Safety, Tolerability, and Efficacy of CLTX-305 in Participants With Autosomal Dominant Hypocalcemia (ADH) Type 1

• Status: Active, not recruiting
• Trial end date: 2024-06-01
• Target enrollment: 0
• Participant gender: All

Summary

A Phase 2b open label, dose finding study to evaluate the Safety, Tolerability and Efficacy of CLTX-305 (encaleret) in Autosomal Dominant Hypocalcemia (ADH) Type 1

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Calcilytix Therapeutics, Inc.; Calcilytix Therapeutics, Inc., a BridgeBio company

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.

• Age ≥ 16 years

• Postmenopausal women are allowed to participate in this study

• Body mass index (BMI) ≥ 18.5 to < 39 kg/m2

• Have an activating mutation of the 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 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 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 ng/mL

• Participants with hemoglobin (Hgb) < 13 g/dL for men and < 12 g/dL for women

• Estimated glomerular filtration rate (eGFR) < 25 mL/minute/1.73 m2 using CKD-EPI (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