Allogeneic Human Cells (hMSC)in Patients With Idiopathic Pulmonary Fibrosis Via Intravenous Delivery (AETHER)

• Register: ClinicalTrials.gov
• Last refreshed on: 16 March 2021
• Main ID: NCT02013700
• Date of registration: 02/12/2013
• Prospective Registration: No
• Primary sponsor: Joshua M Hare
• Public title: Allogeneic Human Cells (hMSC)in Patients With Idiopathic Pulmonary Fibrosis Via Intravenous Delivery (AETHER) AETHER
• Scientific title: A Phase I, Randomized, Blinded and Placebo-controlled Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell Infusion in Patients With Idiopathic Pulmonary Fibrosis
• Date of first enrolment: November 13, 2013
• Target sample size: 9
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02013700
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Marilyn K. Glassberg, MD
• Affiliation: University of Miami

Key inclusion & exclusion criteria

Inclusion Criteria:

- Provide written informed consent.

- Subjects age equal to or greater than 40 and equal to or less than 90 years at the
time of signing the Informed Consent Form.

- Have a clinical diagnosis of Idiopathic Pulmonary Fibrosis (IPF) prior to screening

- Forced vital capacity (FVC) = 50% predicted and diffusing capacity (DLCO) =30%
(corrected for hemoglobin but not alveolar volume).

- RVSP equal to or less than 50 mmHg, as documented by Doppler echo or right heart
catheterization.

- Female subjects must be surgically sterile or post-menopausal (greater than 1 year).

Exclusion Criteria:

- HRCT and/or surgical lung biopsy results inconsistent with the diagnosis of IPF.

- Infiltrative lung disease of any type other than Idiopathic Pulmonary Fibrosis (IPF),
lungs disease related to fibrogenic agents, toxins, drugs or other exposures,
granulomatous lung disease, pulmonary vascular disease, or known connective tissue
disease.

- Inability to perform any of the assessments required for endpoint analysis (report
safety or tolerability concerns, perform pulmonary function tests or high resolution
CT (HRCT), undergo blood draws, read and respond to questionnaires.

- Currently receiving (or received within four weeks of screening) any medication,
treatment, or experimental agents for the treatment of Idiopathic Pulmonary Fibrosis
(IPF), except for patients receiving non drug therapies will include oxygen saturation
therapy (oxygen supplementation) and pulmonary rehabilitation.

- Active listing (or expected future listing) for transplant of any organ.

- Clinically important abnormal screening laboratory values, including but not limited
to: hemoglobin <8 g/dl, white blood cell count <3000/mm3, platelets <80,000/mm3, INR >
1.5, aspartate transaminase, alanine transaminase, or alkaline phosphatase > 3 times
upper limit of normal, total bilirubin > 1.5 mg/dl.

- Serious comorbid illness that, in the opinion of the investigator, may compromise the
safety or compliance of the patient or preclude successful completion of the study.
Including, but not limited to: HIV, advanced liver or renal failure, class III/IV
congestive heart failure, myocardial infarction, unstable angina, or cardiac
revascularization within the last six months, or severe obstructive ventilatory
defect.

- Any other condition that, in the opinion of the investigator, may compromise the
safety or compliance of the patient or preclude successful completion of the study.

- Have known allergies to penicillin or streptomycin.

- Be an organ transplant recipient.

- Have a clinical history of malignancy within 5 years (i.e., patients with prior
malignancy must be disease free for 5 years), except curatively- treated basal cell
carcinoma, squamous cell carcinoma, or cervical carcinoma.

- Have a non-pulmonary condition that limits lifespan to less than 1 year.

- Have a history of drug or alcohol abuse within the past 24 months.

- Be serum positive for Human immunodeficiency virus (HIV), hepatitis BsAg or Viremic
hepatitis C.

- Be currently participating (or participated within the previous 30 days) in an
investigational therapeutic or device trial.

- Be a female who is pregnant, nursing, or of childbearing potential while not
practicing effective contraceptive methods. Female patients must undergo a blood or
urine pregnancy test at screening and within 36 hours prior to injection.

- Female subjects must have a FSH less than 25.8 IU/L

- Subject with hypersensitivity to dimethyl sulfoxide (DMSO)

- Saturated oxygen (SpO2 of less than 93% (room air [sea level] at rest). SpO2 of less
than 88% (room air [>5,000 feet above sea level (1524 meters) at rest).



Age minimum: 40 Years
Age maximum: 90 Years
Gender: All

Health Condition(s) or Problem(s) studied

Idiopathic Pulmonary Fibrosis (IPF)

Intervention(s)

Biological: Allogeneic Adult Human Mesenchymal Stem Cells (hMSCs)

Biological: matched placebo

Primary Outcome(s)

To determine the safety and tolerability of intravenous allo hMSCs in patients with Idiopathic Pulmonary Fibrosis (IPF). [Time Frame: One month post infusion]

Secondary Outcome(s)

- To explore effects of allo hMSCs on lung function: forced vital capacity (FVC). [Time Frame: Participants will be followed from 12 weeks to an expected average of 60 weeks following infusion.]

Death from any cause. [Time Frame: 60 weeks.]

To explore effects of allogenic human mesenchymal stem cells on lung function: Diffusing Capacity (DLCO) [Time Frame: Participants will be followed from 12 weeks to an expected average of 60 weeks following infusion.]

To explore effects of allogenic human mesenchymal stem cells on symptom related quality of life. [Time Frame: Participants will be followed from 4 weeks to an expected average of 60 weeks following infusion.]

Difference in frequency of acute exacerbations of Idiopathic Pulmonary Fibrosis (IPF) [Time Frame: 4 weeks following infusion]

Secondary ID(s)

20120946

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Emmes Company, LLC

The Lester And Sue Smith Foundation