Urologic InjuriesPhase 1NCT03240822
Human Penile Tissue Allotransplantation for Devastating Penile and Concomitant Genital Trauma
A Phase 1 study testing Penile Transplant, Monoclonal Antibody (Humanized Anti-CD52) and Tacrolimus in people with Urologic Injuries and Genital Diseases, Male, run by Wake Forest University Health Sciences.
Trial summary
This is a 4-year, non-randomized, single center, patient self-controlled, clinical trial (though enrolled subjects will be followed for life as are all transplant patients) for patients seeking allotransplantation of the male external genitalia (MEG), or penile tissue, as a feasible reconstructive strategy for the… Read more about this study
- Who is it for?
- Ages 18 to 35 years, men only
- Conditions the study is for
- Urologic Injuries, Genital Diseases, Male, Amputation
- What is being tested?
- Penile Transplant, Monoclonal Antibody (Humanized Anti-CD52) and Tacrolimus
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jan 2017to Dec 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 35 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Male
- 18-40 years
- Irreversible and devastating genitalia damage not amenable to conventional reconstruction.
- Patent main vessels leading into the remaining penile stump, as confirmed by angiography.
- Adequate penile stump (> 2.5cm) to facilitate placement of a tourniquet, debridement of the distal portion and sufficient distal mobilization of the structures requiring anastomosis
- Eligible for long term standard of care coverage
Who cannot join
- Documented history of:
- cancer
- renal impairment
- hepatic disorders
- neurologic disorders (sensory or motor function deficits)
- severe scarring with poor host tissue bed
Show all 40Show fewer
- penile resection or implant surgery
- diabetes
- hypertension
- hyperlipidemia
- coronary artery disease
- untreated genital cancer
- HIV, Hepatitis B or C, or any infectious disease
- erectile dysfunction
- Peyronie's disease
- urethral stricture disease
- balanitis
- xerotica obliterans
- pelvic embolization
- pelvic radiation
- untreated hypogonadism
- prior prostate surgery
- recurrent urinary tract infections (UTIs)
- nephrolithiasis
- connective tissue disease or collagen disease
- lipopolysaccharidosis or amyloidosis
- Use of 5-alpha-reductase inhibitors
- External signs, sequelae or positive serology of sexually transmitted disease (including HPV)
- Active UTI, stones, urethral edema and other pathology that prevents urethral anastomosis
- Current or past substance abuse
- Current or past smoker (within past 3 months)
- Use of any medications known to cause vasoconstriction
- Psychiatric illness or psychological problems, or deemed unsuitable on psychiatric evaluation
- Any condition that may prevent transplantation (positive cross match, high panel reactive antibody (PRA), etc.)
- Uncontrolled bleeding disorder, platelet count > 50,000, hemophilia or any other inherited coagulopathy or need to routinely receive blood products for bleeding disorders
- Concurrent participation in any other clinical investigation during the period of this investigation
- Inability to undergo leukapheresis
- Inability to participate in all necessary study activities due to physical or mental limitations
- Inability or unwillingness to return for all required follow-up visits.
- Inability or unwillingness to sign the patient informed consent document.
About this study
Official title: Vascularized Composite Allotransplantation (VCA) for Devastating Penile and Concomitant Genital Trauma
This is a 4-year, non-randomized, single center, patient self-controlled, clinical trial (though enrolled subjects will be followed for life as are all transplant patients) for patients seeking allotransplantation of the male external genitalia (MEG), or penile tissue, as a feasible reconstructive strategy for the treatment of devastating and irreversible injuries to the genitalia. In addition to receiving penile allotransplantation and post-operative monitoring and support, enrolled patients will receive an innovative and clinically proven immunomodulatory protocol that combines lymphocyte depletion of the recipient with donor bone marrow cell infusion. Patients will be treated with lymphocyte depleting induction therapy, donor bone marrow cell infusion and tacrolimus. After the first year, maintenance immunosuppression will be modified gradually and cautiously (tapered dose reduction or spaced frequency dosing of tacrolimus) in selected patients based on a critical evaluation of clinical and immunologic outcomes. Outcomes will include but not be limited to functional metrics (sensation, erection, voiding), psychosocial (body integrity, adaptation to transplant) and health related quality of life (HRQOL) measures.
Read the detailed description
Initial treatment with an antibody targeting recipient immune cells followed by triple-drug maintenance therapy represents the current standard in clinical VCA. Our protocol is different from the existing conventional drug treatments used in MEG allotransplantation in that it combines donor bone marrow infusion with an immunosuppression sparing protocol. The primary goal of this pilot clinical trial is to determine the feasibility of using allogeneic penile tissue to repair/replace irreversibly damaged external genitalia. Enrolled and transplanted subjects will be followed for their lifetime as a transplant patient.
- Conditions
- Urologic Injuries, Genital Diseases, Male, Amputation
- Being tested
- Penile Transplant, Monoclonal Antibody (Humanized Anti-CD52), Tacrolimus
- Keywords
- Penile Trauma, Allotransplantation, Vascularized Composite Allotransplantation, VCA, Genital Trauma, Penile Transplantation, Amputation, Penile Injury
- Medical subject headings
- Genital Diseases, Male
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Penile TransplantProcedure or surgery
Penile Allotransplantation
Monoclonal Antibody (Humanized Anti-CD52)Biological
TacrolimusDrug
| Study group | Role | What they receive |
|---|---|---|
| Penile Allotransplant and Immunosuppression Treatment | Experimental | Penile transplantation with monoclonal antibody induction therapy of humanized anti CD52 followed by donor bone marrow infusion and tacrolimus monotherapy. Receives: Procedure: Penile Transplant; Biological: Monoclonal Antibody (Humanized Anti-CD52); Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Need for Immunosuppressive drug therapy
The amount of immunosuppressive drug therapy will be recorded.
Measured: 6 months
Secondary outcomes (6)
erectile function
This will be assessed following intracorporal injection of vaso-active agents, such as prostaglandin, phentolamine, papaverine, or some mixture of these drugs (i.e. Trimix). and through high-resolution duplex Doppler ultrasonography
Measured: 6 months
Assessment of sensory return and sensory thresholds
This will be accomplished by penile biothesiometry
Measured: 6 months
Determination of hemodynamic status
Dynamic infusion pharmaco-cavernosometry and cavernosography will be utilized to better understand these changes
Measured: 6 months
The Reintegration to Normal Living (RNL) Index
The RNL is an 11-item questionnaire that asks how a person manages activities, roles, and relationships on a day-to-day basis.
Measured: 6 months
The Situational Inventory of Body-Image Dysphoria (SIBID)
The SIBID is a multidimensional body-image survey assessment of people's negative body-image emotions in everyday situations and physical experiences.
Measured: 6 months
The Function and Body Image Survey
This survey consists of six open-ended questions regarding personal thoughts, opinions, and experiences about the transplant, and the individual's current functional abilities.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2017
- Study startJan 2017
- Primary completionDec 2022 (estimated)
- Study completionDec 2025 (estimated)
Why it stopped: Lack of Funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urologic InjuriesStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- All subjects receive transplant and immunosuppression therapy
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anthony Atala, MD, Principal investigator, Professor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03240822
- Sponsor's protocol ID
- IRB00027539
- Status last verified
- Jul 2020
- First submitted
- 2 Aug 2017
- Last updated
- 14 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vascularized Composite Allotransplantation (VCA) for Devastating Penile and Concomitant Genital Trauma
- ClinicalTrials.gov ID
- NCT03240822
- Acronym / sponsor ID
- IRB00027539
- Conditions
- Urologic Injuries, Genital Diseases, Male, Amputation
- Keywords
- Penile Trauma, Allotransplantation, Vascularized Composite Allotransplantation, VCA, Genital Trauma, Penile Transplantation, Amputation, Penile Injury
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of Funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Study start
- Jan 2017 (actual)
- Primary completion
- Dec 2022 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 7 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 4-year, non-randomized, single center, patient self-controlled, clinical trial (though enrolled subjects will be followed for life as are all transplant patients) for patients seeking allotransplantation of the male external genitalia (MEG), or penile tissue, as a feasible reconstructive strategy for the treatment of devastating and irreversible injuries to the genitalia. In addition to receiving penile allotransplantation and post-operative monitoring and support, enrolled patients will receive an innovative and clinically proven immunomodulatory protocol that combines lymphocyte depletion of the recipient with donor bone marrow cell infusion. Patients will be treated with lymphocyte depleting induction therapy, donor bone marrow cell infusion and tacrolimus. After the first year, maintenance immunosuppression will be modified gradually and cautiously (tapered dose reduction or spaced frequency dosing of tacrolimus) in selected patients based on a critical evaluation of clinical and immunologic outcomes. Outcomes will include but not be limited to functional metrics (sensation, erection, voiding), psychosocial (body integrity, adaptation to transplant) and health related quality of life (HRQOL) measures.
Detailed description
Initial treatment with an antibody targeting recipient immune cells followed by triple-drug maintenance therapy represents the current standard in clinical VCA. Our protocol is different from the existing conventional drug treatments used in MEG allotransplantation in that it combines donor bone marrow infusion with an immunosuppression sparing protocol. The primary goal of this pilot clinical trial is to determine the feasibility of using allogeneic penile tissue to repair/replace irreversibly damaged external genitalia. Enrolled and transplanted subjects will be followed for their lifetime as a transplant patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All subjects receive transplant and immunosuppression therapy
| Arm | Type | Receives |
|---|---|---|
| Penile Allotransplant and Immunosuppression Treatment | Experimental | Procedure: Penile Transplant, Biological: Monoclonal Antibody (Humanized Anti-CD52), Drug: TacrolimusPenile transplantation with monoclonal antibody induction therapy of humanized anti CD52 followed by donor bone marrow infusion and tacrolimus monotherapy. |
Who may qualify
- Ages
- 18 to 35 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Male
- 18-40 years
- Irreversible and devastating genitalia damage not amenable to conventional reconstruction.
- Patent main vessels leading into the remaining penile stump, as confirmed by angiography.
- Adequate penile stump (> 2.5cm) to facilitate placement of a tourniquet, debridement of the distal portion and sufficient distal mobilization of the structures requiring anastomosis
Key exclusion criteria (40 listed)
- Documented history of:
- Use of 5-alpha-reductase inhibitors
- External signs, sequelae or positive serology of sexually transmitted disease (including HPV)
- Active UTI, stones, urethral edema and other pathology that prevents urethral anastomosis
- Current or past substance abuse
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Penile TransplantPenile Allotransplantation | Procedure or surgery | Penile Allotransplant and Immunosuppression Treatment |
| Monoclonal Antibody (Humanized Anti-CD52) | Biological | Penile Allotransplant and Immunosuppression Treatment |
| Tacrolimus | Drug | Penile Allotransplant and Immunosuppression Treatment |
Primary outcome
Need for Immunosuppressive drug therapy
Time frame: 6 months
The amount of immunosuppressive drug therapy will be recorded.
6 secondary outcomes
erectile function
Time frame: 6 months
Assessment of sensory return and sensory thresholds
Time frame: 6 months
Determination of hemodynamic status
Time frame: 6 months
The Reintegration to Normal Living (RNL) Index
Time frame: 6 months
The Situational Inventory of Body-Image Dysphoria (SIBID)
Time frame: 6 months
The Function and Body Image Survey
Time frame: 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anthony Atala, MD principal investigator
Professor
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Penile Transplant, Monoclonal Antibody (Humanized Anti-CD52) and Tacrolimus in people with Urologic Injuries and Genital Diseases, Male, run by Wake Forest University Health Sciences.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 40 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 14 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Need for Immunosuppressive drug therapy (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2021.