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Stage 2 Pulmonary SarcoidosisPhase 1NCT01440192

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

A Phase 1 study testing PDA001 (cenplacel-L) in people with Stage 2 Pulmonary Sarcoidosis and Stage 3 Pulmonary Sarcoidosis, run by Celularity Incorporated at 4 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
PDA001 (cenplacel-L)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
4 participants
Where
4 sitesUnited States
Study dates
Sep 2011to Feb 2014
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
Show all 31Show fewer
  • 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
  • Active infection requiring treatment within 30 days prior to screening
  • Pregnant or lactating females
  • Aspartate transaminase (AST), alanine aminotransferase (ALT) or creatine phosphokinase (CPK) > 2 x the upper limit of normal at screening
  • Active infection with hepatitis B or hepatitis C
  • Known infection with human immunodeficiency virus (HIV)
  • Creatinine level > 1.5 times the upper limit of normal
  • Platelet count < 100,000/µL (< 100 x 109/L)
  • White blood cell count < 3,000/cu mm (< 3.0 x 109/L) or >20,000/cu mm (> 20 x 109/L)
  • Organic heart disease (e.g., congestive heart failure, cor pulmonale), myocardial infarction within six months prior to screening
  • Clinically significant findings on electrocardiogram (ECG) at screening (eg, arrhythmia)
  • 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)
  • Documented prior history of neurological disease or evidence of ongoing neurological disease
  • Known allergy to bovine or porcine products
  • 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)
  • Subject who has received previous cell therapy
  • 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

Trial locations and contacts

4 sites in 1 country.

United States4 sites

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

    Birmingham, Alabama, 35223

  • University of Cincinatti Medical Center

    Cincinnati, Ohio, 45267-0565

  • The Cleveland Clinic Foundation - Respiratory Institute

    Cleveland, Ohio, 44195

  • National Jewish Health

    Denver, Colorado, 80206

Other trials in United States

About this study

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

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.

Being tested
PDA001 (cenplacel-L)
Keywords
Sarcoidosis, Stem cells, PDA001, Celgene, Human Placenta-Derived cells, Cenplacel-L
Medical subject headings
Sarcoidosis, Pulmonary, Sarcoidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PDA001 (cenplacel-L)Biological

    Also called Human Placenta-Derived Cells

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

Study groups in NCT01440192
Study groupRoleWhat they receive
Cohort A: 1 Unit PDA001 (cenplacel-L)ExperimentalNot described
Receives: Biological: PDA001 (cenplacel-L)
Cohort B: 1 Unit PDA001 (cenplacel-L)Experimental1 unit PDA001(cenplacel-L)
Receives: Biological: PDA001 (cenplacel-L)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Evaluate pulmonary artery pressure during infusion

    Evaluate pulmonary artery pressure during infusion

    Measured: Day 1

  2. Adverse Events

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

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

  3. Evaluate pulse oximetry during infusion

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

    Measured: Day 1 and Day 8

Secondary outcomes (7)

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

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

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

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

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

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

  3. 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 diffusing capacity of the lung for carbon monoxide (DLCO)

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

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

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

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

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

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

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

  6. Change from baseline thru study day 731 in Fatigue Assessment Score (FAS)

    Change from baseline thru study day 731 in Fatigue Assessment Score (FAS)

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

  7. Change from baseline thru study day 731 in baseline dyspnea index (BDI)/ transitional dyspnea index (TBI

    Change from baseline thru study day 731 in baseline dyspnea index (BDI)/ transitional dyspnea index (TBI

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Sep 2011 (estimated)
  2. Study startSep 2011
  3. Primary completionFeb 2014
  4. Study completionFeb 2014

Why it stopped: Study Terminated by Sponsor

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
4 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01440192
Sponsor's protocol ID
CCT-PDA001-SAR-001
Status last verified
Jul 2014
First submitted
19 Sep 2011
Last updated
1 Mar 2018
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing PDA001 (cenplacel-L) in people with Stage 2 Pulmonary Sarcoidosis and Stage 3 Pulmonary Sarcoidosis, run by Celularity Incorporated at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 1 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Study Terminated by Sponsor). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluate pulmonary artery pressure during infusion (time frame: Day 1). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01440192.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Mar 2018.

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