Phase
Condition
Parathyroid Disorders
Hypoparathyroidism
Treatment
Encaleret
Calcium supplements and active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)
Standard of Care
Clinical Study ID
Ages > 16 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Participants must have a documented pathogenic or likely pathogenic activatingvariant, 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 thethiazide is being used as an antihypertensive, alternative therapy will beprescribed by the Investigator as needed.
Participants treated with phosphate binders (other than calcium salts) mustdiscontinue the phosphate binders at least one day prior to the SoC OptimizationVisit 1.
Participants treated with magnesium or potassium supplements must be willing todiscontinue such treatment prior to the first dose of encaleret.
Participants treated with potassium-sparing diuretics must be willing to discontinuesuch treatment prior to the first dose of encaleret.
Participants must meet SoC Optimization criteria as defined in the protocol.
Exclusion
Key Exclusion Criteria:
History of hypocalcemic seizure within the past 3 months preceding Screening.
History of thyroid or parathyroid surgery.
History of renal transplantation.
Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positivebeta-human chorionic gonadotropin (β-hCG) laboratory test.
History of treatment with parathyroid hormone (PTH) 1-84 or 1-34 within the 2 monthspreceding Screening and requiring SoC doses exceeding >1.2× their pre-PTH treatmenttotal 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 chronickidney disease-EPI creatinine equation refit without the race variable (chronickidney disease-EPI creatinine equation refit without the race variable [CKD-EPIcr_R]) (for participants <18 years old the Bedside Schwartz equation shouldbe used).
Participants with positive Hepatitis B surface antigen (HBsAg), Hepatitis Aimmunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test atthe Screening Visit. Participants who are in complete remission from Hepatitis Cvirus (HCV) as evidenced by sensitive assay ≥12 weeks after completion of HCVtherapy may participate in the study.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Study Design
Study Description
Connect with a study center
Royal North Shore Hospital
Saint Leonards, New South Wales 2065
AustraliaSite Not Available
Royal Brisbane and Women's Hospital
Herston, Queensland 4029
AustraliaSite Not Available
Bone Research & Education Centre
Oakville, Ontario L6M 1M1
CanadaSite Not Available
Vseobecna fakultni nemocnice v Praze
Nové Město, 128 08
CzechiaSite Not Available
Aarhus University Hospital
Aarhus, 8200
DenmarkSite Not Available
CHU Bicetre
Le Kremlin-Bicêtre, 94270
FranceSite Not Available
Hôpital Edouard Herriot - HCL
Lyon, 69003
FranceSite Not Available
IRCCS Ospedale San Raffaele
Milano, 20132
ItalySite Not Available
University Hospital of Pisa
Pisa, 56124
ItalySite Not Available
Fondazione Policlinico Universitario Campus Bio-Medico
Roma, 00128
ItalySite Not Available
Osaka University Hospital
Osaka, 565-0871
JapanSite Not Available
The University of Tokyo Hospital
Tokyo, 113-8655
JapanSite Not Available
Eramus MC
Rotterdam, 3015 AA
NetherlandsSite Not Available
National Cheng Kung University Hospital
Tainan, 70428
TaiwanSite Not Available
Freeman Hospital
Newcastle Upon Tyne, NE7 7DN
United KingdomSite Not Available
Norfolk and Norwich University Hospital
Norwich, NR4 7UY
United KingdomSite Not Available
UCSF Benioff Children's Hospital, Oakland
Oakland, California 94609
United StatesSite Not Available
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesSite Not Available
University of Chicago - Medical Center
Chicago, Illinois 60637
United StatesSite Not Available
Indiana University Health University Hospital
Indianapolis, Indiana 46202
United StatesSite Not Available
Johns Hopkins Bayview Medical Center
Baltimore, Maryland 21287
United StatesSite Not Available
NIH
Bethesda, Maryland 20892
United StatesSite Not Available
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesActive - Recruiting
Massachusetts General Hospital
Boston, Massachusetts 02114
United StatesSite Not Available
Mayo Clinic - Rochester
Rochester, Minnesota 55905
United StatesSite Not Available
Columbia University Irving Medical Center
New York, New York 10032
United StatesSite Not Available
Physicians East
Greenville, North Carolina 27834
United StatesSite Not Available
Ohio State University Medical Center
Columbus, Ohio 43203
United StatesSite Not Available
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Houston Methodist Hospital
Houston, Texas 77030
United StatesSite Not Available
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