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.
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
Biological: ExoFlo
Intravenous administration of bone marrow mesenchymal stem cell derived extracellular vesicles
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.
Direct Biologics Investigational Site
Palo Alto, California, United States
Direct Biologics Investigational Site
San Francisco, California, United States
Bill Arana
1-800-791-1021
clinicalaffairs@directbiologics.com
Bill Arana, Study Director
Direct Biologics, LLC