CLINICAL TRIAL

Lomecel-B medicinal signaling cells for Hypoplastic Left Heart Syndrome

Stage II
Recruiting · < 18 · All Sexes · Cincinnati, OH

This study is evaluating whether Lomecel-B works in treating patients with hypoplastic left heart syndrome.

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About the trial for Hypoplastic Left Heart Syndrome

Eligible Conditions
Hypoplastic Left Heart Syndrome · Syndrome

Treatment Groups

This trial involves 2 different treatments. Lomecel-B Medicinal Signaling Cells is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Lomecel-B medicinal signaling cells
BIOLOGICAL
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

Eligibility

This trial is for patients born any sex aged 18 and younger. There is one eligibility criterion to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
1. All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline, 12 Months
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline, 12 Months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline, 12 Months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Lomecel-B medicinal signaling cells will improve 20 secondary outcomes in patients with Hypoplastic Left Heart Syndrome. Measurement will happen over the course of 30 days post Stage II palliation surgery.

Participants experiencing treatment emergent serious adverse events (TE-SAEs)
30 DAYS POST STAGE II PALLIATION SURGERY
Safety will be reported as the number of participants experiencing treatment emergent serious adverse events (TE-SAEs) assessed by treating physician within the 30 days following study procedure. These include: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention; cardiogenic shock; unplanned cardiovascular operation for bleeding due to right ventricular intramyocardial injection site bleeding in the first five days after stage II operation; worsening of cardiac function; local infection or systemic infection; need for new permanent pacemaker; death.
30 DAYS POST STAGE II PALLIATION SURGERY
Change in right ventricular ejection fraction (RVEF)
BASELINE, 6 MONTHS
Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
BASELINE, 6 MONTHS
Participants experiencing major adverse cardiac events (MACE)
BASELINE, 12 MONTHS
Safety will be reported as the number of participants with adjudicated events including cardiovascular morbidity; need for transplantation; re-hospitalizations; cardiovascular mortality; all-cause mortality.
BASELINE, 12 MONTHS
Change in PedsQL™ Infant Scales
BASELINE, 12 MONTHS
The PedsQL™ Infant Scales is a generic health related quality of life instrument specifically for healthy and ill infants ages 1-24 months. Efficacy measured as change in PedsQL total improvement score and will be measured serially by parental proxy-report. Higher scores are indicative of better quality of life.
BASELINE, 12 MONTHS
Change in biomarkers/cytokines
BASELINE, 12 MONTHS
Efficacy measured as change in biomarkers/cytokines in pg/ml and/or ng/ml and will be measured serially by blood draw.
BASELINE, 12 MONTHS
Change in modified Ross Heart Failure Classification score
BASELINE, 12 MONTHS
The Ross Heart Failure Classification provides a global assessment of heart failure severity in infants. Efficacy measured as change in classification and will be measured serially via physician's assessment using the modified Ross Heart Failure Classification; classifications defined as Class 1 (no limitations of physical activity); Class 2 (may experience symptoms during moderate exercise but not during rest); Class 3 (symptoms with minimal exertion that interfere with normal daily activity); Class 4 (unable to carry out physical activity/has symptoms at rest that worsen with exertion).
BASELINE, 12 MONTHS
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Who is running the study

Principal Investigator
S. K.
Sunjay Kaushal, Principal Investigator
Ann & Robert H Lurie Children's Hospital of Chicago

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can hypoplastic left heart syndrome be cured?

This case is an example of the unpredictable nature of the surgical result in a young child. Treatment was originally proposed, but it was not feasible due to technical complexity and prohibitive cost. The long-term prognosis is challenging but surgical results are promising with early follow-up.

Anonymous Patient Answer

What are the signs of hypoplastic left heart syndrome?

Aspirin, hypothyroidism, and poor growth charts should be systematically screened in children with HLHS. Congenital heart problems should be evaluated with Doppler echocardiography or cardiac catheterization/ angiography. The initial presentation of severe hypoplastic left heart syndrome with symptoms of failure to thrive, cyanosis, and tachypnea should prompt evaluation with cardiocardiography or other definitive testing.

Anonymous Patient Answer

What causes hypoplastic left heart syndrome?

Cardiac anomalies are common. The hypoplastic left heart syndrome is more than a genetic disease but is strongly associated with other cardiac anomalies. Some of these cardiovascular anomalies are in turn caused by some cardiac malformations. It appears that most patients with hypoplastic left heart syndrome can be predicted with an ultrasound examination, and these antenatal imaging findings can be used to guide treatment, particularly in low-risk cases.

Anonymous Patient Answer

How many people get hypoplastic left heart syndrome a year in the United States?

Hypoplastic left heart syndrome is prevalent. Although no significant differences were demonstrated in the incidence of hypoplastic left heart syndrome between whites and blacks, infants from whites and blacks had similar survival.

Anonymous Patient Answer

What are common treatments for hypoplastic left heart syndrome?

Treatment of hypoplastic left heart syndrome is multifaceted and requires clinical judgment regarding risk stratification, patient selection, and timing and sequence of treatment. These decisions are influenced by complex, evolving, and interactive genetic and environmental factors.

Anonymous Patient Answer

What is hypoplastic left heart syndrome?

These data suggest that children with aortic arch obstruction and LV hypoplasia should be considered candidates for bidirectional cavopulmonary anastomosis due to the significant risk of mortality with HLHS.

Anonymous Patient Answer

Has lomecel-b medicinal signaling cells proven to be more effective than a placebo?

Lomecel-b has a greater safety profile and enhanced antitumor activity than the placebo. The mechanisms for this unexpected antitumor activity appear to be mediated through the induction of apoptosis and autophagy following treatment with Lomecel-b.

Anonymous Patient Answer

Is lomecel-b medicinal signaling cells typically used in combination with any other treatments?

The data suggest that Lomecel-B signal cells used in combination with radiation therapy are effective in killing malignant tumor cells in the hypoplastic left heart syndrome mouse model of pediatric heart disease.

Anonymous Patient Answer

What is the average age someone gets hypoplastic left heart syndrome?

Because the incidence of this condition decreases with age, the earlier detection of newborns with hypoplastic left heart syndrome has become an important and necessary issue. The use of the echocardiogram for identifying newborn with hypoplastic left heart syndrome in primary care may reduce costs and unnecessary visits.

Anonymous Patient Answer

What does lomecel-b medicinal signaling cells usually treat?

Lomecel-B medical signaling cells could be expected to promote a self-healing mechanism through cytokine and/or paracrine action. Injection of lomecel-b medical signaling cells promotes collateral circulation formation, reduces apoptotic cell number, and stimulates angiogenesis; therefore, it is a promising and promising medical treatment method that deserves further investigation.

Anonymous Patient Answer

What is the latest research for hypoplastic left heart syndrome?

While a lot is being written about hypoplastic left heart syndrome and associated issues, many things are known about cardiovascular management with the congenital disease. The most recent data should help with the clinical management of any new case of infant cardiologists.

Anonymous Patient Answer

Is lomecel-b medicinal signaling cells safe for people?

A single course of lomecel-b cells is safe in children with HLHS. Recent findings support the recent findings that lomecel-b cell treatment may support myocardial health, which may improve exercise capacity in children with HLHS.

Anonymous Patient Answer
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