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

Celularity Incorporated50 enrolled

Overview

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.

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

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Crohn's Disease

Interventions

Human Placenta-Derived Cells PDA001 Intravenous InfusionBIOLOGICAL

Vehicle ControlDRUG

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

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).

Locations (13)

Cedars Sinai Medical Center

Los Angeles, California, United States

University of California, Irvine

Orange, California, United States

Yale School of Medicine Digestive Diseases

New Haven, Connecticut, United States

Outcomes

Primary Outcomes

Percentage of Subjects With Clinical Response Based on Crohn's Disease Activity Index (CDAI) at Both Week 4 and Week 6

Clinical response was defined as a reduction from baseline of at least 25% and/or at least 100 points in the Crohn's Disease Activity Index (CDAI) score at both Week 4 (Day 29) and Week 6 (Day 43). The Crohn's Disease Activity Index (CDAI) is a composite clinical index used to assess disease activity in Crohn's disease based on clinical signs, symptoms, and laboratory findings. Scores range from approximately 0 to 600, with higher scores indicating more severe disease activity and lower scores indicating less severe disease activity. Scores of 150 or less generally indicate clinical remission, whereas scores greater than 450 indicate very severe disease activity.

Time frame: Week 4 (Day 29) and Week 6 (Day 43)

Secondary Outcomes

Percentage of Subjects With Clinical Remission Based on Crohn's Disease Activity Index (CDAI) at Both Week 4 and Week 6

Clinical remission was defined as a Crohn's Disease Activity Index (CDAI) score of 150 or less at both Week 4 (Day 29) and Week 6 (Day 43). The Crohn's Disease Activity Index (CDAI) is a composite clinical index used to assess disease activity in Crohn's disease based on clinical signs, symptoms, and laboratory findings. Scores range from approximately 0 to 600, with higher scores indicating more severe disease activity and lower scores indicating less severe disease activity. Scores of 150 or less generally indicate clinical remission, whereas scores greater than 450 indicate very severe disease activity.

Time frame: Week 4 (Day 29) and Week 6 (Day 43)

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.