PDA001 for the Treatment of Adults With Moderate-to-Severe Crohn's Disease

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Intravenous PDA001 for the Treatment of Adults With Moderate-to-Severe Crohn's Disease

This Phase 2a study evaluated the efficacy, safety, and tolerability of intravenous human placenta-derived cells (PDA001) compared with placebo in adults with moderate-to-severe Crohn's disease. Three PDA001 dose levels were evaluated.

Study Overview

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

Detailed Description

This was a Phase 2a, multicenter study designed to evaluate the safety, tolerability, and efficacy of intravenous human placenta-derived cells (PDA001) in adults with moderate-to-severe Crohn's disease.

The study consisted of sequential cohorts. Initial open-label dose-escalation cohorts evaluated PDA001 administered intravenously at a dose of 1.6 × 10^9 cells (8 units). A subsequent randomized, double-blind, placebo-controlled cohort evaluated PDA001 administered intravenously at doses of 200 × 10^6 cells (1 unit) and 800 × 10^6 cells (4 units) compared with placebo.

The primary efficacy objective was to evaluate induction of clinical response, defined as a reduction from baseline of 25% and/or at least 100 points in the Crohn's Disease Activity Index (CDAI) at both Week 4 and Week 6. Secondary objectives included evaluation of clinical remission, safety, and tolerability.

Subjects were followed during an induction phase and could participate in an extension phase with re-treatment according to protocol-defined criteria.

• 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

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 Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 200 × 10^6 cells of Human Placenta-Derived Cells PDA001; PDA001 administered intravenously at a dose of 1 unit, approximately 200 × 10^6 cells, on Study Day 0 and Study Day 7.	Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion
Experimental: 800 × 10^6 cells of Human Placenta-Derived Cells PDA001; PDA001 administered intravenously at a dose of 4 units, approximately 800 × 10^6 cells, on Study Day 0 and Study Day 7.	Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion
Placebo Comparator: vehicle control; Matching placebo administered intravenously on Study Day 0 and Study Day 7.	Drug: Vehicle Control; Other Names: Placebo
Experimental: 1.6 × 10^9 cells of Human Placenta-Derived Cells PDA001; PDA001 administered intravenously at a dose of 8 units, approximately 1.6 × 10^9 cells, on Study Day 0 and Study 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
• Investigators: Study Director: sharmila koppisetti, MD, Celularity Incorporated

Publications and helpful links

General Publications

• Melmed GY, Pandak WM, Casey K, Abraham B, Valentine J, Schwartz D, Awais D, Bassan I, Lichtiger S, Sands B, Hanauer S, Richards R, Oikonomou I, Parekh N, Targan S, Johnson K, Hariri R, Fischkoff S. Human Placenta-derived Cells (PDA-001) for the Treatment of Moderate-to-severe Crohn's Disease: A Phase 1b/2a Study. Inflamm Bowel Dis. 2015 Aug;21(8):1809-16. doi: 10.1097/MIB.0000000000000441.

Helpful Links

• PDA-001 for treatment of Crohns disease

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 (Estimated): July 1, 2010

Study Record Updates

• Last Update Posted (Actual): July 7, 2026
• Last Update Submitted That Met QC Criteria: July 1, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Crohns disease; Human Placenta-Derived Cells
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease
• Other Study ID Numbers: CCT-PDA001-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No