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

This study has been terminated.
(Study Terminated by Sponsor)
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT01440192
First received: September 19, 2011
Last updated: July 14, 2014
Last verified: July 2014
  Purpose

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.


Condition Intervention Phase
Stage 2 Pulmonary Sarcoidosis
Stage 3 Pulmonary Sarcoidosis
Biological: PDA001 (cenplacel-L)
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1B, Multi-Center, Open-Label, Single Dose Study to Evaluate the Safety of Intravenous Infusion of Human Placental-Derived Cells (PDA001) for the Treatment of Adults With Stage II or III Pulmonary Sarcoidosis.Sarcoidosis

Resource links provided by NLM:


Further study details as provided by Celgene Corporation:

Primary Outcome Measures:
  • Evaluate pulmonary artery pressure during infusion [ Time Frame: Day 1 ] [ Designated as safety issue: Yes ]
    Evaluate pulmonary artery pressure during infusion

  • Adverse Events [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: Yes ]
    Number of Participants experiencing adverse events during the initial and extended follow-up periods

  • Evaluate pulse oximetry during infusion [ Time Frame: Day 1 and Day 8 ] [ Designated as safety issue: Yes ]
    Evaluate pulse oximetry during infusion on Day 1 and on Day 8.


Secondary Outcome Measures:
  • Change from baseline thru study day 731 in forced vital capacity (FVC) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in forced vital capacity (FVC)

  • Change from baseline thru study day 731 in forced expiratory volume (FEV1) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in forced expiratory volume (FEV1)

  • Change from baseline thru study day 731 in diffusing capacity of the lung for carbon monoxide (DLCO) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in diffusing capacity of the lung for carbon monoxide (DLCO)

  • Change from baseline thru study day 731 in 6 minute walk test (6MWT) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in 6 minute walk test (6MWT)

  • Change from baseline thru study day 731 in St. George's Respiratory Questionnaire (SGRQ). [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in St. George's Respiratory Questionnaire (SGRQ).

  • Change from baseline thru study day 731 in Fatigue Assessment Score (FAS) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in Fatigue Assessment Score (FAS)

  • Change from baseline thru study day 731 in baseline dyspnea index (BDI)/ transitional dyspnea index (TBI [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ] [ Designated as safety issue: No ]
    Change from baseline thru study day 731 in baseline dyspnea index (BDI)/ transitional dyspnea index (TBI


Enrollment: 4
Study Start Date: September 2011
Study Completion Date: February 2014
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort A: 1 Unit PDA001 (cenplacel-L) Biological: PDA001 (cenplacel-L)
1 unit PDA001 (approximately 200 x 106 cells) IV on Days 1 & 8
Other Names:
  • Human Placenta-Derived Cells
  • PDA001 (cenplacel-L)
Experimental: Cohort B: 1 Unit PDA001 (cenplacel-L)
1 unit PDA001(cenplacel-L)
Biological: PDA001 (cenplacel-L)
1 unit PDA001 (approximately 200 x 106 cells) IV on Days 1 & 8
Other Names:
  • Human Placenta-Derived Cells
  • PDA001 (cenplacel-L)

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female subjects 18 years to 75 years of age at the time of signing the informed consent document
  2. Understand and voluntarily sign an informed consent document prior to any study-related assessments/procedures are conducted
  3. Must be able to adhere to the study visit schedule and other protocol requirements
  4. Weight must be ≥ 50 kg
  5. 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
  6. 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
  7. Refractory to one or more of the following; methotrexate, immunosuppressants or cytotoxic agents
  8. Forced vital capacity (FVC) of ≥ 45% and ≤ 80% of predicted normal value at screening
  9. Must be on a stable dose of prednisone, methotrexate, and/or azathioprine for pulmonary Sarcoidosis for 4 weeks prior to infusion of the IP

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 that confounds the ability to interpret data from the study
  3. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study
  4. Subjects with Stage I or Stage IV sarcoidosis
  5. Subjects with cutaneous sarcoidosis only
  6. Subjects with neurosarcoidosis or (clinically apparent) cardiac sarcoidosis
  7. Lung disease, other than sarcoid related, such as asthma, chronic obstructive pulmonary disease (COPD), interstitial lung disease (ILD)
  8. History of listeriosis, coccidiomycosis, histoplasmosis, blastomycosis, treated or untreated tuberculosis or exposure to individuals with tuberculosis
  9. History of pulmonary emboli or deep vein thrombus
  10. Active smoker or previous smoker > 10 pack years (PY). Previous smokers must have discontinued smoking for at least 1 year
  11. Morbidly obese [Body Mass Index (BMI)] > 35 at screening)
  12. Inability to perform 6 Minute Walk Test (6MWT) or Pulmonary Function Test (PFT) maneuvers
  13. Sickle cell disease (Hemoglobin SS, Hemoglobin SC, and sickle cell-beta thalassemia)
  14. Treatment at any time with B cell depleting therapies
  15. Any biologic anti-tumor necrosis factor (anti-TNF) therapy within the previous year
  16. Active infection requiring treatment within 30 days prior to screening
  17. Pregnant or lactating females
  18. Aspartate transaminase (AST), alanine aminotransferase (ALT) or creatine phosphokinase (CPK) > 2 x the upper limit of normal at screening
  19. Active infection with hepatitis B or hepatitis C
  20. Known infection with human immunodeficiency virus (HIV)
  21. Creatinine level > 1.5 times the upper limit of normal
  22. Platelet count < 100,000/µL (< 100 x 109/L)
  23. White blood cell count < 3,000/cu mm (< 3.0 x 109/L) or >20,000/cu mm (> 20 x 109/L)
  24. Organic heart disease (e.g., congestive heart failure, cor pulmonale), myocardial infarction within six months prior to screening
  25. Clinically significant findings on electrocardiogram (ECG) at screening (eg, arrhythmia)
  26. History of other malignancies within 5 years (except basal cell carcinoma of the skin that is surgically cured, remote history of cancer now considered cured or positive Pap smear with subsequent negative follow-up)
  27. Documented prior history of neurological disease or evidence of ongoing neurological disease
  28. Known allergy to bovine or porcine products
  29. Subject has received an investigational agent (an agent or device not approved by Federal Drug Administration (FDA) for marketed use in any indication) within 90 days (or 5 half-lives, whichever is longer) prior to treatment with investigational product (IP)
  30. Subject who has received previous cell therapy
  31. Subject is expecting to have elective surgery within 12 weeks prior to or post dosing with IP if the surgery would be expected to confound evaluation of outcome endpoints
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01440192

Locations
United States, Alabama
University of Alabama, Birmingham - Division of Pulmonary, Allergy, and Critical Care Medicine
Birmingham, Alabama, United States, 35223
United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
United States, Ohio
University of Cincinatti Medical Center
Cincinnati, Ohio, United States, 45267-0565
The Cleveland Clinic Foundation - Respiratory Institute
Cleveland, Ohio, United States, 44195
Sponsors and Collaborators
Celgene Corporation
Investigators
Study Director: Steven Fischkoff Celgene Corporation
  More Information

No publications provided

Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT01440192     History of Changes
Other Study ID Numbers: CCT-PDA001-SAR-001
Study First Received: September 19, 2011
Last Updated: July 14, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Celgene Corporation:
Sarcoidosis
Stem cells
PDA001
Celgene
Human Placenta-Derived cells
Cenplacel-L

Additional relevant MeSH terms:
Sarcoidosis
Sarcoidosis, Pulmonary
Lymphoproliferative Disorders
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on July 31, 2014