Functional Ovarian Reserve in Sickle Cell Disease
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexFemale
Age10-18
SponsorSt. Jude Children's Research Hospital
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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
Females
Disqualifiers
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
Trial population
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
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
440 Participants
are grouped into 2 trial groupsGroup A: Participants with Sickle Cell Disease
Group B: Healthy Controls
Trial outcomes
Primary outcomes
1
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.
Time frame
Earliest AMH collection after enrollment, up to 2 years after study activation2
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.
Time frame
Earliest AMH collection after enrollment, up to 2 years after study activationSponsors and contacts
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Source ClinicalTrials.gov