Allogeneic Bone Marrow Mesenchymal Stem Cells for Patients With Interstitial Lung Disease (ILD) & Connective Tissue Disorders (CTD)

A Phase I Study to Evaluate the Safety of Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for Interstitial Lung Disease in Patients With Connective Tissue Disorders

Researchers are trying to find out more about the safety of a new treatment, Allogeneic (coming from a healthy donor) Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs) which is still experimental, for Interstitial Lung Disease (ILD) associated with Connective Tissue Disorder (CTD).

Study Overview

• Status: Completed
• Conditions: Connective Tissue Diseases; Interstitial Lung Disease
• Intervention / Treatment: Biological: Allogeneic Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs)

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic in Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Adults over 18 years of age and less than 80 years of age
• Both female and male
• Patients with new diagnosis of interstitial lung disease associated with connective tissue disorders, Antineutrophil cytoplasmic antibodies (ANCA) associated vasculitis or idiopathic pneumonia with autoimmune features (IPAF) or established diagnosis of ILD associated with CTD under conventional therapy for at least 6 months but less than 24 months, with no evidence of improvement.
• Competent and able to provide written informed consent, and ability to comply with protocol

Exclusion Criteria:

• Patients with interstitial lung disease without evidence of a concomitant rheumatologic autoimmune disorder
• Exposure to rituximab or cyclophosphamide on the previous 2 months
• Severe interstitial lung disease defined by the presence of severe hypoxemia at rest (SO2 < 88% at rest)
• Clinical assessment that indicates active chronic infections such as osteomyelitis or active tuberculosis (TB), or acute infections such as pneumonia, active bronchitis, cellulitis, etc. or active solid tumors or hematologic malignancies
• Previous treatment with mesenchymal stem cells
• Clinically significant medical conditions within the six months before administration of BMD-MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
• Medical history of human immunodeficiency virus (HIV), Hepatitis B or C
• Abnormal complete blood count (CBC), creatinine, Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) at screening
• Pregnant or breast feeding
• Unwilling to agree to use acceptable contraception methods during participation in the trial
• Inability to provide informed consent

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Interstitial Lung Disease with Connective Tissue Disorder; Subjects with Interstitial Lung Disease (ILD) associated with Connective Tissue Disorder (CTD) will receive a new treatment called Allogeneic (coming from a healthy donor) Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs)	Biological: Allogeneic Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs); Subjects will be treated with regular standard of care plus 0.5-1 million MSC/Kg intravenously.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Number of adverse events reported with infusion of each intravenous Mesenchymal Stem Cells (MSC) dose	6 months

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Andy Abril, MD, Mayo Clinic

Publications and helpful links

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start (Actual): November 5, 2019
• Primary Completion (Actual): July 27, 2021
• Study Completion (Actual): February 2, 2022

Study Registration Dates

• First Submitted: April 24, 2019
• First Submitted That Met QC Criteria: April 24, 2019
• First Posted (Actual): April 26, 2019

Study Record Updates

• Last Update Posted (Actual): April 7, 2023
• Last Update Submitted That Met QC Criteria: April 6, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Connective Tissue Diseases; Lung Diseases, Interstitial
• Other Study ID Numbers: 18-007216

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No