Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Recruiting
Phase 2
Phase 3
Interventional Study
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 – 17
- Sex
- Any
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 28 (estimated)
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
* Provide written informed consent (if legally permitted), or have written informed consent from a parent/legal guardian and provide assent (where required and as appropriate per local requirements)
* Have a documented pathogenic or likely pathogenic activating variant, or v…
Contacts