Semilunar Heart Valve ReplacementNCT05372757
Partial Heart Transplantation for Severe Pediatric Semilunar Valve Dysfunction
A study testing Partial Heart Transplantation in people with Semilunar Heart Valve Replacement, run by Medical University of South Carolina at 1 site in United States.
Trial summary
The long-term goal of this research initiative is to develop a new valve replacement option for neonates, infants and young children. The central hypothesis is that transplantation of a freshly isolated heart valve will be associated with superior outcomes compared to currently available options, including preserved… Read more about this study
- Who is it for?
- Ages 1 day to 2 years
- Conditions the study is for
- Semilunar Heart Valve Replacement
- What is being tested?
- Partial Heart Transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 3 May 2023to 30 Nov 2023
- Phase
- Interventional
- 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 1 day to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children less than 2 years of age who are referred for a cardiac operation that involves a primary semilunar valve replacement or children less than 2 years of age who are referred for a cardiac operation involves an initial replacement of a previously placed prior homograft, bioprosthetic, or mechanical valve in the aortic or pulmonary position.
- Deemed acceptable for partial heart transplantation based on the standard evaluation process used for orthotopic heart transplantation (see Appendix 1) Insurance approval.
- Written informed consent of both parents/guardians; if there is only one parent/guardian, consent from that individual will be adequate.
Who cannot join
- Absolute contraindications for orthotopic heart transplantation.
- Severe bilateral long segment pulmonary arterial hypoplasia
- Bilateral pulmonary vein stenosis
- <34 weeks corrected gestational age
- Persistent acidosis with a pH < 7.1
- Diagnosis of immune deficiency.
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- Inability for the parent to understand English or Spanish.
- Failure to pass the following psychosocial evaluation:
- The candidate should reside within 4 hours traveling time from Medical University of South Carolina for a minimum of four to six months post-transplantation to assure careful follow-up
- The candidate's family should be capable of long-term supportive care of the child and be able to support the medical needs of the child in follow-up
- Parental (custodial) alcohol and/or substance abuse
- Documented parental (custodial) child abuse or neglect
- Parent (custodian) with cognitive/psychiatric impairment severe enough to limit comprehension of medical regimen
- Infectious Disease Exclusion Criteria
- Evidence of sepsis
- Hepatitis B surface antigenemia
- HIV positivity
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical University of South Carolina
Charleston, South Carolina, 29401
About this study
The long-term goal of this research initiative is to develop a new valve replacement option for neonates, infants and young children. The central hypothesis is that transplantation of a freshly isolated heart valve will be associated with superior outcomes compared to currently available options, including preserved cadaver valves, bioprosthetic tissue valves, or mechanical valves. This new operation has been named "partial heart transplantation".
The proposed study is a single-center, nonrandomized single arm pilot trial of "partial heart transplantation" in neonates, infants and young children who require semilunar heart valve replacement. This "first in man" trial seeks to determine whether valve replacement using partial heart transplant is feasible and safe. Primary aims are survival one year and five years following the procedure. The hypothesis is that, when compared to historical controls who have undergone homograft valve replacement, those undergoing partial heart transplantation will have equal or superior survival one year and five years following the procedure. Secondary aims are to assess growth and function of the transplanted valve. The hypothesis is that when compared to historical controls who have undergone conventional valve replacement, those undergoing partial heart transplantation will have valve growth that corresponds with somatic growth and superior valve function 1 year following the procedure. Up to five patients will be enrolled in this trial over three years.
- Conditions
- Semilunar Heart Valve Replacement
- Being tested
- Partial Heart Transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Partial Heart TransplantationProcedure or surgery
The donor heart will be recovered and the new heart valve will be removed from this heart by the clinical team. The child will be scheduled for an emergent operation once the donor heart valve is deemed acceptable. The child will have general anesthesia during surgery. This is a state of unconsciousness, which is carefully controlled by the anesthesiologist with a mixture of very potent drugs, to prevent or lessen pain. The child will have a partial heart transplant using the donated heart valve to replace the dysfunctional heart valve. Expected hospital stay will range from weeks to months. The medical care after surgery is performed by the child's clinical team. This type of re-operation could similarly be required after a standard heart valve replacement.
| Study group | Role | What they receive |
|---|---|---|
| Partial Heart Transplantation Arm | Experimental | Not described Receives: Procedure: Partial Heart Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The ability to perform the new operation in at least 1 patient per year for 3 years (averaged) after enrollment opens.
This is how feasibility will be measured. This will be answered as "yes" or "no".
Measured: Duration of the study (3 years after enrollment opens).
Number of participants who achieve survival.
Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled.
Measured: 6 months after all 5 subjects have been enrolled.
Number of participants who achieve survival.
Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled.
Measured: 1 year after all 5 subjects have been enrolled.
Secondary outcomes (3)
Valve annulus growth.
The valve annulus diameter will be measured using echocardiography in 2 planes at baseline (i.e., during postoperative hospitalization 5-10 days after surgery) and every 6 months following the operation. It is expected that the valve annulus diameter Z score will be relatively unchanged over time, which would reflect an absolute increase in valve annulus growth corresponding with an increase in body surface area.
Measured: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
The level of valve stenosis.
Mean valve stenosis will be assessed by transthoracic echocardiogram using pulse Doppler every 6 months following the operation.
Measured: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
The level of valve regurgitation.
Mean valve regurgitation will be assessed with color Doppler echocardiography.
Measured: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2022
- Study start3 May 2023
- Primary completion30 Nov 2023
- Study completion30 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Semilunar Heart Valve ReplacementStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Taufiek Rajab, MD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05372757
- Sponsor's protocol ID
- 00114653
- Status last verified
- Sep 2024
- First submitted
- 9 May 2022
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Rajab TK, Ochoa B, Zilinskas K, Kwon J, Taylor CL, Henderson HT, Savage AJ, Kavarana M, Turek JW, Costello JM. Partial heart transplantation for pediatric heart valve dysfunction: A clinical trial protocol. PLoS One. 2023 Feb 7;18(2):e0280163. doi: 10.1371/journal.pone.0280163. eCollection 2023.
PubMed 36749770
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Partial Heart Transplantation for Severe Pediatric Semilunar Valve Dysfunction
- ClinicalTrials.gov ID
- NCT05372757
- Acronym / sponsor ID
- 00114653
- Conditions
- Semilunar Heart Valve Replacement
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Study start
- 3 May 2023 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 13 May 2022 (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.
The long-term goal of this research initiative is to develop a new valve replacement option for neonates, infants and young children. The central hypothesis is that transplantation of a freshly isolated heart valve will be associated with superior outcomes compared to currently available options, including preserved cadaver valves, bioprosthetic tissue valves, or mechanical valves. This new operation has been named "partial heart transplantation".
The proposed study is a single-center, nonrandomized single arm pilot trial of "partial heart transplantation" in neonates, infants and young children who require semilunar heart valve replacement. This "first in man" trial seeks to determine whether valve replacement using partial heart transplant is feasible and safe. Primary aims are survival one year and five years following the procedure. The hypothesis is that, when compared to historical controls who have undergone homograft valve replacement, those undergoing partial heart transplantation will have equal or superior survival one year and five years following the procedure. Secondary aims are to assess growth and function of the transplanted valve. The hypothesis is that when compared to historical controls who have undergone conventional valve replacement, those undergoing partial heart transplantation will have valve growth that corresponds with somatic growth and superior valve function 1 year following the procedure. Up to five patients will be enrolled in this trial over three years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Partial Heart Transplantation Arm | Experimental | Procedure: Partial Heart Transplantation |
Who may qualify
- Ages
- 1 day to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children less than 2 years of age who are referred for a cardiac operation that involves a primary semilunar valve replacement or children less than 2 years of age who are referred for a cardiac operation involves an initial replacement of a previously placed prior homograft, bioprosthetic, or mechanical valve in the aortic or pulmonary position.
- Deemed acceptable for partial heart transplantation based on the standard evaluation process used for orthotopic heart transplantation (see Appendix 1) Insurance approval.
- Written informed consent of both parents/guardians; if there is only one parent/guardian, consent from that individual will be adequate.
Key exclusion criteria (17 listed)
- Absolute contraindications for orthotopic heart transplantation.
- Severe bilateral long segment pulmonary arterial hypoplasia
- Bilateral pulmonary vein stenosis
- <34 weeks corrected gestational age
- Persistent acidosis with a pH < 7.1
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 |
|---|---|---|
| Partial Heart TransplantationThe donor heart will be recovered and the new heart valve will be removed from this heart by the clinical team. The child will be scheduled for an emergent operation once the donor heart valve is deemed acceptable. The child will have general anesthesia during surgery. This is a state of unconsciousness, which is carefully controlled by the anesthesiologist with a mixture of very potent drugs, to prevent or lessen pain. The child will have a partial heart transplant using the donated heart valve to replace the dysfunctional heart valve. Expected hospital stay will range from weeks to months. The medical care after surgery is performed by the child's clinical team. This type of re-operation could similarly be required after a standard heart valve replacement. | Procedure or surgery | Partial Heart Transplantation Arm |
Primary outcome
The ability to perform the new operation in at least 1 patient per year for 3 years (averaged) after enrollment opens.
Time frame: Duration of the study (3 years after enrollment opens).
This is how feasibility will be measured. This will be answered as "yes" or "no".
Number of participants who achieve survival.
Time frame: 6 months after all 5 subjects have been enrolled.
Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled.
Number of participants who achieve survival.
Time frame: 1 year after all 5 subjects have been enrolled.
Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled.
3 secondary outcomes
Valve annulus growth.
Time frame: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
The level of valve stenosis.
Time frame: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
The level of valve regurgitation.
Time frame: Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Derived from this study: PubMed 36749770Rajab TK, Ochoa B, Zilinskas K, Kwon J, Taylor CL, Henderson HT, Savage AJ, Kavarana M, Turek JW, Costello JM. Partial heart transplantation for pediatric heart valve dysfunction: A clinical trial protocol. PLoS One. 2023 Feb 7;18(2):e0280163. doi: 10.1371/journal.pone.0280163. eCollection 2023.
- Individual participant dataSharing planned: Yes
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.
Taufiek Rajab, MD principal investigator
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Partial Heart Transplantation in people with Semilunar Heart Valve Replacement, run by Medical University of South Carolina at 1 site in United States.
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 3 inclusion and 17 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: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The ability to perform the new operation in at least 1 patient per year for 3 years (averaged) after enrollment opens. (time frame: Duration of the study (3 years after enrollment opens).). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05372757, which also lists 1 linked publication.
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 19 Sep 2024.