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

Phase 2

Completed

• Conditions: Crohn's Disease
• Interventions: Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion; Drug: Vehice Control

• Registration Number: NCT01155362
• Lead Sponsor: Celularity Incorporated

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-26
• Study First Submitted QC: 2010-06-30
• Study First Posted: 2010-07-01
• Study Start: 2010-08
• Primary Completion: 2012-02
• Disposition First Submitted: 2013-08-07
• Disposition First Submitted QC: 2013-08-07
• Disposition First Posted: 2013-08-12
• Study Completion: 2014-04
• Status Verified: 2014-07
• Last Update Submitted: 2020-07-20
• Last Update Posted: 2020-07-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1 unit Human Placenta-Derived Cells PDA001	Human Placenta-Derived Cells PDA001 Intravenous Infusion	1 unit PDA001 in 240 millilters (mL) infused intravenously in one arm on Day 0 and Day 7.
4 units Human Placenta-Derived Cells PDA001	Human Placenta-Derived Cells PDA001 Intravenous Infusion	4 units PDA001 in 240 mL infused intravenously in one arm on Day 0 and Day 7.
vehicle control	Vehice Control	4 units placebo in 240 mL infused intravenously in one arm on Day 0 and Day 7.
8 units Human Placenta-Derived Cells PDA001	Human Placenta-Derived Cells PDA001 Intravenous Infusion	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

Primary Outcome Measures

Name	Time	Method
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).	Week 4 (Day 29) and 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.

Secondary Outcome Measures

Name	Time	Method
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)	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.

Trial Locations

Locations (13)

University of Chicago Medical Center; 🇺🇸 Chicago, Illinois, United States

Case Western Reserve University; 🇺🇸 Cleveland, Ohio, United States

Yale School of Medicine Digestive Diseases; 🇺🇸 New Haven, Connecticut, United States

Indiana University; 🇺🇸 Indianapolis, Indiana, United States

Mt Sinai Hospital; 🇺🇸 New York, New York, United States

Stony Brook University; 🇺🇸 Stony Brook, New York, United States

Rochester General Hospital; 🇺🇸 Rochester, New York, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Cedars Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Vanderbilt University; 🇺🇸 Nashville, Tennessee, United States

McGuire VA Medical Center; 🇺🇸 Richmond, Virginia, United States

University of California, Irvine; 🇺🇸 Orange, California, United States