A Phase 1 interventional study of c-kit+ cells in Hypoplastic Left Heart Syndrome, sponsored by Joshua M Hare. Completed at 4 sites in United States. Open to participants aged 1 Day to 21 Days. Per ClinicalTrials.gov, last updated 2026-01-23.
Sponsored by Joshua M Hare · Phase 1, Interventional, and Treatment
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Among 44 studies in Hypoplastic Left Heart Syndrome with a site in United States — 6 open to participants now, 10 under way, 1 of them phase 3. Hypoplastic Left Heart Syndrome · United States.
Among the 44 studies in Hypoplastic Left Heart Syndrome with a site in United States, this one is the 17th largest by enrollment of the 26 interventional ones that give a count, is one of 3 that started in 2019, is one of 9 in phase 1, and was the 30th registered.
67 studies on the registry are indexed under Hypoplastic Left Heart Syndrome; 12 are open to participants now.
Browse Hypoplastic Left Heart Syndrome studies →6 of the 44 studies in Hypoplastic Left Heart Syndrome with a site in United States are open now. Recruiting first, the newest first:
HeartWorks, Inc. · Sites in United States
Ages 0+
Queen Silvia Children's Hospital, Gothenburg, Sweden · Sites in United States, Austria, Canada and 5 more countries
Ages 23 wk–31 wk
Nationwide Children's Hospital · Sites in United States
Children's Hospital Medical Center, Cincinnati · Sites in United States, Canada, United Kingdom
Ages up to 15 mo
Mauro H. Schenone · Sites in United States
Ages up to 45 · Women only · Healthy volunteers
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 9 Hypoplastic Left Heart Syndrome studies recruiting now, 3 set no upper age limit and 6 stop at 75 or younger; 9 take children under 18; none take healthy volunteers.
This study: 1 Day to 21 Days · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
44 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 12 · Phase 2 7 · Phase 3 2
First in 2001; this study’s year is marked. 3 started in 2019.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 25 is below the median of 30 across 41 interventional studies indexed under Hypoplastic Left Heart Syndrome.
Median enrollment 30 across the 26 interventional studies, 96 across the 18 observational. Of the 32 that completed or were terminated, 7 (22%) have results posted.
This tab as a report to print or save as PDF → With an account.
2 of 44 have an industry lead sponsor. The 10 most frequent:
Joshua M Hare is the lead sponsor of 15 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 7 (78%) have results posted.
Joshua M Hare has 1 study filed under Hypoplastic Left Heart Syndrome — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 44 studies in Hypoplastic Left Heart Syndrome with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
For inclusion in the study, subjects must meet all of the inclusion criteria:
Exclusion Criteria:
Candidates will be excluded from the study if any of the following conditions are met:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
Biological: c-kit+ cells
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
Biological: c-kit+ cells
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
Drug page: Cell Types
The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
Also known as: Autologous c-kit-positive cells (c-kit+ cells)
Number of Incidence of Treatment Related Major Adverse Cardiac Events
Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
Time frame: 30 days
Number of C-kit+ Products
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Time frame: Day 1
Number of Participants Completing Magnetic Resonance Imaging (MRI)
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Time frame: Baseline, 6 Months, 12 months
Change in Right Ventricular Function (RVEF)
Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.
Time frame: Baseline, 6 Months, 12 months
Change in Right Ventricular End-diastolic Volume (RVEDV)
Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Time frame: Baseline, 6 months, 12 months
Change in Right Ventricular End-systolic Volume (RVESV)
Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Time frame: Baseline, 6 months, 12 months
Change in Tricuspid Regurgitation
Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.
Time frame: Baseline, 6 months, 12 months
Number of Incidence of Serious Adverse Events
Incidence of the following after the BDCPA / GLENN Procedure including: All-cause mortality; Cardiovascular mortality; Need for transplantation; Hospitalization for heart failure; Cardiovascular morbidity, including stroke or heart failure or sustained/symptomatic arrhythmias.
Time frame: Up to 12 months
Change in Somatic Growth Velocity - Length (cm)
Changes in somatic growth velocity will be evaluated by length (cm) over 12 months post study product injection.
Time frame: Baseline, 6 months, 12 months
Change in Somatic Growth Velocity - Weight (kg)
Changes in somatic growth velocity will be evaluated by weight (kg) over 12 months post study product injection.
Time frame: Baseline, 6 months, 12 months
Change in Somatic Growth Velocity - Head Circumference (cm)
Changes in somatic growth velocity will be evaluated by head circumference (cm) over 12 months post study product injection.
Time frame: Baseline, 6 months, 12 months
Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health
The Infant Toddler Quality of Life Survey (ITQOL) is a validated 47-item parent-reported questionnaire assessing multiple domains of child health, including overall health, physical abilities, growth and development, discomfort/pain, mood, and behavior. Domain scores are transformed to a 0-100 scale, where 0 reflects the poorest health-related quality of life and 100 reflects the best. Higher scores indicate better quality of life.
Time frame: Baseline, 12 months
Incidence of Mortality or Need for Transplantation
Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported
Time frame: Up to 12 months
| Milestone | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Started | 9 | 8 | 8 |
| Completed | 9 | 8 | 8 |
| Not completed | 0 | 0 | 0 |
Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
| Events | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Number of Incidence of Treatment Related Major Adverse Cardiac Events | 1 | 0 | 6 |
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
| Products | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Number of C-kit+ Products | 9 | 7 | 0 |
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
| Participants | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 9 | 7 | 7 |
| Month 6 | 6 | 6 | 5 |
| Month 12 | 6 | 6 | 2 |
Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.
| percentage | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 49.33 ± 4.44 | 55 ± 7.19 | 45.4 ± 10.25 |
| Month 6 | 48 ± 3.63 | 50.83 ± 6.74 | 47 ± 6.86 |
| Month 12 | 44.67 ± 8.36 | 49.83 ± 3.76 | 48.5 ± 3.54 |
Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
| milliliters per square meter | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 112.57 ± 20.75 | 119.1 ± 35.08 | 123.98 ± 18.92 |
| Month 6 | 129.5 ± 44.21 | 123.08 ± 11.83 | 130.27 ± 37.08 |
| Month 12 | 126.78 ± 23.55 | 113.8 ± 13.97 | 129 ± 18.1 |
Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
| milliliters per square meter | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 57.43 ± 13.22 | 55.53 ± 23.87 | 68.66 ± 22.04 |
| Month 6 | 68.38 ± 26.43 | 55.17 ± 21.36 | 70.66 ± 28.95 |
| Month 12 | 71.7 ± 21.64 | 56.66 ± 5.43 | 68.05 ± 12.8 |
Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.
| percentage | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 8.28 ± 5.21 | 5 ± 0 | 5 ± 0 |
| Month 6 | 35.07 ± 27.81 | 9.23 ± 6.03 | 15.35 ± 16.95 |
| Month 12 | 21.93 ± 6.03 | 14.72 ± 7.27 | 7.75 ± 3.75 |
Incidence of the following after the BDCPA / GLENN Procedure including: All-cause mortality; Cardiovascular mortality; Need for transplantation; Hospitalization for heart failure; Cardiovascular morbidity, including stroke or heart failure or sustained/symptomatic arrhythmias.
| Events | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Number of Incidence of Serious Adverse Events | 1 | 0 | 6 |
Changes in somatic growth velocity will be evaluated by length (cm) over 12 months post study product injection.
| centimeters | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 60.33 ± 3.82 | 59.5 ± 3.28 | 60.07 ± 4.94 |
| Month 6 | 69.72 ± 5.57 | 69.79 ± 4.18 | 70.8 ± 5.39 |
| Month 12 | 76.98 ± 5.87 | 77.03 ± 3.7 | 79.63 ± 4.02 |
Changes in somatic growth velocity will be evaluated by weight (kg) over 12 months post study product injection.
| kilograms | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 5.7 ± 0.66 | 5.6 ± 0.74 | 5.65 ± 0.69 |
| Month 6 | 7.67 ± 0.84 | 8.39 ± 1.21 | 8.36 ± 0.65 |
| Month 12 | 9.74 ± 1.65 | 10.45 ± 1.19 | 10.01 ± 0.68 |
Changes in somatic growth velocity will be evaluated by head circumference (cm) over 12 months post study product injection.
| centimeters | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 38.51 ± 3.01 | 39.58 ± 2.69 | 38.33 ± 3.18 |
| Month 6 | 45.04 ± 1.33 | 45.18 ± 1.62 | 45.42 ± 2.42 |
| Month 12 | 47.88 ± 0.76 | 48.48 ± 1.57 | 46.92 ± 2.39 |
The Infant Toddler Quality of Life Survey (ITQOL) is a validated 47-item parent-reported questionnaire assessing multiple domains of child health, including overall health, physical abilities, growth and development, discomfort/pain, mood, and behavior. Domain scores are transformed to a 0-100 scale, where 0 reflects the poorest health-related quality of life and 100 reflects the best. Higher scores indicate better quality of life.
| score on a scale | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Baseline | 86 ± 16.36 | 62.14 ± 24.64 | 77.86 ± 12.2 |
| Month 12 | 88 ± 6.71 | 85.71 ± 13.36 | 72.5 ± 13.69 |
Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported
| Participants | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| Incidence of Mortality or Need for Transplantation | 0 | 0 | 3 |
Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open Label C-kit+ Cells Group A | 0/9 (0%) | 8/9 (88.9%) | 5/9 (55.6%) |
| C-kit+ Cells Group B | 0/8 (0%) | 4/8 (50%) | 6/8 (75%) |
| No Intervention Group | 1/8 (12.5%) | 4/8 (50%) | 6/8 (75%) |
| Event | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/9 | 2/8 | 3/8 |
| HaematocheziaGastrointestinal disorders | 3/9 | 0/8 | 0/8 |
| BradycardiaCardiac disorders | 1/9 | 1/8 | 2/8 |
| Cardiac arrestCardiac disorders | 1/9 | 0/8 | 2/8 |
| SepsisInfections and infestations | 0/9 | 0/8 | 2/8 |
| Feeding intoleranceMetabolism and nutrition disorders | 0/9 | 2/8 | 1/8 |
| Tricuspid valve incompetenceCardiac disorders | 2/9 | 0/8 | 1/8 |
| Viral infectionInfections and infestations | 2/9 | 0/8 | 0/8 |
| Aortic restenosisInjury, poisoning and procedural complications | 2/9 | 0/8 | 0/8 |
| Acute right ventricular failureCardiac disorders | 0/9 | 0/8 | 1/8 |
| Event | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group |
|---|---|---|---|
| COVID-19Infections and infestations | 0/9 | 2/8 | 0/8 |
| PneumoniaInfections and infestations | 1/9 | 0/8 | 2/8 |
| BradycardiaCardiac disorders | 0/9 | 0/8 | 1/8 |
| Tricuspid valve incompetenceCardiac disorders | 0/9 | 1/8 | 0/8 |
| HaematocheziaGastrointestinal disorders | 0/9 | 1/8 | 0/8 |
| Necrotising colitisGastrointestinal disorders | 0/9 | 1/8 | 1/8 |
| VomitingGastrointestinal disorders | 0/9 | 1/8 | 0/8 |
| Device embolisationGeneral disorders | 0/9 | 0/8 | 1/8 |
| Drug withdrawal syndromeGeneral disorders | 0/9 | 0/8 | 1/8 |
| GranulomaGeneral disorders | 0/9 | 0/8 | 1/8 |
| Age, Continuous(Months) | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group | Total |
|---|---|---|---|---|
| Mean | 4.18 ± 0.57 | 4.66 ± 0.85 | 4.7 ± 0.89 | 4.51 ± 0.77 |
| Sex: Female, Male(Participants) | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group | Total |
|---|---|---|---|---|
| Female | 1 | 3 | 4 | 8 |
| Male | 8 | 5 | 4 | 17 |
| Ethnicity (NIH/OMB)(Participants) | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group | Total |
|---|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 1 | 4 |
| Not Hispanic or Latino | 8 | 6 | 7 | 21 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Open Label C-kit+ Cells Group A | C-kit+ Cells Group B | No Intervention Group | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 1 | 0 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 4 | 1 | 0 | 5 |
| White | 3 | 6 | 7 | 16 |
| More than one race | 0 | 0 | 1 | 1 |
| Unknown or Not Reported | 1 | 1 | 0 | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Oct 2019 to primary completion in Jul 2024, 56 months.
Results were first posted in Jan 2026, 1 year 6 months after primary completion.
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Studies of the drug tested here: Cell Types
Hypoplastic Left Heart Syndrome →
Joshua M Hare