Allogeneic Human Cells (hMSC) Via Intravenous Delivery in Patients With Mild Asthma

NCT03137199 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: 20160350
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 1 investigation will be performed to test the safety of two doses of bone marrow-derived MSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion.

Conditions

Asthma

Keywords

stem cell; mesenchymal stem cell; lung; asthma

Outcome Measures

Primary Outcomes (1)

• Number of Participant with treatment emergent serious adverse events

  as defined as the incidence of any treatment-emergent serious adverse events; these are a composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities

  Week 4 post infusion

Secondary Outcomes (4)

• Difference in lung function

  Participants will be followed from 1 week to an expected average of 48 weeks following infusion

• Decrease in peripheral eosinophilia

  Participants will be followed from 1 week to an expected average of 48 weeks following infusion

• Difference in subject reported dyspnea and quality of life assessments

  Participants will be followed from 1 week to an expected average of 48 weeks following infusion

• Death from any cause

  Participants will be followed from 1 week to an expected average of 48 weeks following infusion

Study Arms (2)

Group receiving 20 million hMSCs

EXPERIMENTAL

3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion

Biological: hMSCs

Group receiving 100 million hMSCs

EXPERIMENTAL

3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion

Biological: hMSCs

Interventions

hMSCs BIOLOGICAL

intravenous infusion of bone marrow-derived allogeneic stem cells

Also known as: allogeneic mesenchymal stem cell

Group receiving 100 million hMSCs; Group receiving 20 million hMSCs

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide written informed consent
• be between 18 and 65 years at the time of signing the Informed Consent
• have a clinical diagnosis of asthma prior to screening in accordance with the guidelines of the American Thoracic Society/European Respiratory Society
• ACQ over 1.25
• have a smoking history of less than 10 pack-years total and have not been smoking for at least the last 12 months
• Perform a positive methacholine challenge at screening and repeat positive methacholine challenge at baseline visit (14 days later)
• Have normal or mild obstructive spirometry
• Have normal right heart function as documented by Doppler echo or right heart catheterization
• If female, be surgically sterile, post-menopausal (more than 1 year), or practice double barrier methods of birth control
• Subjects may receive non-drug therapies including oxygen supplementation no greater than 2L/minute, and pulmonary rehabilitation
• Subjects may be on standard of care asthma medications including inhaled corticosteroids-long acting beta agonist at a dose not greater than 1 mg of a fluticasone equivalent

You may not qualify if:

• Have any active infection that is not treated
• Be unable to perform any of the assessments required for endpoint analysis.
• currently receive (or have received within four weeks of screening) experimental agents for the treatment of asthma
• be actively listed (or expecting to be listed in the near future) for transplant of any organ
• Have clinically important abnormal screening laboratory values : blood screening tests (Hematology, Chemistry, CBC including Eosinophil count) results that are not within normal limits (according to UMHC Laboratory Reference Ranges) Have a 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
• 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
• Have a non-pulmonary condition that limits lifespan to less than a year.
• Have a history of drug or alcohol abuse within the past 24 months.
• Be serum positive for HIV, hepatitis BsAg or Viremia hepatitis C
• Be currently participating (or have participated within the previous 30 days) in an investigational therapeutic or device trial.
• Have hypersensitivity to dimethyl sulfoxide (DMSO)
• Have a resting oxygen saturation (SpO2) on room air of more than 93% at sea level or more than 88% at an altitude above 5,000 feet above sea level (1524 meters)

Sponsors & Collaborators

• Marilyn Glassberg: lead
• The Marcus Foundation: collaborator

Study Sites (1)

University of Miami Miller School of Medicine

Miami, Florida, 33125, United States

Location

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Related Links

• Global Initiative for Asthma 2015 from the Global Strategy for Asthma Management and Prevention

MeSH Terms

Conditions

Asthma

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Officials

• Marilyn K Glassberg, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine, Surgery, and Pediatrics

Model Details: a first group of 3 patients will receive a lower dose of 20 million hMSCs, followed by a second group of 3 patients to receive a dose of 100 million hMSCs

Study Record Dates

• First Submitted: April 24, 2017
• First Posted: May 2, 2017
• Study Start: June 22, 2017
• Primary Completion: March 3, 2020
• Study Completion: April 29, 2020
• Last Updated: May 21, 2020

Record last verified: 2020-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)