CALIBRATE: A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Status: Active_not_recruiting
Location: See all (24) locations...
Intervention Type: Drug, Dietary supplement
Study Type: Interventional
Study Phase: Phase 3
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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
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• 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.

Locations
United States
California
UCSF Benioff Children's Hospital, Oakland
Oakland
Colorado
Children's Hospital Colorado
Aurora
Indiana
Indiana University Health University Hospital
Indianapolis
Massachusetts
Massachusetts General Hospital
Boston
Maryland
NIH
Bethesda
Minnesota
Mayo Clinic - Rochester
Rochester
North Carolina
Physicians East
Greenville
New York
Columbia University Irving Medical Center
New York
Ohio
Ohio State University Medical Center
Columbus
Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia
Texas
Houston Methodist Hospital
Houston
Other Locations
Australia
Royal Brisbane and Women's Hospital
Herston
Royal North Shore Hospital
Saint Leonards
Canada
Bone Research & Education Centre
Oakville
Denmark
Aarhus University Hospital
Aarhus
France
CHU Bicetre
Le Kremlin-bicêtre
Hôpital Edouard Herriot - HCL
Lyon
Italy
IRCCS Ospedale San Raffaele
Milan
University Hospital of Pisa
Pisa
Fondazione Policlinico Universitario Campus Bio-Medico
Roma
Japan
The University of Tokyo Hospital
Tokyo
Netherlands
Eramus MC
Rotterdam
United Kingdom
Freeman Hospital
Newcastle Upon Tyne
Norfolk and Norwich University Hospital
Norwich
Time Frame
Start Date: 2023-01-06
Completion Date: 2029-08
Participants
Target number of participants: 67
Treatments
Experimental: Encaleret
Participants will receive encaleret at a dose as needed based on calcium levels.
Other: Standard of Care (SoC)
Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)
Related Therapeutic Areas
Sponsors
Leads: Calcilytix Therapeutics, Inc., a BridgeBio company

This content was sourced from clinicaltrials.gov

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