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

NCT ID: NCT01769755

Last Updated: 2018-03-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-03-31
• Study Completion Date: 2014-11-30

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.

Conditions

Crohns Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Human Placenta-Derived Cells PDA001 Intravenous Infusion

Intravenous infusion of Human Placenta-Derived Cells PDA001 over the course of 2 hours.

Group Type: EXPERIMENTAL

PDA001

Intervention Type: BIOLOGICAL

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.

Vehicle controlled placebo

Intravenous infusion of Vehicle Controlled Placebo over the course of 2 hours

Group Type: PLACEBO_COMPARATOR

Vehicle Controlled Placebo

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: BIOLOGICAL

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.

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celularity Incorporated

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Monica E Luchi, MD

Role: STUDY_DIRECTOR

Celularity Incorporated

Locations

Cedars Sinai Medical Center, Inflammatory Bowel Disease Center

Los Angeles, California, United States

University of Colorado Health Science Center

Denver, Colorado, United States

University of Florida

Gainesville, Florida, United States

University of Miami School of Medicine

Miami, Florida, United States

Rochester General Hospital

Rochester, New York, United States

University of Cincinnati Medical Center

Cincinnati, Ohio, United States

Case Western Reserve University Hospitals of Cleveland

Cleveland, Ohio, United States

Erlanger Baroness Hospital

Chattanooga, Tennessee, United States

Vanderbilt - Ingram Cancer Center

Nashville, Tennessee, United States

Baylor College of Medicine

Houston, Texas, United States

University of Utah

Salt Lake City, Utah, United States

McGuire Veterans Affairs Medical Center

Richmond, Virginia, United States

Countries

United States

Other Identifiers

CCT-PDA001-CD-003

Identifier Type: -

Identifier Source: org_study_id