0
During study treatment

Eligibility
Key inclusion criteria
1. Written informed consent

2. Male or female, age >/= 18 years

3. Confirmed diagnosis of Crohn's disease (endoscopic or radiological evaluation) at
least 4 months prior to receiving the first dose of study drug

4. Moderately to severely active Crohn's disease at time of screening (i.e., CDAI greater
than or equal to 220 and less than or equal to 475 points)

5. If under treatment for Crohn's disease, medication must be stable for at least 4 weeks
prior to receiving the first dose of study drug. The following therapies are allowed:

- Oral therapy with salicylates (mesalamine, sulfasalazine, olsalazine, or
balsalazide) for Crohn's disease

- Antibiotics or probiotics for Crohn's disease

- Topical rectal therapy with mesalamine

6. Females of child-bearing potential:

Negative pregnancy test within 72 hours prior to receiving the first dose of study
drug

7. Sexually-active males and females of child-bearing potential:

Agreement to use adequate method of contraception throughout the study

8. Ability to self-inject study drug or availability of a designee who can do so
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Pregnancy or breast-feeding

2. Colostomy or ileostomy

3. Immediate need for gastrointestinal (GI) surgery for active GI bleeding, peritonitis,
intestinal obstruction, or intra-abdominal or perianal abscess requiring surgical
drainage

4. GI surgery within 6 months prior to receiving the first dose of study drug

5. Symptoms of bowel obstruction or confirmed evidence of a clinically-significant
stricture within the last 6 months that has not been surgically corrected

6. Positive stool test results for any of the following:

Bacteria:

- Salmonella spec.

- Shigella spec.

- Campylobacter spec.

Bacterial toxin:

- Clostridium difficile

Ova and parasites:

- Amoeba spec.

- Giardia spec.

- Cryptosporidium spec.

7. Any of the following laboratory abnormalities:

- Serum creatinine >/= 2.0 mg/dL

- Alkaline phosphatase (AP), aspartate aminotransferase (AST), alanine
aminotransferase (ALT), or total bilirubin >/=; 2 x the upper limit of normal

- Hemoglobin (Hgb) < 8.0 g/dL

- Absolute neutrophil count (ANC) </= 1,000 cells/µL or > cells 20,000/µL

8. Planned in-patient hospitalization during the study

9. Presence or history of cancer of any type (except treated basal cell carcinoma) or
definite dysplasia of the colon within the last 5 years

10. Use of any of the following medications during the specified period of time prior to
receiving the first dose of study drug:

At any time:

- Recombinant human GM CSF (sargramostim or molgramostim)

- Granulocyte colony-stimulating factor (G CSF; filgrastim or pegfilgrastim)

- Natalizumab 8 weeks: or 5 half-lives (whichever is longer)

- Licensed/registered and/or experimental anti-tumor necrosis factor (TNF) therapy
such as infliximab or adalimumab 4 weeks:

- 6-mercaptopurine

- Azathioprine

- Cyclophosphamide

- Methotrexate

- Mycophenolate mofetil

- Tacrolimus

- Cyclosporine

- Thalidomide

- Glucocorticoids, including budesonide and prednisone, or local glucocorticoid
therapy for Crohn's disease

- Any other immunosuppressive drugs

11. Use of any investigational drug within 4 weeks or 5 half-lives (whichever is longer)
prior to receiving the first dose of study drug

12. Use of nutritional therapy (parenteral nutrition or enteral nutrition with elemental
or semi-elemental diets) within 4 weeks prior to receiving the first dose of study
drug. If the physician judges that nutritional supplementation is needed, enteral
nutritional supplements will be allowed for patients who have been receiving a stable
regimen for at least 4 weeks prior to receiving the first dose of study drug and that
is intended to continue through the 8 week treatment period.

13. History of allergy to yeast products or to sargramostim or to any other excipient of
the study drug formulation

14. Active drug or alcohol abuse

15. Clinically important co-morbid conditions unrelated to Crohn's disease as determined
by the investigator

16. Previous randomization into this study, or into any other study of the sponsor's
sargramostim development program

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2006
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2006
Sample size
Target
Accrual to date
Final
33
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
- Liverpool
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- Caboolture
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- Adelaide
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- Frankston
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- Melbourne
Recruitment hospital [6] 0 0
- Brisbane
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- Hamilton
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- Sydney
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
- Caboolture
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5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3199 - Frankston
Recruitment postcode(s) [5] 0 0
3084 - Melbourne
Recruitment postcode(s) [6] 0 0
QLD 4000 - Brisbane
Recruitment postcode(s) [7] 0 0
3204 - Hamilton
Recruitment postcode(s) [8] 0 0
- Sydney
Recruitment outside Australia
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United States of America
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Alabama
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California
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Colorado
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District of Columbia
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Florida
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Georgia
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Indiana
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Kentucky
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Michigan
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Minnesota
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Missouri
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Nebraska
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New Jersey
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New York
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North Carolina
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Ohio
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Oklahoma
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Pennsylvania
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Texas
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Virginia
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Washington
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Brazil
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Santa Catarina
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SP
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Brazil
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Santos
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Brazil
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Sao Paulo
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Sofia
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Varna
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British Columbia
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Canada
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Manitoba
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New Brunswick
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Canada
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Newfoundland and Labrador
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Ontario
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Quebec
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Israel
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Haifa
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Israel
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Jerusalem
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Israel
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Rehovot
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Israel
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Tel Aviv
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Israel
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Zerifin
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Auckland
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New Zealand
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Dunedin
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New Zealand
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Hamilton
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New Zealand
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Tauranga
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Romania
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Bucharest
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Romania
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Craiova
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Russian Federation
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Kazan
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Russian Federation
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Krasnodar
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Russian Federation
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Moskva
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Russian Federation
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Sankt-Peterburg
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Russian Federation
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St. Petersburg
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South Africa
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Gauteng
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Western Cape
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South Africa
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Durban
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Ukraine
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Dnepropetrovsk
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Ivano-Frankovsk
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Kharkiv
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Kiev
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Lviv
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Ukraine
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Zaporozhye

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate if Leukine can induce clinical response or remission
in patients with Crohn's disease.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00295165
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00295165