Safety of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Adults With Stage II or III Pulmonary Sarcoidosis

NCT01440192 | Terminated Phase 1 DMC

• 1 other identifier: CCT-PDA001-SAR-001
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 4

Brief Summary

The primary objective of the study is to assess the safety and tolerability of a single dose of PDA001 (given twice) in subjects with Stage II or III Pulmonary Sarcoidosis (PS) who are refractory to one or more of the following treatments for PS: methotrexate,immunosuppressants or cytotoxic agents.

Conditions

Stage 2 Pulmonary Sarcoidosis; Stage 3 Pulmonary Sarcoidosis

Keywords

Sarcoidosis; Stem cells; PDA001; Celgene; Human Placenta-Derived cells; Cenplacel-L

Outcome Measures

Primary Outcomes (3)

• Evaluate pulmonary artery pressure during infusion

  Evaluate pulmonary artery pressure during infusion

  Day 1

• Adverse Events

  Number of Participants experiencing adverse events during the initial and extended follow-up periods

  24 months ( 2 years) from first dose - Study Day 1

• Evaluate pulse oximetry during infusion

  Evaluate pulse oximetry during infusion on Day 1 and on Day 8.

  Day 1 and Day 8

Secondary Outcomes (7)

• Change from baseline thru study day 731 in forced vital capacity (FVC)

  24 months ( 2 years) from first dose - Study Day 1

• Change from baseline thru study day 731 in forced expiratory volume (FEV1)

  24 months ( 2 years) from first dose - Study Day 1

• Change from baseline thru study day 731 in diffusing capacity of the lung for carbon monoxide (DLCO)

  24 months ( 2 years) from first dose - Study Day 1

• Change from baseline thru study day 731 in 6 minute walk test (6MWT)

  24 months ( 2 years) from first dose - Study Day 1

• Change from baseline thru study day 731 in St. George's Respiratory Questionnaire (SGRQ).

  24 months ( 2 years) from first dose - Study Day 1

Study Arms (2)

Cohort A: 1 Unit PDA001 (cenplacel-L)

EXPERIMENTAL

Biological: PDA001 (cenplacel-L)

Cohort B: 1 Unit PDA001 (cenplacel-L)

EXPERIMENTAL

1 unit PDA001(cenplacel-L)

Biological: PDA001 (cenplacel-L)

Interventions

PDA001 (cenplacel-L) BIOLOGICAL

1 unit PDA001 (approximately 200 x 106 cells) IV on Days 1 \& 8

Also known as: Human Placenta-Derived Cells

Cohort A: 1 Unit PDA001 (cenplacel-L); Cohort B: 1 Unit PDA001 (cenplacel-L)

Eligibility Criteria

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

You may qualify if:

• Male or female subjects 18 years to 75 years of age at the time of signing the informed consent document
• Understand and voluntarily sign an informed consent document prior to any study-related assessments/procedures are conducted
• Must be able to adhere to the study visit schedule and other protocol requirements
• Weight must be ≥ 50 kg
• A female of childbearing potential (FCBP) must have a negative serum or urine pregnancy test within 24 hours prior to treatment with study therapy. In addition, sexually active FCBP must agree to use two of the following adequate forms of contraception methods simultaneously such as: oral, injectable or implantable hormonal contraception; tubal ligation; intrauterine device; barrier contraceptive with spermicide; or vasectomized partner for the duration of the study and the follow-up period. 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
• Diagnosis of sarcoidosis as evidenced by parenchymal disease on chest radiograph (Stage II or III), as well as histologic confirmation of granulomatous inflammation and disease duration of ≥ 1 year
• Refractory to one or more of the following; methotrexate, immunosuppressants or cytotoxic agents
• Forced vital capacity (FVC) of ≥ 45% and ≤ 80% of predicted normal value at screening
• Must be on a stable dose of prednisone, methotrexate, and/or azathioprine for pulmonary Sarcoidosis for 4 weeks prior to infusion of the IP

You may not qualify if:

• Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study
• Any condition that confounds the ability to interpret data from 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
• Subjects with Stage I or Stage IV sarcoidosis
• Subjects with cutaneous sarcoidosis only
• Subjects with neurosarcoidosis or (clinically apparent) cardiac sarcoidosis
• Lung disease, other than sarcoid related, such as asthma, chronic obstructive pulmonary disease (COPD), interstitial lung disease (ILD)
• History of listeriosis, coccidiomycosis, histoplasmosis, blastomycosis, treated or untreated tuberculosis or exposure to individuals with tuberculosis
• History of pulmonary emboli or deep vein thrombus
• Active smoker or previous smoker \> 10 pack years (PY). Previous smokers must have discontinued smoking for at least 1 year
• Morbidly obese \[Body Mass Index (BMI)\] \> 35 at screening)
• Inability to perform 6 Minute Walk Test (6MWT) or Pulmonary Function Test (PFT) maneuvers
• Sickle cell disease (Hemoglobin SS, Hemoglobin SC, and sickle cell-beta thalassemia)
• Treatment at any time with B cell depleting therapies
• Any biologic anti-tumor necrosis factor (anti-TNF) therapy within the previous year

Sponsors & Collaborators

• Celularity Incorporated: lead

Study Sites (4)

University of Alabama, Birmingham - Division of Pulmonary, Allergy, and Critical Care Medicine

Birmingham, Alabama, 35223, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Cincinatti Medical Center

Cincinnati, Ohio, 45267-0565, United States

Location

The Cleveland Clinic Foundation - Respiratory Institute

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Sarcoidosis, Pulmonary; Sarcoidosis

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Study Officials

• Monica E Luchi, MD

  Celularity Incorporated

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2011
• First Posted: September 26, 2011
• Study Start: September 1, 2011
• Primary Completion: February 1, 2014
• Study Completion: February 1, 2014
• Last Updated: March 1, 2018

Record last verified: 2014-07

Locations

US (4)