Safety, Tolerability, and Efficacy of Encaleret in Participants With Autosomal Dominant Hypocalcemia (ADH) Type 1

NCT ID: NCT04581629

Last Updated: 2024-11-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-09-20
• Study Completion Date: 2023-09-07

Brief Summary

The primary purpose of this study is to evaluate the safety, tolerability and effectiveness of encaleret in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).

Conditions

Autosomal Dominant Hypocalcemia (ADH); Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1: Ascending + Steady-State Dose

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.

Group Type: EXPERIMENTAL

Encaleret

Intervention Type: DRUG

Tablets administered orally

Cohort 2: Steady-State Dose

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.

Group Type: EXPERIMENTAL

Encaleret

Intervention Type: DRUG

Tablets administered orally

Interventions

Encaleret

Tablets administered orally

Intervention Type: DRUG

Other Intervention Names

CLTX-305

Eligibility Criteria

Inclusion Criteria

• Be able to understand and sign a written informed consent or assent form, which must be obtained prior to initiation of study procedures.
• Postmenopausal women are allowed to participate in this study
• Body mass index (BMI) ≥ 18.5 to < 39 kilograms (kg)/square meter (m^2)
• Have an activating mutation of the Calcium-sensing receptor (CASR) gene
• Participants being treated with thiazide diuretics may be enrolled if they are willing and able to discontinue thiazides
• Participants being treated with strong Cytochrome P3A4 (CYP3A4) inhibitors should ideally, if clinically appropriate, discontinue these medications during the screening period
• Participants being treated with magnesium or potassium citrate supplements should discontinue such treatment starting on Day -1 during Period 1 and Period 2 and may be asked to discontinue treatment during Period 3

Exclusion Criteria

• History of treatment with parathyroid hormone (PTH) 1-84 or 1-34 within the previous 3 months
• History of hypocalcemic seizure within the past 3 months
• Blood 25-OH Vitamin D level < 25 nanograms (ng)/milliliter (mL)
• Participants with hemoglobin (Hgb) < 13 grams (g)/deciliter (dL) for men and < 12 g/dL for women
• Estimated glomerular filtration rate (eGFR) < 25 mL/minute/1.73 m^2 using Chronic Kidney Disease Epidemiology Collaboration (for participants <18 years old the Schwartz equation will be calculated)
• 12-lead resting electrocardiogram (ECG) with clinically significant abnormalities
• Participants with positive hepatitis B surface antigen (HBsAg), hepatitis A immunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test results at the Screening Visit
• Pregnant or nursing (lactating) women
• History of drug or alcohol dependency within 12 months preceding the Screening Visit
• History of thyroid or parathyroid surgery
• Current participation in other investigational drug studies
• Unwillingness to refrain from blood donation within 12 weeks prior to Screening Visit from the start of the study enrollment through one year after the last dose of the study drug

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Calcilytix Therapeutics, Inc., a BridgeBio company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Calcilytix Medical Director

Role: STUDY_DIRECTOR

Calcilytix Therapeutics, Inc., a BridgeBio company

Locations

National Institutes of Health (NIH) Clinical Center

Bethesda, Maryland, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CLTX-305-201

Identifier Type: -

Identifier Source: org_study_id