A Randomized, Double-Blind, Placebo-Controlled Dose-Escalation Study to Determine the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Crohn's Disease
- Conditions
- Crohns Disease
- Interventions
- Drug: Vehicle Controlled PlaceboBiological: PDA001
- Registration Number
- NCT01769755
- Lead Sponsor
- Celularity Incorporated
- Brief Summary
To assess the safety and efficacy of intravenous (IV) PDA001 infused every two weeks for up to 5 total infusions in subjects with Crohn's disease who are refractory to one or more standard Crohn's disease therapies.
- Detailed Description
This is a randomized, double-blind, placebo-controlled, dose-escalation study to study 3 cohorts of subjects with Crohn's Disease including (but not limited to) those with colonic involvement. Each cohort (n = 9) will include PDA001 treated subjects (n = 6) as well as placebo (vehicle control) treated subjects (n = 3). Cohorts will be enrolled sequentially, beginning with the lowest dose cohort (1/4 unit PDA001) and progressing until the maximum tolerated dose of IV PDA001 is determined.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 14
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• Males and females 18 - 75 years of age at the time of signing the informed consent document.
- Minimum weight of subject is 40 kg at screening.
- Subject must have inflammatory Crohn's Disease (CD) diagnosed at least 6 months but no greater than 15 years prior to treatment with Investigational Product (IP).
- Subject must have confirmation of ongoing CD by ileocolonoscopy at screening.
- Subject must have a Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450 as assessed between Visit 1 and Visit 2.
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Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study including but not limited to
- Liver Function Tests Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) > 2.5 x the upper limit of normal at screening.
- Serum creatinine concentration > 2.0 mg/dl at screening. Alkaline phosphatase > 2.5 x the upper limit of normal at screening.
- Bilirubin level > 2 mg/dL (unless subject has known Gilbert's disease).
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Pregnant or lactating females.
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Morbidly obese subjects Body Mass Index (BMI) > 35 at screening).
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Subject has untreated chronic infection including Clostridium difficile toxin positive at screening or treatment of any infection with antibiotics within 4 weeks prior to dosing with IP (other than a treated urinary tract infection or drained perianal abscess). Note: Stable doses of antibiotics used to treat Crohn's Disease are allowed.
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Subject has organic heart disease (eg, congestive heart failure), clinically significant arrhythmia or clinically significant abnormal findings on Electrocardiograms (ECG).
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Subject has a history of other malignancies within 5 years (except basal cell carcinoma of the skin that is surgically cured, remote history of cancer now considered cured or positive Pap smear with subsequent negative follow up).
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Subject has had a stricture of the bowel requiring hospitalization within 182 days prior to treatment with IP.
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Subject has had bowel surgery other than perianal (for example, fistulotomy, seton placement, or abscess drainage) or previous abscess drainage within 182 days prior to treatment with IP.
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Subject has had any surgery within 28 days prior to treatment with IP.
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Subject has a colostomy, ileostomy or ileal pouch anal anastomosis.
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Subject has received an investigational agent -an agent or device not approved by FDA for marketed use in any indication-within 90 days (or 5 half-lives, whichever is longer) prior to treatment with investigational product.
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Subject has received previous cell therapy.
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Subject is expecting to have elective surgery at any time between Visit 1 (screening) and Visit 7 (end of induction phase).
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Subject has concurrent diagnosis of ulcerative colitis.
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Subjects with protein C or S deficiency.
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Subjects with prior history of thrombophlebitis or other pathological arterial or venous thrombosis.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Vehicle controlled placebo Vehicle Controlled Placebo Intravenous infusion of Vehicle Controlled Placebo over the course of 2 hours Human Placenta-Derived Cells PDA001 Intravenous Infusion PDA001 Intravenous infusion of Human Placenta-Derived Cells PDA001 over the course of 2 hours.
- Primary Outcome Measures
Name Time Method Adverse Events Up to 1 year Number of participants experiencing adverse events during the initial and extended follow-up periods
- Secondary Outcome Measures
Name Time Method Clinical Response Up to 1 year A clinical response is defined as a reduction from baseline by 25% and/or ≥ 100 points in the Crohn's Disease Activity Index (CDAI) score. The Crohn's Disease Activity Index or CDAI is a research tool used to quantify the symptoms of patients with Crohn's disease.
Clinical Remission Up to 1 year Clinical remission is defined as a Crohn's Disease Activity Index (CDAI) score CDAI score of ≤ 150 points
Related Research Topics
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Trial Locations
- Locations (12)
McGuire Veterans Affairs Medical Center
🇺🇸Richmond, Virginia, United States
Cedars Sinai Medical Center, Inflammatory Bowel Disease Center
🇺🇸Los Angeles, California, United States
University of Miami School of Medicine
🇺🇸Miami, Florida, United States
Case Western Reserve University Hospitals of Cleveland
🇺🇸Cleveland, Ohio, United States
University of Cincinnati Medical Center
🇺🇸Cincinnati, Ohio, United States
Vanderbilt - Ingram Cancer Center
🇺🇸Nashville, Tennessee, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Erlanger Baroness Hospital
🇺🇸Chattanooga, Tennessee, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University of Colorado Health Science Center
🇺🇸Denver, Colorado, United States
Rochester General Hospital
🇺🇸Rochester, New York, United States