A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation

NCT01708863 | Completed

• 1 other identifier: 12-002887
• Study Type: observational
• Target: 4
• Locations: 1 country
• Sites: 3

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.

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

Study Arms (1)

Children with Hypoplastic Left Heart Syndrome (HLHS)

HLHS patients requiring Stage II Glenn surgery

Eligibility Criteria

• Age: 0 Months - 18 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Timothy J Nelson, MD, PhD: lead
• University of Oklahoma: collaborator
• Children's Hospital of Philadelphia: collaborator
• Mayo Clinic: collaborator

Study Sites (3)

Mayo Clinic

Rochester, Minnesota, 55901, United States

Location

Oklahoma University Children's Hospital

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome; Heart Defects, Congenital

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Timothy J Nelson, MD, PhD

  Mayo Clinic

  STUDY DIRECTOR

• Muhammad Y Qureshi, MBBS

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Program Director

Study Record Dates

• First Submitted: October 15, 2012
• First Posted: October 17, 2012
• Study Start: October 1, 2012
• Primary Completion: May 1, 2020
• Study Completion: May 15, 2020
• Last Updated: October 5, 2020

Record last verified: 2020-10

Locations

US (3)