Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1 (NCT05680818) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
United States67 participantsStarted 2023-01-06
Plain-language summary
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Who can participate
Age range16 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Participants must have a documented history of symptoms or signs of ADH1.
✓. Participants 16 to \<18 years old must have closed growth plates on hand radiograph.
✓. Participants treated with thiazide diuretics must discontinue thiazides for at least 14 days prior to SoC Optimization Visit 1 through Week 24 (Period 3). When the thiazide is being used as an antihypertensive, alternative therapy will be prescribed by the Investigator as needed.
✓. Participants treated with phosphate binders (other than calcium salts) must discontinue the phosphate binders at least one day prior to the SoC Optimization Visit 1.
✓. Participants treated with magnesium or potassium supplements must be willing to discontinue such treatment prior to the first dose of encaleret.
✓. Participants treated with potassium-sparing diuretics must be willing to discontinue such treatment prior to the first dose of encaleret.
✓. Participants must meet SoC Optimization criteria as defined in the protocol.
Exclusion criteria
✕. History of hypocalcemic seizure within the past 3 months preceding Screening.
✕. History of thyroid or parathyroid surgery.
What they're measuring
1
Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range
Timeframe: Up to Week 24
Trial details
NCT IDNCT05680818
SponsorCalcilytix Therapeutics, Inc., a BridgeBio company
✕. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (β-hCG) laboratory test.
✕. History of treatment with parathyroid hormone (PTH) 1-84 or 1-34 within the 2 months preceding Screening and requiring SoC doses exceeding \>1.2× their pre-PTH treatment total daily doses or bone turnover markers, Collagen cross-linked C-telopeptide (CTx )and Procollagen type 1 N-propeptide (P1NP), \> upper limit of normal for sex, age (men only) and menopausal status (women only).
✕. Blood 25-OH Vitamin D level \<25 nanograms (ng)/milliliter (mL).
✕. Estimated glomerular filtration rate (eGFR) \<30 mL/minute/1.73 m\^2 using chronic kidney disease-EPI creatinine equation refit without the race variable (chronic kidney disease-EPI creatinine equation refit without the race variable \[CKD-EPIcr\_R\]) (for participants \<18 years old the Bedside Schwartz equation should be used).
✕. Participants with positive Hepatitis B surface antigen (HBsAg), Hepatitis A immunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test at the Screening Visit. Participants who are in complete remission from Hepatitis C virus (HCV) as evidenced by sensitive assay ≥12 weeks after completion of HCV therapy may participate in the study.