Clinical trial NCT01708863: A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation
Official title: A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hypoplastic Left Heart Syndrome
Keywords: Hypoplastic Left Heart Syndrome
Stage II palliative surgery
Glenn procedure
Congenital heart defect
Congenital heart disease
Underdeveloped left ventricle
Lead sponsor: ReGen Theranostics, Inc.
Sponsor class: OTHER
Collaborators: name: University of Oklahoma
name: Children's Hospital of Philadelphia
name: Mayo Clinic
First posted: 2012-10-17
Enrollment: 4
Sex: ALL
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with HLHS up to 18 months of age requiring a planned Stage II bi-directional Glenn surgery

Brief summary: Hypoplastic left heart syndrome (HLHS) is a severe form of congenital heart disease that consists of multiple obstructions to flow through the left heart and aorta, as well as hypoplasia of the left ventricle. Most patients require a three-stage surgical protocol starting within days of birth. Stage I of this process is the Norwood reconstruction (within the first few days of life), Stage II (usually required within 3-8 months) involves creation of a direct connection between the patient's superior vena cava and the pulmonary arterial confluence (bidirectional Glenn anastomosis), and the last stage is creation of a Fontan circulation (typically within the first 2-4 years). This "single ventricle" approach requires the right ventricle to perform as the only circulatory pump for the entire body.

Our long-term goal is to develop regenerative strategies to strengthen and augment the right ventricular muscle of the single-ventricle heart following surgical palliation in HLHS patients. To determine the safety and feasibility of a cell-based therapeutic intervention at the Stage II surgery, we aim to document the natural history of post-surgical care in HLHS patients having undergone standard of care with protocol specific follow-up over the course of a 6-month period.

This prospective study will document the natural history in patients with HLHS after planned Stage II surgical palliation with a focus on cardiovascular parameters within 6 months following surgery in 10 patients.


Eligibility criteria: Inclusion Criteria:

* Individuals with HLHS having undergone Stage I surgical palliation and undergoing planned Stage II palliative Glenn surgery.
* Individuals up to 18 months of age are eligible if written informed consent can be obtained from both parents and/or legal guardians, unless one parent is not reasonably available.

Exclusion Criteria:

* Severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer

The following complications of congenital heart disease:

* Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage II surgical repair.
* Severe pulmonary hypertension (reported in the medical record as \>70% systemic pressure).
* Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe complications or very poor outcome during or after Stage II surgical repair.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Oklahoma City
country: United States
facility: Oklahoma University Children's Hospital
state: Oklahoma
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Mayo Clinic
name: Timothy J Nelson, MD, PhD
role: STUDY_DIRECTOR
affiliation: Mayo Clinic
name: Muhammad Y Qureshi, MBBS
role: PRINCIPAL_INVESTIGATOR
References: