Hypoplastic Left Heart SyndromeNCT01708863
A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation
An observational study of Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 18 months
- Conditions the study is for
- Hypoplastic Left Heart Syndrome
- How many people
- 4 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2012to 15 May 2020
- Phase
- Observational
- 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 0 to 18 months
- All sexes
- No healthy volunteers
- Child
Study population: Children with HLHS up to 18 months of age requiring a planned Stage II bi-directional Glenn surgery
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Oklahoma University Children's Hospital
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55901
About this study
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
- Medical subject headings
- Hypoplastic Left Heart Syndrome, Heart Defects, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Children with Hypoplastic Left Heart Syndrome (HLHS) | HLHS patients requiring Stage II Glenn surgery |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2012 (estimated)
- Study startOct 2012
- Primary completionMay 2020
- Study completion15 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypoplastic Left Heart SyndromeStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Timothy J Nelson, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- University of Oklahoma, Children's Hospital of Philadelphia, Mayo Clinic
- Responsible party
- Sponsor-investigator, Timothy J Nelson, MD, PhD, ReGen Theranostics, Inc.
- Investigators
- Timothy J Nelson, MD, PhD, Study director, Mayo ClinicMuhammad Y Qureshi, MBBS, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01708863
- Sponsor's protocol ID
- 12-002887
- Status last verified
- Oct 2020
- First submitted
- 15 Oct 2012
- Last updated
- 5 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation
- ClinicalTrials.gov ID
- NCT01708863
- Acronym / sponsor ID
- 12-002887
- 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 type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Timothy J Nelson, MD, PhDUniversity, hospital or other
- Collaborators
- University of Oklahoma, Children's Hospital of Philadelphia, Mayo Clinic
- Study start
- Oct 2012 (actual)
- Primary completion
- May 2020 (actual)
- Study completion
- 15 May 2020 (actual)
- First posted
- 17 Oct 2012 (estimated)
- 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children with Hypoplastic Left Heart Syndrome (HLHS) | – | –HLHS patients requiring Stage II Glenn surgery |
Who may qualify
- Ages
- 0 to 18 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with HLHS up to 18 months of age requiring a planned Stage II bi-directional Glenn surgery
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (5 listed)
- Severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
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 5 Oct 2020.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
Related trials for Hypoplastic Left Heart Syndrome
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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.
Timothy J Nelson, MD, PhD study director
Mayo Clinic
Muhammad Y Qureshi, MBBS principal investigator
Mayo Clinic
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 3 sites 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 2 inclusion and 5 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 5 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
Where can I read the original study information?
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 5 Oct 2020.