Congenital Adrenal Hyperplasia

Researchers are evaluating the safety, effectiveness, how the body processes, and the effects of atumelnant treatment in children with classic congenital adrenal hyperplasia (CAH). This Phase 2/3 study includes an open-label extension and involves pediatric participants aged 1 to under 18 years. The study is designed to assess these factors through multiple parts, including initial open-label cohorts, a randomized placebo-controlled phase, and a long-term extension. The study has three parts: Part A includes open-label, semi-sequential cohorts for different age groups with safety reviews before progressing; Part B is a double-blind, randomized, placebo-controlled phase comparing atumelnant to placebo; Part C is an open-label extension where participants from Parts A and B can continue treatment. Atumelnant and placebo are given as once-daily oral tablets with dosing based on weight. Participants will undergo various assessments including measuring morning serum androstenedione and 17-hydroxyprogesterone levels, pharmacokinetic and pharmacodynamic studies, and monitoring glucocorticoid doses. Safety and efficacy are evaluated at multiple time points up to 260 weeks. The study also includes long-term monitoring of hormone levels and glucocorticoid dosing while tracking participant adherence and health status throughout the study duration.

Researchers are evaluating the effects of atumelnant in adults with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. This Phase 3, global, multicenter, randomized, double-blind, placebo-controlled study aims to assess the drug's efficacy, safety, pharmacokinetics, and pharmacodynamics in participants aged 18 to under 75 years who have been on stable glucocorticoid therapy for at least two months. Participants will be randomly assigned in a 2:1 ratio to receive either atumelnant 80 mg once daily or a matching placebo for 32 weeks. There is an option to increase the atumelnant dose to 120 mg daily at Week 20. Before treatment begins, a screening period of 3 to 6 weeks will determine eligibility. Approximately 150 participants may take part in this study. During the study, participants will be monitored for hormone levels related to CAH and glucocorticoid dosing, with primary outcomes measured at Week 32. Assessments include morning serum androstenedione levels and other hormone markers. Safety and adherence to study procedures will also be closely observed. The study period extends up to 32 weeks of treatment following screening, with detailed evaluations throughout.

Researchers are evaluating the effects of traditional Chinese medicine combined with intradermal acupuncture on girls with idiopathic precocious puberty, a condition characterized by early onset of puberty without an identifiable cause. This phase 3 randomized controlled trial aims to determine if combining acupuncture point stimulation with herbal treatment can improve symptoms and prevent the condition from worsening. The study focuses on girls with mild to moderate precocious puberty and explores whether this combined approach offers benefits beyond traditional herbal medicine alone. Participants will be randomly assigned to one of two groups. One group will receive Ziyin-Xiehuo traditional Chinese medicine granules twice daily along with intradermal acupuncture point stimulation targeting specific points, applied three times daily with needles changed every two days, for a duration of six months. The other group will receive only the traditional Chinese medicine granules twice daily for the same six-month period. This design allows comparison between combined therapy and herbal medicine alone. During the study, researchers will monitor participants' breast development changes, particularly the reduction or disappearance of mammary nucleus size at three months. They will also assess bone age changes compared to chronological age over six months. Various evaluations such as ultrasound measurements of uterus and ovary size, Tanner staging, and hormone treatment history will be collected. Safety and efficacy will be carefully observed throughout the six-month treatment period.

Researchers are evaluating different doses of Lu AG13909 in adults with congenital adrenal hyperplasia (CAH), a rare genetic disorder that affects hormone production. The study aims to understand the safety, tolerability, how the body processes Lu AG13909, and the body's response to this investigational drug. The research is conducted across multiple phases to assess these aspects carefully. Participants will receive multiple intravenous doses of Lu AG13909 according to specified dosing schedules divided into three parts: Part A, Part B, and Part C. Each part starts after confirming the safety and tolerability of the dose from the previous part. After Part C, participants may have the option to continue treatment in an extension phase. The dosing and treatment schedules are carefully planned and monitored throughout the study. During the trial, participants will undergo regular assessments including blood tests to measure hormone levels like 17-hydroxyprogesterone and androstenedione, as well as monitoring for treatment-emergent adverse events and the presence of anti-drug antibodies. Pharmacokinetic parameters such as serum concentration and elimination half-life of Lu AG13909 will be tracked up to several months. The total participation duration can extend up to about a year or more depending on the study part and extension involvement.

Researchers are evaluating the effectiveness of acupuncture compared to metformin, both combined with lifestyle management, in improving glucose control in women with insulin resistance and polycystic ovary syndrome (PCOS). The study aims to see if these treatments improve ovulation, hormone balance, quality of life, and symptoms of anxiety and depression, while also investigating if acupuncture has fewer side effects. They also explore whether these treatments can reverse molecular changes that may help prevent type 2 diabetes. Participants will be randomly assigned to one of three groups: lifestyle management alone, lifestyle management plus acupuncture, or lifestyle management plus metformin. Acupuncture treatments involve inserting sterile needles into specific abdominal and leg acupuncture points related to ovarian and pancreatic function, administered three times weekly for four weeks, then twice weekly for twelve weeks. Metformin is taken orally, starting at a low dose and gradually increasing to 1500 mg daily over 16 weeks. All women receive lifestyle advice on weight management, diet, and exercise at the start. During the study, participants undergo assessments at the start, after 4 months of treatment, and again 4 months post-treatment. These include blood tests measuring insulin sensitivity, glucose levels, hormone levels, and molecular markers from tissue samples. Researchers will also monitor menstrual patterns, ovulation, quality of life, mental health symptoms, eating habits, and treatment side effects. The study lasts 8 months with ongoing evaluations to determine the cost-effectiveness and overall impact of each treatment approach.

Researchers are investigating how progesterone affects luteinizing hormone (LH) pulse frequency during waking and sleep periods in mid- to late pubertal girls, both with and without hyperandrogenism (HA). The study aims to find out if progesterone reduces waking LH pulse frequency more than sleep-associated LH pulse frequency in girls without HA, and whether this effect is less in girls with HA. The study is a randomized, placebo-controlled, double-blind crossover trial involving mid- to late pubertal girls at Tanner breast stage 3 or higher but no more than 2 years after menarche. Participants will attend two 18-hour clinical research unit admissions across separate menstrual cycles, receiving either oral micronized progesterone (0.8 mg/kg at 7 AM, 3 PM, 11 PM, and 7 AM) or placebo. Blood samples will be collected every 10 minutes overnight to measure LH pulses and other hormones. After at least two months, participants will switch treatments for the second admission. Participants will be closely monitored throughout the study with frequent blood tests to characterize LH secretion patterns. The main measure is LH pulse frequency while awake, with sleep LH pulse frequency as a secondary measure. The study includes detailed hormone analysis and statistical modeling to compare effects between girls with and without HA. The total participation includes two inpatient admissions separated by at least two months.

Researchers are assessing the long-term safety, tolerability, and effectiveness of atumelnant (CRN04894) in people with congenital adrenal hyperplasia (CAH). This open-label, single-arm extension study enrolls participants who have already completed earlier studies with atumelnant and who are judged by investigators to benefit from continued treatment. About 150 participants are expected to take part in this Phase 2 trial. Participants will receive atumelnant orally, with dosing up to the highest level previously tested in earlier studies for CAH. The treatment period may last up to two years, during which participants will continue to take stable doses of glucocorticoid replacement therapy such as hydrocortisone or equivalent. The study focuses on continued monitoring of safety and effectiveness as participants use atumelnant over this extended timeframe. Throughout the study, participants will have regular assessments including blood tests to measure hormone levels like androstenedione and 17-hydroxyprogesterone, monitoring for adverse events, and evaluations of glucocorticoid doses. Researchers will track hospitalizations related to CAH, adrenal crises, and other safety outcomes up to week 108. This long-term approach aims to provide detailed information about the impact of atumelnant on participant health and disease management over time.

This research aims to validate the B-COMPASS, a computational model developed by the BEAMER project to improve patient adherence to treatment across various diseases. Adherence to treatment is a widespread issue leading to increased healthcare use and premature death, with about half of medications not taken as prescribed. The study evaluates the model's ability to predict adherence, identify patient support needs, and enhance healthcare engagement in six therapeutic areas, including cardiovascular, endocrinology, immunology, neurology, oncology, and rare diseases. Participants complete the BEAMER questionnaire, which the B-COMPASS uses to group patients based on their adherence needs and predict their adherence. Patients are randomized into a control group receiving standard care or an intervention group receiving enhanced engagement through educational materials for healthcare providers tailored to the patient's B-COMPASS profile. Engagement occurs in person or by phone, depending on patient schedules, and healthcare providers may also be randomized to limit knowledge of the B-COMPASS in control groups. Participants are involved in two main data collections spaced from 2 weeks to 6 months apart, during which adherence measures, patient support needs, and perceptions of engagement are assessed. The study measures the accuracy, validity, and reliability of B-COMPASS predictions, its impact on adherence, patient and provider perceptions, and cost-effectiveness. The total participation timeline varies by site and disease area, with continuous monitoring of healthcare utilization and patient experience.

Researchers are studying women with Polycystic Ovary Syndrome (PCOS) to investigate markers related to blood vessel, heart muscle, and inner vessel lining function. The study looks at these markers both before and during a glucose tolerance test, as well as changes after treatment. It explores how metabolic and hormonal abnormalities in PCOS affect these markers and evaluates the impact of different treatments over time. Participants will be divided into four groups: one group will receive lifestyle changes combined with a GLP-1 receptor agonist drug, another will receive lifestyle changes with an oral contraceptive, a third group will receive lifestyle changes with metformin, and the last group will receive lifestyle changes alone. After six months of treatment, the same tests will be repeated to observe changes. Tests include glucose and insulin levels, arterial stiffness, heart function using echocardiography, blood vessel dilation, and hormone levels. During the study, women will undergo detailed measurements at baseline, during the oral glucose tolerance test at multiple time points, and after six months of treatment. These assessments include blood tests, heart ultrasound scans, and blood vessel function tests. Researchers will track changes in heart strain, arterial stiffness, and blood vessel lining thickness as primary outcomes. Secondary outcomes include changes in coronary flow and blood vessel dilation. The study will last over a year with follow-up assessments at six and twelve months.

Researchers are evaluating treatments for congenital adrenal hyperplasia, a genetic disorder caused by 21-hydroxylase deficiency that leads to cortisol deficiency and excess androgens. This trial compares the effects of a once-daily dual-release hydrocortisone preparation versus conventional glucocorticoid therapy on various health aspects including metabolic syndrome, hormonal balance, bone health, quality of life, reproductive and psychological functions, and treatment adherence. The study is open and controlled, aiming to improve management strategies for adults with this condition. Participants are randomly assigned to one of two groups: one continues their conventional glucocorticoid therapy, while the other receives dual-release hydrocortisone tablets once daily while fasting. The dose of hydrocortisone remains the same as before entering the trial. The dual-release formulation is designed to better mimic the body's natural cortisol rhythm, potentially reducing adverse effects seen with current treatments. Throughout the study, participants are monitored regularly with various assessments including cholesterol levels, blood sugar, BMI, blood pressure, hormone levels, bone density, quality of life surveys, sexual function evaluations, and mental health status. These measurements are taken at the start and at 6, 12, and 24 months to track changes over time. Safety is also monitored by recording any adverse events. The study duration allows researchers to observe both short- and long-term effects of the treatments.