Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Conditions

• Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Eligibility Criteria

Sex

ALL

Age

0 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Encaleret — DRUG
  Oral tablets, age-appropriate pediatric formulation (currently under development).

Study Details

The overall objective of this study is to evaluate the pharmacokinetics (PK), efficacy, and safety of encaleret in pediatric participants from birth to 17 years of age with ADH1.

Key Dates

Start date

Jan 30, 2026

Status verified

Mar 2026

Primary completion

Dec 31, 2028

Completion

Dec 31, 2030

Study Design

Enrollment

28 participants (estimated)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Encaleret
  Participants will receive encaleret dosing in Period 1 (6 days), Period 2 (20 weeks) and Period 3 (4 weeks). Following completion of Period 3, participants will have the option to enter a long-term extension (LTE) to continue encaleret for an additional approximately 24 months or until the sponsor decides to end the study, whichever occurs first.

Primary Outcome Measure

Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3 [ Time Frame: 5 days ]

Central Contacts

• Medical Information
  650-600-3610
  [email protected]

Locations (4)

Find similar trials in New Haven, CT