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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00482092




Registration number
NCT00482092
Ethics application status
Date submitted
30/05/2007
Date registered
4/06/2007
Date last updated
13/01/2022

Titles & IDs
Public title
Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
Scientific title
A Phase III, Multicenter, Placebo-controlled, Randomized, Double-blind Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Induction of Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease
Secondary ID [1] 0 0
CRD 603
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Crohn's 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 - Prochymal®
Treatment: Drugs - Placebo

Placebo comparator: Placebo - Participants will receive matching placebo administered as intravenous (IV) infusions.

Active comparator: Prochymal® - Low dose - Participants will receive a total dose of Prochymal® 600 x 10\^6 cells, IV infusion, on four days, once daily.

Active comparator: Prochymal® - High dose - Participants will receive a total dose of Prochymal® 1200 x 10\^6 cells, IV infusion, on four days, once daily.


Treatment: Drugs: Prochymal®
Prochymal® IV infusion

Treatment: Drugs: Placebo
Prochymal® Placebo-matching IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Disease remission (CDAI at or below 150)
Timepoint [1] 0 0
28 days
Secondary outcome [1] 0 0
Disease improvement (Reduction by at least 100 points in CDAI)
Timepoint [1] 0 0
28 days
Secondary outcome [2] 0 0
Improvement in quality of life (IBDQ)
Timepoint [2] 0 0
28 days
Secondary outcome [3] 0 0
Reduction in number of draining fistulas
Timepoint [3] 0 0
28 days

Eligibility
Key inclusion criteria
* failed (within last 2 yr) or intolerant of at least one steroid AND at least one immunosuppressant AND at EXACTLY one biologic
* CDAI between 250 and 450, inclusive
* endoscopically or radiographically confirmed Crohn's disease of ileus or colon or both
* C-Reactive Protein Test (CRP) of at least 5 mg/l (0.5 mg/dl)*OR* CDAI of at least 300
* weight between 40 and 150 kg, inclusive
* adequate renal function
* negative tuberculosis skin (PPD) test (or evaluated low risk of TB activation)
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* HIV or hepatitis infection active
* allergy to CT contrast agents, or to bovine or porcine products
* symptomatic fibrostenotic Crohn's disease
* permanent ostomy
* biologic therapy within past 90 d
* prednisone greater than 20 mg/d within past month
* short-bowel syndrome
* total parenteral nutrition
* abnormal liver function
* malignancy active within past 5 years (except completely resected basal or squamous cell carcinoma of skin)
* enteric pathogens, including C. difficile
* history of colonic mucosal dysplasia
* current or prior evidence of tuberculosis (TB) (unless risk of activation or re-activation deemed low)

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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/09/2007
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/09/2014
Sample size
Target
Accrual to date
Final
330
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment outside Australia
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United States of America
State/province [1] 0 0
California
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United States of America
State/province [2] 0 0
Colorado
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United States of America
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Florida
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United States of America
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Georgia
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Illinois
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Indiana
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Kansas
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Kentucky
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Louisiana
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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New Hampshire
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New York
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North Carolina
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Oklahoma
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Pennsylvania
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Tennessee
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Texas
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Vermont
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United States of America
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Virginia
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United States of America
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Washington
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Canada
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Alberta
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Canada
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Manitoba
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Canada
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Ontario
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New Zealand
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Christchurch
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New Zealand
State/province [29] 0 0
Hamilton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Mesoblast, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Protocol 603 is enrolling subjects with moderate-to-severe Crohn's disease who are intolerant to, or have previously failed therapy with, at least one steroid and at least one immunosuppressant and a biologic monoclonal anti-body to tumor necrosis factor alpha. The protocol investigates the safety and efficacy of using PROCHYMAL® adult human stem cells to induce remission. PROCHYMAL is delivered through a vein in the arm four times over two weeks, for approximately an hour each time.
Trial website
https://clinicaltrials.gov/study/NCT00482092
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mahboob Rahman, MD
Address 0 0
Mesoblast, Inc.
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/study/NCT00482092