The CALIBRATE study is a clinical research study designed to find out whether a study medicine can balance calcium levels in people with autosomal dominant hypocalcemia type 1 (ADH1), a genetic condition that causes low blood calcium levels.
Study participants may be able to join the study if they are 16 years of age or older and have a confirmed diagnosis of autosomal dominant hypocalcemia type 1 (ADH1). If you do not know whether you have ADH1, ask your doctor about a test that can help to provide this information. Contact us to speak with a study doctor and learn more about what is required to participate in the study.
The study medicine is an investigational drug called encaleret. Investigational means the drug has not been approved by any health or regulatory agency and can only be used in clinical studies.
This research study will check how safe and effective encaleret is in people with ADH1. Encaleret is a tablet taken by mouth. Some participants will receive standard of care supplements (calcium supplements and/or active vitamin D analogues) instead of encaleret during the first part of the study, but all participants will receive encaleret if they are eligible and choose to participate in the Long-term Extension.
The study medicine is an investigational drug called encaleret. Investigational means it has not been approved by any health or regulatory agency and can only be used in clinical studies.
This research study will check how safe and effective encaleret is in people with ADH1. Encaleret is a tablet taken by mouth. Some participants will receive standard of care supplements (calcium supplements and/or active vitamin D analogues) instead of encaleret during the first part of the study, but all participants will receive encaleret if they are eligible and choose to participate in the Long-term Extension.
Participation in the CALIBRATE study lasts about 1 year, followed by an optional extension of up to 4 years. Study participants can expect the following:
Informed Consent
The participant (or a parent or legal guardian if they are younger than 18 years old) is required to read and sign the Informed Consent Form (ICF) before the participant has any tests or procedures.
Screening and SoC Optimization (up to 5 months)
Participants will take calcium supplements and/or active vitamin D (for example, calcitriol or alfacalcidol) which are considered to be current Standard of Care (SoC) for ADH1. Participants will receive study tests and procedures to determine if they qualify for the CALIBRATE Study.
SoC Maintenance (about 1 month)
Participants will continue to take SoC supplements and attend two study visits for study tests and procedures.
Study Medication or SoC Adjustment (about 5 months)
Participants will be assigned randomly to either a group that continues SoC supplements or to a group that takes the study medication. There will be twice as many participants assigned to the study medication group. Participants will attend regular study visits for study tests and procedures.
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Study Medication or SoC Maintenance (about 1 month)
Participants continue taking study medicine or SoC supplements (as assigned) and attend 1 study visit for study tests and procedures.
Optional Long-Term Extension (4 years)
All eligible participants who elect to take part in the long-term extension study will receive the study medicine and attend study visits regularly every 3 months (if you were previously assigned to SoC, you will have more frequent visits at first) for up to 4 years. About half of these visits may be able to be completed at home.
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
