Functional Ovarian Reserve in Sickle Cell Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 440
- 试验地点
- 1
- 主要终点
- 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
研究概览
简要总结
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:
- To evaluate whether AMH levels are lower in pre-teens and adolescent females with SCD when compared with healthy female controls (siblings, relatives, non-relatives of similar race/ethnicity) at the same age and pubertal stage.
Secondary Objectives:
- To evaluate whether AMH has a similar trajectory in female pre-teens and adolescents with SCD when compared with the general population and controls.
- To describe pubertal timing, menstrual history, and markers of functional ovarian reserve (FOR), as well as prevalence of premature ovarian insufficiency (POI) as determined by medical history and laboratory markers in pre-teens and adolescents with SCD in comparison with their female controls.
- To correlate AMH levels with FSH and estradiol levels, normal pubertal timing, and menstrual history in children and adolescents with SCD.
- To correlate the severity of SCD (number of vaso-occlusive events) with pubertal timing, presence of normal vs abnormal menstruation, and laboratory markers of FOR, in pre-teens and adolescents with SCD.
- To correlate the use of SCD modifying treatment modalities with pubertal timing, menstrual pattern, and laboratory markers of FOR in pre-teens and adolescents with SCD.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 10 Years 至 18 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 是
入选标准
- •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
- •Age at enrollment ≥ 10 years and < 19 years
排除标准
- •History of hematopoietic stem cell transplantation or gene therapy prior to enrollment or preparing for hematopoietic stem cell transplantation or gene therapy prior to enrollment
- •Inability or unwillingness of research participant or legal guardian/representative to give written informed consent
- •Pregnancy
研究组 & 干预措施
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)
结局指标
主要结局
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
时间窗: Earliest AMH collection after enrollment, up to 2 years after study activation
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.
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
时间窗: Earliest AMH collection after enrollment, up to 2 years after study activation
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.
次要结局
未报告次要终点