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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Crohn's Disease

Intervention / Treatment
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* Biological: Human Placenta-Derived Cells PDA001 Intravenous Infusion
* Drug: Vehice Control


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females ages 18-75 years Understand and voluntarily sign an informed consent Able to adhere to the study visit schedule and other protocol requirements Minimum weight of 50 kg 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. Subject must have inflammatory CD 6 months prior to treatment and have confirmation of ongoing disease activity by colonoscopy or (other method) at screening Subject must have a CDAI score > 220 and < 450 as assessed between Visit 1 and Visit 2 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: Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study 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: has not undergone a hysterectomy or bilateral oophorectomy 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).

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1 unit Human Placenta-Derived Cells PDA001<br>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<br> <br> |
| Experimental: 4 units Human Placenta-Derived Cells PDA001<br>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<br> <br> |
| Placebo Comparator: vehicle control<br>4 units placebo in 240 mL infused intravenously in one arm on Day 0 and Day 7. | Drug: Vehice Control<br> <br> * Other names: Placebo;|
| Experimental: 8 units Human Placenta-Derived Cells PDA001<br>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<br> <br> |

What is the study measuring?
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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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Placenta-Derived Cells for the Treatment of Moderate-to-Severe Crohn's Disease