Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1

NCT ID: NCT05680818

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

67 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-01-06

Study Completion Date

2029-08-31

Brief Summary

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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).

Detailed Description

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Conditions

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Autosomal Dominant Hypocalcemia (ADH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Encaleret

Participants will receive encaleret at a dose as needed based on calcium levels.

Group Type EXPERIMENTAL

Encaleret

Intervention Type DRUG

Administered as film-coated tablet for oral use

Standard of Care (SoC)

Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)

Group Type OTHER

Standard of Care

Intervention Type DIETARY_SUPPLEMENT

Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)

Interventions

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Encaleret

Administered as film-coated tablet for oral use

Intervention Type DRUG

Standard of Care

Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)

Intervention Type DIETARY_SUPPLEMENT

Other Intervention Names

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CLTX-305 Encaleret Sulfate

Eligibility Criteria

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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 history of symptoms or signs of ADH1.
3. Participants 16 to \<18 years old must have closed growth plates on hand radiograph.
4. 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.
5. 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.
6. Participants treated with magnesium or potassium supplements must be willing to discontinue such treatment prior to the first dose of encaleret.
7. Participants treated with potassium-sparing diuretics must be willing to discontinue such treatment prior to the first dose of encaleret.
8. Participants must meet SoC Optimization criteria as defined in the protocol.

Exclusion Criteria

1. History of hypocalcemic seizure within the past 3 months preceding Screening.
2. History of thyroid or parathyroid surgery.
3. History of renal transplantation.
4. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (β-hCG) laboratory test.
5. 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).
6. Blood 25-OH Vitamin D level \<25 nanograms (ng)/milliliter (mL).
7. 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).
8. 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.
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Calcilytix Therapeutics, Inc., a BridgeBio company

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Calcilytix Medical Director

Role: STUDY_DIRECTOR

Calcilytix Therapeutics, Inc., a BridgeBio company

Locations

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UCSF Benioff Children's Hospital, Oakland

Oakland, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Indiana University Health University Hospital

Indianapolis, Indiana, United States

Site Status

NIH

Bethesda, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Site Status

Columbia University Irving Medical Center

New York, New York, United States

Site Status

Physicians East

Greenville, North Carolina, United States

Site Status

Ohio State University Medical Center

Columbus, Ohio, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

Royal North Shore Hospital

Saint Leonards, New South Wales, Australia

Site Status

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Site Status

Bone Research & Education Centre

Oakville, Ontario, Canada

Site Status

Vseobecna fakultni nemocnice v Praze

New Town, , Czechia

Site Status

Aarhus University Hospital

Aarhus, , Denmark

Site Status

CHU Bicetre

Le Kremlin-Bicêtre, Le Kremlin-Bicêtre, France

Site Status

Hôpital Edouard Herriot - HCL

Lyon, , France

Site Status

IRCCS Ospedale San Raffaele

Milan, , Italy

Site Status

University Hospital of Pisa

Pisa, , Italy

Site Status

Fondazione Policlinico Universitario Campus Bio-Medico

Roma, , Italy

Site Status

The University of Tokyo Hospital

Tokyo, , Japan

Site Status

Eramus MC

Rotterdam, , Netherlands

Site Status

Freeman Hospital

Newcastle upon Tyne, , United Kingdom

Site Status

Norfolk and Norwich University Hospital

Norwich, , United Kingdom

Site Status

Countries

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United States Australia Canada Czechia Denmark France Italy Japan Netherlands United Kingdom

Other Identifiers

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CLTX-305-302

Identifier Type: -

Identifier Source: org_study_id

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