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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
Oral tablets, age-appropriate pediatric formulation (currently under development).
Yale University
New Haven, Connecticut, United States
RECRUITINGThe Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGPeriod 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3
Time frame: 5 days
Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3
Time frame: 5 days
Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response
Time frame: Week 25
Period 1: Change from Baseline in Blood cCa
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in Blood Intact Parathyroid Hormone (iPTH) Concentration
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in Blood Phosphate Concentration
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in Blood Magnesium Concentration
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in 24-hour UCa in Toilet Trained Participants
Time frame: Baseline up to Day 5
Period 1: Change from Baseline in Spot Ratio of UCa/Urinary Creatinine (UCr) In Non-toilet Trained Participants
Time frame: Baseline up to Day 5
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Period 3: Number of Participants with Blood iPTH Within the Reference Range
Time frame: Week 25 (post-dose)
Period 3: Number of Participants with Blood Phosphate Within the Reference Range
Time frame: Week 25
Period 3: Number of Participants with Blood Magnesium Within the Reference Range
Time frame: Week 25
Period 3: Number of Participants with Blood 1,25-(OH)2 Vitamin D Within the Reference Range
Time frame: Week 25
Periods 1, 2, and 3: Change from Baseline in Short Form-10 Health Survey for Children (SF-10) in Participants Aged ≥6 Years
Time frame: Baseline up to Week 25
Periods 1, 2, and 3: Dose of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy
Time frame: 25 weeks
Periods 1, 2, and 3: Dosing Frequency of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy
Time frame: 25 weeks
Periods 1, 2, 3, and LTE: Number of Participants with Adverse Events (AEs) and Serious AEs (SAEs)
Time frame: 145 weeks
LTE: Change from Baseline in Blood cCa
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in Blood iPTH Concentration
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in Blood Phosphate Concentration
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in Blood Magnesium Concentration
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in 24-hour UCa In Toilet Trained Participants
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in Spot Ratio of UCa/UCr In Non-toilet Trained Participants
Time frame: Baseline up to 145 weeks
LTE: Change from Baseline in SF-10 in Participants Aged ≥6 Years
Time frame: Baseline up to 145 weeks