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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

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.

Detailed Summary:
Sponsor: Celgene Corporation

Current Primary Outcome:

  • Evaluate pulmonary artery pressure during infusion [ Time Frame: Day 1 ]
    Evaluate pulmonary artery pressure during infusion
  • Adverse Events [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ]
    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 ]
    Evaluate pulse oximetry during infusion on Day 1 and on Day 8.


Original Primary Outcome: Rise in pulmonary artery pressure mean of ≥ 10 points [ Time Frame: Pre-dose and Post-dose on Day 1 ]

Current Secondary Outcome:

  • Change from baseline thru study day 731 in forced vital capacity (FVC) [ Time Frame: 24 months ( 2 years) from first dose - Study Day 1 ]
    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 ]
    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 ]
    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 ]
    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 ]
    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 ]
    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 ]
    Change from baseline thru study day 731 in baseline dyspnea index (BDI)/ transitional dyspnea index (TBI


Original Secondary Outcome:

  • ≥15% drop in oximetry from Day 1 or Day 8 pre-infusion value (after correction with supplemental oxygen) [ Time Frame: Pre-dose and Post-dose on Days 1 and 8 ]
  • Fever of ≥ 102 F [ Time Frame: 8 days ]
  • Rise in pulse of ≥ 100% or to an absolute value of 140 (whichever is lower) [ Time Frame: Pre-dose and Post-dose on Days 1 and 8 ]
  • Rise in systemic diastolic pressure of ≥ 20 mm Hg or to an absolute value of 120 [ Time Frame: Pre-dose and Post-dose on Days 1 and 8 ]


Information By: Celgene

Dates:
Date Received: September 19, 2011
Date Started: September 2011
Date Completion:
Last Updated: July 14, 2014
Last Verified: July 2014