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

NCT ID: NCT01440192

Last Updated: 2018-03-01

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-09-30
• Study Completion Date: 2014-02-28

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort A: 1 Unit PDA001 (cenplacel-L)

Group Type: EXPERIMENTAL

PDA001 (cenplacel-L)

Intervention Type: BIOLOGICAL

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

Cohort B: 1 Unit PDA001 (cenplacel-L)

1 unit PDA001(cenplacel-L)

Group Type: EXPERIMENTAL

PDA001 (cenplacel-L)

Intervention Type: BIOLOGICAL

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

Interventions

PDA001 (cenplacel-L)

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

Intervention Type: BIOLOGICAL

Other Intervention Names

Human Placenta-Derived Cells

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celularity Incorporated

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Monica E Luchi, MD

Role: STUDY_DIRECTOR

Celularity Incorporated

Locations

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

Birmingham, Alabama, United States

National Jewish Health

Denver, Colorado, United States

University of Cincinatti Medical Center

Cincinnati, Ohio, United States

The Cleveland Clinic Foundation - Respiratory Institute

Cleveland, Ohio, United States

Countries

United States

Other Identifiers

CCT-PDA001-SAR-001

Identifier Type: -

Identifier Source: org_study_id