Expanded Access for Use of ExoFlo in Abdominal Solid Organ Transplant Patients

Intermediate Size Expanded Access for the Use of ExoFlo in the Treatment of Abdominal Solid Organ Transplant Patients Who Are at Risk of Worsening Allograft Function With Conventional Immunosuppressive Therapy Alone

This Expanded Access Protocol will provide access to the IMP ExoFlo for patients who have severe or life-threatening abdominal solid organ transplant rejection or who are evaluated and determined to be at high risk of progression to severe or life-threatening condition related to rejection of an abdominal solid organ transplant, at risk of worsening allograft function, or at risk of complications from current immunosuppressive therapeutic regimens.

Study Overview

• Status: No longer available
• Conditions: Solid Organ Transplant Rejection; Organ Rejection Transplants; Organ Rejection
• Intervention / Treatment: Biological: ExoFlo

Detailed Description

Objectives:

The primary objective of this expanded access program is to evaluate safety and tolerability of ExoFlo in patients with graft inflammation, or worsening graft function, or evidence of rejection of an abdominal solid organ transplant despite conventional therapies. The secondary objective is to characterize the improvement seen in serum inflammatory markers and inflammation of solid abdominal organ.

Endpoints:

Primary Endpoint:

Incidence of AEs or SAEs.

Secondary Endpoint:

Improvement seen in serum inflammatory markers and inflammation of solid abdominal organ. This could include improvements seen in the following transplant types:

• Liver transplant: overall improved liver function, as seen by reduction in liver function test and improved albumin.
• Small bowel transplant: decreased inflammation on biopsy
• Pancreas: decreased amylase and lipase
• Multivisceral: any of the above criteria

Number of subjects: 20

Phase: Expanded Access Protocol

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Direct Biologics Investigational Site
    • San Francisco, California, United States, 94143
      • Direct Biologics Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Provision of signed and dated informed consent form.
2. Stated willingness to comply with all study procedures and availability for the duration of the study.
3. Male or female, ages 18 to 75.
4. Previous abdominal solid organ transplant, including intestinal transplant.
5. Diagnosis of acute or chronic rejection / inflammation based on clinical observations, laboratory analysis, histological evaluation.
6. Diagnosis of worsening allograft function based on clinical observations, laboratory analysis, histological evaluation.
7. Failed primary and alternate standard of care therapies.
8. Serious or life-threatening condition or progressing to serious or life-threatening condition if not treated as evaluated by the treating physician.
9. In otherwise good general health as evidenced by medical history.
10. Male or female of reproductive potential must utilize a medically effective birth control method during from time of enrollment to 6 months after last dose of IMP.

Exclusion Criteria:

1. Pregnant or breastfeeding.
2. Treatment with another investigational drug or other intervention within 30 days of enrollment.

Study Plan

How is the study designed?

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: No SAE attributable to IP in clinical investigational use to date.; 1 AE, grade 2 skin hyperpigmentation at the infusion site, was reported; this was in DB-EF-EXPANDEDACCESS-001. Clinical investigational use includes the following: Pilot study for COVID-19 IND #21669, DB-EF-PhaseII-001 (met target enrollment of N=121), and DB-EF-EXPANDEDACCESS-001 (enrolled 75 subjects; target enrollment = 100) as well as 13 single patient Emergency INDs for COVID-19 associated ARDS.

Biological: Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles Infusion Treatment; Intravenous Infusion over 60 minutes; Other Names: ExoFlo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse/serious adverse events	20 patients who meet the criteria for Expanded Access as determined by the principal investigator and who do not otherwise qualify for an available clinical trial	70 weeks

Collaborators and Investigators

• Sponsor: Direct Biologics, LLC
• Investigators: Study Director: Bill Arana, Direct Biologics, LLC

Publications and helpful links

Helpful Links

• Direct Biologics, LLC

Study record dates

Study Major Dates

• Study Start: February 1, 2023
• Primary Completion: February 1, 2023
• Study Completion: February 1, 2023

Study Registration Dates

• First Submitted: January 18, 2022
• First Submitted That Met QC Criteria: January 18, 2022
• First Posted (Actual): January 31, 2022

Study Record Updates

• Last Update Posted (Actual): July 29, 2024
• Last Update Submitted That Met QC Criteria: July 26, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: ExoFlo; Organ Rejection; Organ Rejection Transplants; Solid Organ Transplant Rejection
• Other Study ID Numbers: DB-EF-TP_ASO-1a

Drug and device information, study documents

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