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

NCT01261403 | Terminated Phase 2 DMC

• 1 other identifier: CCT-PDA001-RA-001
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 16

Brief Summary

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.

Conditions

Rheumatoid Arthritis

Keywords

Human Placenta-Derived Cells

Outcome Measures

Primary Outcomes (1)

• Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response within 12 weeks following the initial dose of study drug

  A participant is a responder if the following 3 criteria for improvement from Baseline were met: • ≥ 20% improvement in 68 tender joint count; • ≥ 20% improvement in 66 swollen joint count; and • ≥ 20% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale \[VAS\]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein

  Baseline through Week 12

Secondary Outcomes (21)

• Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response at 6 months

  Baseline and 6 months

• Number/percent of subjects achieving an ACR 20 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.

  Baseline and each scheduled visit through 12 months following the first infusion of study drug

• Number/percent of subjects achieving an ACR 50 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.

  Baseline and each scheduled visit through 12 months following the first infusion of study drug.

• Number/percent of subjects achieving an ACR 70 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.

  Baseline and each scheduled visit through 12 months following the first infusion of study drug.

• Number/percent of subjects achieving a clinical response based on their Clinical Disease Activity Index (CDAI) at each scheduled visit during the first 12 months following the first infusion of study drug.

  Baseline and each scheduled visit through 12 months following the first infusion of study drug

Study Arms (3)

Group 1

EXPERIMENTAL

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

Biological: PDA001; Drug: Vehicle Controlled Placebo

Group 2

EXPERIMENTAL

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

Biological: PDA001; Drug: Vehicle Controlled Placebo

Vehicle control

PLACEBO COMPARATOR

Placebo - Vehicle Control Arm

Drug: Vehicle Controlled Placebo

Interventions

PDA001 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).

Group 1; Group 2

Vehicle Controlled Placebo 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 1; Group 2; Vehicle control

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must satisfy the following criteria to be enrolled in the study:
• Diagnosis of RA as defined by the 1987-revised ACR criteria.
• 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.
• Non responsive or intolerant to a minimum of 2 RA therapies which are classified as DMARDs, biologics, or corticosteroids.
• 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.
• 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.
• Subjects must be able to tolerate intravenous infusions in both arms.
• 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:
• DMARDs must have must have been stable for least 90 days prior to dosing with IP.
• 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.
• 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:
• Prior use of rituximab and other B-cell depleting therapies, abatacept, prior use of more than 2 biologic therapies.
• Subject has received an investigational agent in any indication-within 60 days (or 5 half-lives, whichever is longer) prior to treatment with IP.
• Subject has received previous cell therapy.

Sponsors & Collaborators

• Celularity Incorporated: lead
• Celgene Corporation: collaborator

Study Sites (16)

Pinnacle Research Group

Anniston, Alabama, 36207, United States

Location

Advanced Pain Research Institute

Arcadia, California, 91007, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

Desert Medical Advances

Palm Desert, California, 92260, United States

Location

Sanitas Research

Coral Gables, Florida, 33134, United States

Location

Compass Research, LLC

Orlando, Florida, 32806, United States

Location

Progressive Medical Research

Port Orange, Florida, 32127, United States

Location

Four Rivers Clinical Research Inc.

Paducah, Kentucky, 42003, United States

Location

Arthritis and Diabetes Clinic, Inc

Monroe, Louisiana, 71203, United States

Location

St. Paul Rheumatology, PA

Eagan, Minnesota, 55121, United States

Location

SNS Rheumatology

Lakewood, New Jersey, 08701, United States

Location

David R. Mandel, MD, Inc.

Mayfield, Ohio, 44143, United States

Location

Health Research of Oklahoma

Oklahoma City, Oklahoma, 73103, United States

Location

Health Research Institute

Oklahoma City, Oklahoma, 73109, United States

Location

Altoona Center for Clinical Research

Duncansville, Pennsylvania, 16635, United States

Location

Metroplex Clinical Research Center

Dallas, Texas, 75231, United States

Location

MeSH Terms

Conditions

Arthritis, Rheumatoid

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Solveig Ericson, MD

  Celularity Incorporated

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2010
• First Posted: December 16, 2010
• Study Start: December 1, 2010
• Primary Completion: February 1, 2012
• Study Completion: September 1, 2013
• Last Updated: July 22, 2020

Record last verified: 2014-07

Locations

US (16)