Study of Human Placenta-derived Cells (PDA001) to Evaluate the Safety and Effectiveness for Patients With Active Rheumatoid Arthritis

NCT ID: NCT01261403

Last Updated: 2020-07-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

26 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-12-31

Study Completion Date

2013-09-30

Brief Summary

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The primary objective of the study is to assess the safety and efficacy of 2 dose groups (PDA001 versus vehicle control) in subjects with active rheumatoid Arthritis. The secondary objectives of the study are to determine the clinical response at defined visit intervals, determine the time to flare of RA symptoms and to quantify changes in inflammatory markers including C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum amyloid A (SAA), and IL-6.

Detailed Description

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Conditions

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Rheumatoid Arthritis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Group 1

1 Unit of PDA001 or 4 units Vehicle Control intravenous on Day 0 and Day 7

Group Type EXPERIMENTAL

PDA001

Intervention Type BIOLOGICAL

Dose escalation study: Subjects will be assigned to 1 of 2 treatment groups (1 unit or 4 units vs. vehicle control) based on the order in which they enroll in the study. Intravenous infusion will be administered on days 0 and 7. Nonresponders will be unblinded after 12 weeks of study. Non- responders on vehicle control will be re-dosed with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). Nonresponders taking active PDA001 will enter the safety follow-up portion of the study. Responders at 12 weeks will continue in the safety and efficacy follow-up portion of the study until 12 months of study. Responders will be treated for RA flare between 3-9 months of study with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart).

Vehicle Controlled Placebo

Intervention Type DRUG

Cohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7

Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7

Group 2

4 Units PDA001 or 4 units Vehicle Control intravenous (Placebo) on Day 0 and Day 7

Group Type EXPERIMENTAL

PDA001

Intervention Type BIOLOGICAL

Dose escalation study: Subjects will be assigned to 1 of 2 treatment groups (1 unit or 4 units vs. vehicle control) based on the order in which they enroll in the study. Intravenous infusion will be administered on days 0 and 7. Nonresponders will be unblinded after 12 weeks of study. Non- responders on vehicle control will be re-dosed with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). Nonresponders taking active PDA001 will enter the safety follow-up portion of the study. Responders at 12 weeks will continue in the safety and efficacy follow-up portion of the study until 12 months of study. Responders will be treated for RA flare between 3-9 months of study with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart).

Vehicle Controlled Placebo

Intervention Type DRUG

Cohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7

Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7

Vehicle control

Placebo - Vehicle Control Arm

Group Type PLACEBO_COMPARATOR

Vehicle Controlled Placebo

Intervention Type DRUG

Cohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7

Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7

Interventions

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PDA001

Dose escalation study: Subjects will be assigned to 1 of 2 treatment groups (1 unit or 4 units vs. vehicle control) based on the order in which they enroll in the study. Intravenous infusion will be administered on days 0 and 7. Nonresponders will be unblinded after 12 weeks of study. Non- responders on vehicle control will be re-dosed with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). Nonresponders taking active PDA001 will enter the safety follow-up portion of the study. Responders at 12 weeks will continue in the safety and efficacy follow-up portion of the study until 12 months of study. Responders will be treated for RA flare between 3-9 months of study with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart).

Intervention Type BIOLOGICAL

Vehicle Controlled Placebo

Cohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7

Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Subjects must satisfy the following criteria to be enrolled in the study:

1. Diagnosis of RA as defined by the 1987-revised ACR criteria.
2. RA, characterized by at least 6 out of 66 swollen joints and 6 out of 68 tender joints at Screening and Baseline, and at least 2 of the following: a C reactive protein level (CRP), greater than the upper limit of normal (ULN) ³ 1 mg/dl, an erythrocyte sedimentation rate (ESR) of at least 28 mm per hour, or morning stiffness lasting longer than 45 minutes.
3. Non responsive or intolerant to a minimum of 2 RA therapies which are classified as DMARDs, biologics, or corticosteroids.
4. Biological medication must be discontinued 30 day prior to the first dose of study drug, except golimumab and actemra which are 60 days, and infliximab, alefacept and efalizumab, which must have been discontinued 90 days prior to the first dose of IP.
5. Cytotoxic agents, including but not limited to chlorambucil, cyclophosphamide, nitrogen mustard, or other alkylating agents must have been discontinued 90 days prior to the first dose of IP.
6. Subjects must be able to tolerate intravenous infusions in both arms.
7. There should be no change in RA medication dose anticipated during the initial treatment and re treatment periods and the respective 12 week follow-up period for each dosing treatment period. The following rules apply for anti-rheumatic medications taken during the study:
8. DMARDs must have must have been stable for least 90 days prior to dosing with IP.
9. Low -dose corticosteroids are permitted (prednisolone ≤ 10 mg per day or equivalent). Corticosteroids must have been stable for at least 30 days prior to dosing with IP.
10. DMARDs and corticosteroids must remain stable throughout the initial 3 months of study and throughout subsequent treatment periods

The presence of any of the following will exclude a subject from enrollment:

1. Prior use of rituximab and other B-cell depleting therapies, abatacept, prior use of more than 2 biologic therapies.
2. Subject has received an investigational agent in any indication-within 60 days (or 5 half-lives, whichever is longer) prior to treatment with IP.
3. Subject has received previous cell therapy.
4. Serum creatinine concentration \> 2.0 mg/dl at screening.
5. Alkaline phosphatase \> 2.5x the upper limit of normal at screening.
6. Bilirubin level \> 1.5 mg/dL (unless subject has known Gilbert's disease).
7. Untreated chronic infection or treatment of any infection with antibiotics within 4 weeks prior to dosing with IP.
8. Positive HIV test at Screening. Positive Hepatitis B surface antigen at Screening. Positive Hepatitis C antibody at Screening.
9. Organic heart disease (e.g., congestive heart failure), myocardial infarction within six (6) months prior to screening or clinically significant findings on ECG at screening. Clinically significant arrhythmia.
10. Primary or secondary immunodeficiency.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Celgene Corporation

INDUSTRY

Sponsor Role collaborator

Celularity Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Solveig Ericson, MD

Role: STUDY_DIRECTOR

Celularity Incorporated

Locations

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Pinnacle Research Group

Anniston, Alabama, United States

Site Status

Advanced Pain Research Institute

Arcadia, California, United States

Site Status

UCLA

Los Angeles, California, United States

Site Status

Desert Medical Advances

Palm Desert, California, United States

Site Status

Sanitas Research

Coral Gables, Florida, United States

Site Status

Compass Research, LLC

Orlando, Florida, United States

Site Status

Progressive Medical Research

Port Orange, Florida, United States

Site Status

Four Rivers Clinical Research Inc.

Paducah, Kentucky, United States

Site Status

Arthritis and Diabetes Clinic, Inc

Monroe, Louisiana, United States

Site Status

St. Paul Rheumatology, PA

Eagan, Minnesota, United States

Site Status

SNS Rheumatology

Lakewood, New Jersey, United States

Site Status

David R. Mandel, MD, Inc.

Mayfield, Ohio, United States

Site Status

Health Research of Oklahoma

Oklahoma City, Oklahoma, United States

Site Status

Health Research Institute

Oklahoma City, Oklahoma, United States

Site Status

Altoona Center for Clinical Research

Duncansville, Pennsylvania, United States

Site Status

Metroplex Clinical Research Center

Dallas, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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CCT-PDA001-RA-001

Identifier Type: -

Identifier Source: org_study_id

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