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

This study is currently recruiting participants.
Verified March 2014 by Celgene Corporation
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT01769755
First received: January 15, 2013
Last updated: March 17, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Crohns Disease
Biological: PDA001
Drug: Vehicle Controlled Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Celgene Corporation:

Primary Outcome Measures:
  • Adverse Events [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]
    Number of participants experiencing adverse events during the initial and extended follow-up periods


Secondary Outcome Measures:
  • Efficacy [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
    To evaluate the time course of clinical efficacy of ascending doses of IV PDA001 versus placebo infused every other week for 8 weeks (5 total infusions) as measured by the Crohn's Disease Activity Index (CDAI) at each scheduled visit


Estimated Enrollment: 27
Study Start Date: January 2013
Estimated Study Completion Date: May 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Human Placenta-Derived Cells PDA001 Intravenous Infusion
Intravenous infusion of Human Placenta-Derived Cells PDA001 over the course of 2 hours.
Biological: PDA001

Cohort 1 Dose Level 1: ¼ unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart.

Cohort 2 Dose Level 2: ½ unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart.

Cohort 3 Dose Level 3: 1 unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart.

Placebo Comparator: Vehicle controlled placebo
Intravenous infusion of Vehicle Controlled Placebo over the course of 2 hours
Drug: Vehicle Controlled Placebo

Cohort 1 Dose Level 1: ¼ unit vehicle controlled placebo infused a total of 5 times 2 weeks apart.

Cohort 2 Dose Level 2: ½ unit vehicle controlled placebo infused a total of 5 times 2 weeks apart.

Cohort 3 Dose Level 3: 1 unit vehicle controlled placebo infused a total of 5 times 2 weeks apart.


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.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • • 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.

Exclusion Criteria:

  • 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).
  • Pregnant or lactating females.
  • Morbidly obese subjects Body Mass Index (BMI) > 35 at screening).
  • 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.
  • Subject has organic heart disease (eg, congestive heart failure), clinically significant arrhythmia or clinically significant abnormal findings on Electrocardiograms (ECG).
  • 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).
  • Subject has had a stricture of the bowel requiring hospitalization within 182 days prior to treatment with IP.
  • 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.
  • Subject has had any surgery within 28 days prior to treatment with IP.
  • Subject has a colostomy, ileostomy or ileal pouch anal anastomosis.
  • 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.
  • Subject has received previous cell therapy.
  • Subject is expecting to have elective surgery at any time between Visit 1 (screening) and Visit 7 (end of induction phase).
  • Subject has concurrent diagnosis of ulcerative colitis.
  • Subjects with protein C or S deficiency.
  • Subjects with prior history of thrombophlebitis or other pathological arterial or venous thrombosis.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01769755

Contacts
Contact: Associate Director Clinical Trial Disclosure 1-888-260-1599 clinicaltrialdisclosure@celgene.com
Contact: Lynn Ann O'Connell 732-652-6146 loconnell@celgene.com

Locations
United States, California
Cedars Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact    310-423-0035      
United States, Colorado
University of Colorado Hospital Recruiting
Aurora, Colorado, United States, 80045
Contact    303-724-7875      
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32608
Contact    352-265-8971      
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact    305-243-6405      
United States, New York
Rochester General Hospital Recruiting
Rochester, New York, United States, 14621
Contact    585-922-3536      
United States, Ohio
University of Cincinnati Recruiting
Cincinnati, Ohio, United States, 45267
Contact    513-475-7505      
Case Western University Recruiting
Cleveland, Ohio, United States, 44106
Contact    216-844-7214      
United States, Tennessee
Erlanger Medical Center Recruiting
Chattanooga, Tennessee, United States, 37403
Contact    423-778-2982      
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37212
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact    713-798-7616      
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact    801-587-9092      
United States, Virginia
McGuire VA Medical Center Recruiting
Richmond, Virginia, United States, 23249
Contact    804-675-6789      
Sponsors and Collaborators
Celgene Corporation
Investigators
Study Director: Steven Fischkoff, MD Celgene Corporation
  More Information

No publications provided

Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT01769755     History of Changes
Other Study ID Numbers: CCT-PDA001-CD-003
Study First Received: January 15, 2013
Last Updated: March 17, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Celgene Corporation:
Crohn's disease
Fistula
Diarrhea
Colon
Stem cells

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

ClinicalTrials.gov processed this record on April 17, 2014