Face transplantation using bone marrow cell therapy and reduced immunosuppression

Human Craniomaxillofacial Allotransplantation

This study is testing a new way to help people with serious facial injuries or deformities by using a special treatment with bone marrow cells and less medication to see if it can improve their appearance and function.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Johns Hopkins University (Academic / other)
• Locations: 1 site (Baltimore, Maryland)
• Trial ID: NCT01889381 on ClinicalTrials.gov

What this trial studies

This Phase II clinical trial aims to evaluate the safety and efficacy of craniomaxillofacial allotransplantation, focusing on patients with significant facial injuries or deformities. The study will utilize a novel approach combining bone marrow cell-based therapy with a one-drug immunosuppression regimen to minimize the adverse effects typically associated with long-term immunosuppression. Participants will undergo a thorough screening process to ensure they meet specific eligibility criteria, including a strong desire for transplantation and the absence of significant co-existing medical conditions. The goal is to improve both the functional and aesthetic outcomes for individuals suffering from severe facial deformities.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18-65 with recent or remote craniomaxillofacial injuries who are non-smokers and have no significant co-existing medical or psycho-social issues.

Not a fit: Patients with active malignancies, significant co-existing medical conditions, or those who are smokers may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly enhance the quality of life for patients with severe facial injuries by providing a more natural and functional appearance.

How similar studies have performed: Other studies have shown promising results with face transplantation, but this specific approach combining bone marrow therapy and reduced immunosuppression is relatively novel.

Eligibility criteria

Inclusion Criteria:

* Recent (≥6 months) or remote (i.e., several decades) craniomaxillofacial injury
* Male or female and of any race, color, or ethnicity.
* Aged 18-65 years.
* Strong desire to undergo craniomaxillofacial transplantation.
* Completes the protocol informed consent form.
* Non-smoker, defined by having never smoked or having quit \>6 consecutive months prior to screening.
* No co-existing medical condition which, in the opinion of the study team, could affect the immunomodulatory protocol, surgical procedure, or functional results (see Exclusion Criteria below. If the condition is amenable to treatment, the study team must agree that said condition should not significantly enhance the surgical risks of full or partial craniomaxillofacial transplantation.)
* No co-existing psycho-social problems (i.e., alcoholism, drug abuse).
* Negative for malignancy for past 5 years.
* Negative for HIV at transplant.
* Negative crossmatch with donor.
* If female of child-bearing potential, negative serum pregnancy test.
* If female of child-bearing potential, consent to use reliable contraception for at least one year following transplantation.
* Consents to cell collection, storage, and bone marrow infusion as part of the treatment regime.
* USA citizen or equivalent.
* Patient agrees to comply with the protocol and states a dedication to the immunomodulatory treatment regime.

Exclusion Criteria:

* Positive for any of the following conditions:

  * Untreated sepsis.
  * HIV (active or seropositive).
  * Active tuberculosis.
  * Active Hepatitis B infection.
  * Hepatitis C.
  * Viral encephalitis.
  * Toxoplasmosis.
  * Malignancy (within past 5 years).
  * Current/recent (within 3 months of donation/screening consent) IV drug abuse.
  * Paralysis of ischemic, traumatic, or congenital origin.
  * Infectious, post infectious, or inflammatory (axonal or demyelinating) neuropathy.
  * Toxic neuropathy (i.e. heavy metal poisoning, drug toxicity, industrial agent exposure).
  * Mixed connective tissue disease.
* Conditions that, in the opinion of the study team, may impact the immunomodulatory protocol potentially exposing the recipient to an unacceptable risk under immunosuppressive treatment.
* A history of medical non-compliance.
* Sensitized recipients with high levels (50%) of panel-reactive Human Leukocyte Antigen (HLA) antibodies.
* Conditions that may impact the success of the surgical procedure or increase the risk of postoperative complications including inherited coagulopathies like Hemophilia, Von-Willebrand's disease, Protein C and S deficiency, Thrombocythemias, Thalassemias, Sickle Cell disease, etc.
* Mixed connective tissue diseases and collagen diseases can result in poor wound healing after surgery.
* Conditions that may impact functional outcomes including Lipopolysaccharidosis and amyloidosis (may impact nerve regeneration) or rare disorders of bone healing like osteopetrosis.
* Subjects with inadequate donor sites for autologous reconstruction in the event of post-transplant flap failure.
* Patients considered unsuitable per the consulted Psychiatric/ Psychologic appraisal.

Where this trial is running

Baltimore, Maryland

• Johns Hopkins University School of Medicine — Baltimore, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Damon Cooney, MD, PHD — Johns Hopkins University
• Study coordinator: Jane Littleton, CRNP, MSN

Conditions Facial Injuries; Traumatic Wounds and Injuries; Craniofacial Injuries; Craniofacial Defects; Facial Transplantation; Facial Deformity; Face Transplant