Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05680818 | Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1 | PHASE3 | ACTIVE NOT_RECRUITING | 67 | — | — | Jan 6, 2023 | Aug 1, 2029 | Oct 31, 2025 | 25 | United States, Australia +8 |
| NCT04581629 | Safety, Tolerability, and Efficacy of Encaleret in Participants With Autosomal Dominant Hypocalcemia (ADH) Type 1 | PHASE2 | COMPLETED | 13 | — | — | Sep 20, 2020 | Sep 7, 2023 | Nov 19, 2024 | 1 | United States |
* cCa within 8.3-10.7 mg/dL (2.08-2.68 millimoles per liter \[mmol/L\]) * 24-hr UCa within the reference range (\< 300 mg/day for men \[7.5 mmol/day\], \< 250 mg/day for women \[6.25 mmol/day\])
Adverse events (AEs) were defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related. Treatment-emergence was defined as any AE(s) regardless of relationship to investigational medicinal product (IMP), that had an onset or worsened in severity on or after the first dose of IMP.
| Arm | Type | Description |
|---|---|---|
| Encaleret | EXPERIMENTAL | Participants will receive encaleret at a dose as needed based on calcium levels. |
| Standard of Care (SoC) | OTHER | Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
| Cohort 1: Ascending + Steady-State Dose | EXPERIMENTAL | 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. |
| Cohort 2: Steady-State Dose | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| Encaleret | DRUG | Administered as film-coated tablet for oral use |
| Standard of Care | DIETARY_SUPPLEMENT | Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
Key Inclusion Criteria: 1. Participants must have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance, of the calcium sensing receptor (CASR) gene associated with biochemical findings of hypoparathyroidism. 2. Participants must have a documented his...