Hereditary SpherocytosisNCT01141621
The Dallas Hereditary Spherocytosis Cohort Study
An observational study of Hereditary Spherocytosis, run by University of Texas Southwestern Medical Center.
Trial summary
The purpose of this study is to 1. better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy 2. evaluate and describe the health-related quality of life in children with HS. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Hereditary Spherocytosis
- How many people
- 55 participants
- Where
- No sites listed yet
- Study dates
- May 2010to May 2013
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Center for Cancer and Blood Disorders outpatient clinics
Who may be able to join
- Diagnosis of HS with or without prior splenectomy
- Age 0 - 21 years
- Spanish-speaking subjects are eligible to participate
Who cannot join
- Unable to provide contact information for follow-up
About this study
The purpose of this study is to
- better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy
- evaluate and describe the health-related quality of life in children with HS.
Read the detailed description
Patients with a new or established diagnosis of HS seen at Children's Medical Center will be asked to enroll in the study. Previous and current medical records will be reviewed to systematically catalogue their history of HS, including diagnosis, complications, hospitalizations, medications and laboratory data. Health-related quality of life questionnaires will be given to the patients and their parents at enrollment and periodically during the follow-up. Those who agree will have up to three small samples of blood collected and frozen for future laboratory studies of complications associated with HS and/or splenectomy.
We anticipate enrolling approximately 200 children and young adults with HS in this study and following them until adulthood (age 18-21 years).
- Conditions
- Hereditary Spherocytosis
- Keywords
- HS, congenital hemolytic anemia, splenectomy, quality of life
- Medical subject headings
- Spherocytosis, Hereditary, Anemia, Hemolytic, Congenital
What is being measured
The primary outcome is the main question the study is designed to answer.
Health related quality of life
PedsQL measurement
Measured: Approximately at 5 years
Secondary outcomes (4)
Primary indications for splenectomy
Primary indication for splenectomy determined at time of splenectomy, if performed.
Measured: Approximately at 5 years
Complications of HS
Measured: Approximately at 5 years and at 10 years
Complications of splenectomy
Measured: Approximately at 5 years
Diagnosis of HS
Measured: At enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2010 (estimated)
- Study startMay 2010
- Primary completionMay 2013
- Study completionMay 2013
Why it stopped: Insufficient staff to continue pursuing project
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary SpherocytosisStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- 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
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01141621
- Sponsor's protocol ID
- IRB # 022010-024
- Status last verified
- May 2013
- First submitted
- 4 Jun 2010
- Last updated
- 14 May 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Dallas Hereditary Spherocytosis Cohort Study
- ClinicalTrials.gov ID
- NCT01141621
- Acronym / sponsor ID
- IRB # 022010-024
- Conditions
- Hereditary Spherocytosis
- Keywords
- HS, congenital hemolytic anemia, splenectomy, quality of life
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Insufficient staff to continue pursuing project
- Enrolment
- 55 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- May 2013 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 10 Jun 2010 (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.
The purpose of this study is to
- better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy
- evaluate and describe the health-related quality of life in children with HS.
Detailed description
Patients with a new or established diagnosis of HS seen at Children's Medical Center will be asked to enroll in the study. Previous and current medical records will be reviewed to systematically catalogue their history of HS, including diagnosis, complications, hospitalizations, medications and laboratory data. Health-related quality of life questionnaires will be given to the patients and their parents at enrollment and periodically during the follow-up. Those who agree will have up to three small samples of blood collected and frozen for future laboratory studies of complications associated with HS and/or splenectomy.
We anticipate enrolling approximately 200 children and young adults with HS in this study and following them until adulthood (age 18-21 years).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Center for Cancer and Blood Disorders outpatient clinics
Key inclusion criteria (3 listed)
- Diagnosis of HS with or without prior splenectomy
- Age 0 - 21 years
- Spanish-speaking subjects are eligible to participate
Key exclusion criteria (1 listed)
- Unable to provide contact information for follow-up
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
Health related quality of life
Time frame: Approximately at 5 years
PedsQL measurement
4 secondary outcomes
Primary indications for splenectomy
Time frame: Approximately at 5 years
Complications of HS
Time frame: Approximately at 5 years and at 10 years
Complications of splenectomy
Time frame: Approximately at 5 years
Diagnosis of HS
Time frame: At enrollment
Results and safety
The study was stopped early (reason given: Insufficient staff to continue pursuing project). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2013.
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Splenectomy
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Spherocytosis, run by University of Texas Southwestern Medical Center.
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 3 inclusion and 1 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: stopped early, as of the sponsor's last update on 14 May 2013. Status can change between updates, so confirm with the study contact before making plans.
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 was stopped early (reason given: Insufficient staff to continue pursuing project). Results are not available in the source record.
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.
What does the primary outcome measure?
Health related quality of life (time frame: Approximately at 5 years). It is what the study was designed to measure, not a finding.
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 14 May 2013.