A Multi-Center Study to Evaluate the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Adults With Moderate-to-Severe Crohn's Disease

A Phase 2a, Randomized, Double Blind, Placebo Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Adults With Moderate-to-Severe Crohn's Disease

The primary objective of the study is to estimate the treatment effect of PDA001 (evaluating 3 different PDA001 dosings) versus placebo in subjects with moderate-to-severe Crohn's Disease. The secondary objective of the study is to assess the safety and tolerability of PDA001 versus placebo in the above-mentioned patient population.

Study Overview

• Status: Completed
• Conditions: Crohn's Disease
• Intervention / Treatment: Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion; Drug: Vehice Control

• Study Type: Interventional
• Enrollment (Actual): 50
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90048
      • Cedars Sinai Medical Center
    • Orange, California, United States, 92868
      • University of California, Irvine
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Yale School of Medicine Digestive Diseases
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Medical Center
  • Indiana
    • Indianapolis, Indiana, United States, 46202-5149
      • Indiana University
  • New York
    • New York, New York, United States, 10029
      • Mt Sinai Hospital
    • Rochester, New York, United States, 14621
      • Rochester General Hospital
    • Stony Brook, New York, United States, 11794-8173
      • Stony Brook University
  • Ohio
    • Cleveland, Ohio, United States, 44106-5066
      • Case Western Reserve University
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine
  • Virginia
    • Richmond, Virginia, United States, 23249
      • McGuire VA Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Males and females ages 18-75 years
2. Understand and voluntarily sign an informed consent
3. Able to adhere to the study visit schedule and other protocol requirements
4. Minimum weight of 50 kg
5. A female of childbearing potential (FCBP)† must have a negative urine pregnancy test at screening (Visit 1) and immediately prior to all doses of IP. In addition, sexually active FCBP must agree to use two of the adequate forms of contraception methods for the duration of the study and the follow-up period. A FCBP must agree to have pregnancy tests at least every 4 weeks during the study. Males (including those who have had a vasectomy) must agree to use barrier contraception (latex condoms) when engaging in reproductive sexual activity with FCBP for the duration of the study and the follow-up period.
6. Subject must have inflammatory CD 6 months prior to treatment and have confirmation of ongoing disease activity by colonoscopy or (other method) at screening
7. Subject must have a CDAI score > 220 and < 450 as assessed between Visit 1 and Visit 2
8. The Subject must have failed, i.e., had an inadequate response or lost response (recurrence of symptoms) to an agent, or documented intolerance to an agent at any time

Exclusion Criteria:

1. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study
2. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study

A female of childbearing potential is a sexually mature woman who:

1. has not undergone a hysterectomy or bilateral oophorectomy
2. has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 1 unit Human Placenta-Derived Cells PDA001; 1 unit PDA001 in 240 millilters (mL) infused intravenously in one arm on Day 0 and Day 7.	Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion
EXPERIMENTAL: 4 units Human Placenta-Derived Cells PDA001; 4 units PDA001 in 240 mL infused intravenously in one arm on Day 0 and Day 7.	Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion
PLACEBO_COMPARATOR: vehicle control; 4 units placebo in 240 mL infused intravenously in one arm on Day 0 and Day 7.	Drug: Vehice Control; Other Names: Placebo
EXPERIMENTAL: 8 units Human Placenta-Derived Cells PDA001; 4 units PDA-001 in 240 mL infused intravenously in each arm on Day 0 and Day 7 or 8 units PDA-001 in 240 mL infused intravenously in one arm on Day 0 and Day 7	Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
A reduction from baseline by 25% and/or > 100 points in the Crohn's Disease Activity Index (CDAI) scores at both Week 4 (Day 29) and at Week 6 (Day 43).	Crohn's disease activity index (CDAI) responders are subjects achieving either clinical response (a reduction in CDAI score of ≥100 points from Week 0), or remission (CDAI ≤150). CDAI is used to quantify the symptoms of subjects with Crohn's disease. A score of 150 or below indicates remission and a score above 450 indicates extremely severe disease.	Week 4 (Day 29) and Week 6 (Day 43)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The induction of a clinical remission defined as a Crohn's Disease Activity Index score of < 150 points at both Week 4 (Day 29) and Week 6 (Day 43)	Crohn's disease activity index (CDAI) is used to quantify the symptoms of subjects with Crohn's disease. A score of 150 or below indicates remission and a score above 450 indicates extremely severe disease.	Week 4 (Day 29) and Week 6 (Day 43)

Collaborators and Investigators

• Sponsor: Celularity Incorporated
• Collaborators: Celgene Corporation

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (ACTUAL): February 1, 2012
• Study Completion (ACTUAL): April 1, 2014

Study Registration Dates

• First Submitted: May 26, 2010
• First Submitted That Met QC Criteria: June 30, 2010
• First Posted (ESTIMATE): July 1, 2010

Study Record Updates

• Last Update Posted (ACTUAL): July 22, 2020
• Last Update Submitted That Met QC Criteria: July 20, 2020
• Last Verified: July 1, 2014

More Information

Terms related to this study

• Keywords: Human Placenta-Derived Cells for the Treatment of Moderate-to-Severe Crohn's Disease
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Inflammatory Bowel Diseases; Crohn Disease
• Other Study ID Numbers: CCT-PDA001-002