A Phase 2b, Open-label Dose-ranging Study Evaluating the Safety, Tolerability, Pharmacodynamics and Pharmacokinetics, and Efficacy of CLTX-305 (Encaleret) in Autosomal Dominant Hypocalcemia (ADH) Type 1
Who is this study for? Child to adult patients with Autosomal Dominant Hypocalcemia
What treatments are being studied? Encaleret
Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The primary purpose of this study is to evaluate the safety, tolerability and effectiveness of encaleret in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
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• Be able to understand and sign a written informed consent or assent form, which must be obtained prior to initiation of study procedures.
• Postmenopausal women are allowed to participate in this study
• Body mass index (BMI) ≥ 18.5 to \< 39 kilograms (kg)/square meter (m\^2)
• Have an activating mutation of the Calcium-sensing receptor (CASR) gene
• Participants being treated with thiazide diuretics may be enrolled if they are willing and able to discontinue thiazides
• Participants being treated with strong Cytochrome P3A4 (CYP3A4) inhibitors should ideally, if clinically appropriate, discontinue these medications during the screening period
• Participants being treated with magnesium or potassium citrate supplements should discontinue such treatment starting on Day -1 during Period 1 and Period 2 and may be asked to discontinue treatment during Period 3
Locations
United States
Maryland
National Institutes of Health (NIH) Clinical Center
Period 1: Participants will receive an ascending dose of encaleret once daily for the first 3 days. Participants will then receive an individualized dose of encaleret twice daily for 2 days.~Period 2: Participants will receive encaleret twice daily for 5 days at a single dose level based on responses from Period 1.~Period 3: After completion of Period 2, participants will be eligible to receive encaleret for an additional 24 weeks.~Long-Term Extension (LTE): At the end of the study, participants will also have an option to receive encaleret for up to an additional 2 years.
Experimental: Cohort 2: Steady-State Dose
Participants will directly be enrolled into Period 2, and receive encaleret twice daily at a dose based on data and responses from Cohort 1 Period 1.~Period 2: Participants will receive encaleret twice daily for 5 days.~Period 3: After completion of Period 2, participants will be eligible to receive encaleret for an additional 24 weeks.~LTE: At the end of the study, participants will also have an option to receive encaleret for up to an additional 2 years.