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This study aims to look at AMH levels in female children with SCD as they go through puberty to see if they are at the same level as other children without SCD at the same age and/or pubertal stage and will also look at how treatment exposures and pain crises affect the AMH levels in children with SCD.
Primary Objective:
Secondary Objectives:
This is a non-therapeutic cross-sectional two-year pilot study within an eight-year longitudinal study.
After the parent and/or patient has given permission to enroll on the research study, review of the electronic medical record and SCCRIP (NCT02098863) data base will be performed to obtain data on current medical therapy, number of vaso-occlusive events and types of events as well as ED or hospital visits, laboratory studies and use of sickle cell disease modifying therapies.
A questionnaire will be distributed annually from time of enrollment until age 19.0 years to participants with SCD and consenting legal guardian (if applicable). For healthy controls, the same questionnaire will be provided to coincide with their one-time research visit. Pubertal status will be collected from questionnaires (prepubertal/no breast tissue, pubertal (breast tissue, but no menses), post-menarcheal. For those menstruating, regularity of menses will be ascertained through questionnaire answers and also date of last menstrual period. Questions for participants will also include questions regarding all forms of hormonal contraception and current opiate-containing medications.
For subjects with SCD, blood will be collected at the time of a clinic visit and occur concurrently with an already scheduled lab draw for clinical care with a goal frequency of annually. For controls, blood will be collected at a one-time study visit.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants with Sickle Cell Disease | Females with sickle cell disease, all genotypes (age at enrollment ≥ 10 years and < 19 years) | ||
| Healthy Controls | Healthy female siblings/relatives of patients with SCD (including those with sickle cell trait), and other females of similar race/ethnicity (age at enrollment ≥ 10 years and < 19 years) |
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| Measure | Description | Time Frame |
|---|---|---|
| Difference in AMH levels in pre-teens and adolescent females with SCD compared with healthy female controls (siblings, relatives, or non-relatives) at the same age | Investigators will address this by targeting the relative difference in mean. For each participant, the earliest AMH measurement will be used. All patients recruited during the cross-sectional stage with at least 1 AMH measurement will be evaluable for the analysis, except for patients who have received hematopoietic stem cell transplant (HSCT) or gene therapy before their first study AMH measurement. Patients who either (1) have no available AMH at the end of the cross-sectional phase or (2) received HSCT or gene therapy before their first study AMH measurement will be considered unevaluable for this analysis. | Earliest AMH collection after enrollment, up to 2 years after study activation |
| Difference in AMH levels in pre-teens and adolescent females with SCD compared with healthy female controls (siblings, relatives, or non-relatives) at the same pubertal stage | Investigators will address this by targeting the relative difference in mean. For each participant, the earliest AMH measurement will be used. Pubertal status will be defined as a nominal variable with the following categories: prepubertal, pubertal but premenarchal, postmenarchal, and 3 years postmenarchal. This will be derived from the self-reported status of any breast development and experiencing menarche by the time of the visit with the AMH draw. | Earliest AMH collection after enrollment, up to 2 years after study activation |
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Inclusion Criteria:
Exclusion Criteria:
Females with Sickle cell disease of any genotype or a healthy sibling, relative, household member, or other females of similar race/ethnicity of a patient with sickle cell disease
Females with Sickle cell disease of any genotype or a healthy sibling, relative, household member, or other females of similar race/ethnicity of a patient with sickle cell disease
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Christine Yu, MD | Contact | 888-226-4343 | referralinfo@stjude.org |
| Name | Affiliation | Role |
|---|---|---|
| Christine Yu, MD | St. Jude Children's Research Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Jude Children's Research Hospital | Recruiting | Memphis | Tennessee | 38105 | United States |
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| Label | URL |
|---|---|
| St. Jude Children's Research Hospital | View source |
| Clinical Trials Open at St. Jude | View source |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |