Recruiting NCT07080385
Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Encaleret.
- Who it may be relevant to
- Registry conditions: Autosomal Dominant Hypocalcemia Type 1 (ADH1). Basic parameters: 0 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2/3, Multicenter, Single-Arm, Open-Label Study Evaluating the Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Overview
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.
Interventions
- Drug Encaleret
Oral tablets, age-appropriate pediatric formulation (currently under development).
Primary outcome measures
- Period 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]
Secondary outcome measures (12)
- 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]
- 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]
Eligibility criteria
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 variant of uncertain significance of the calcium-sensing receptor (CASR), associated with biochemical findings of hypoparathyroidism at screening or a documented history of hypoparathyroidism as manifested by hypocalcemia and intact parathyroid hormone (PTH) <40 picogram per milliliter (pg/mL) (4.2 picomoles per liter \[pmol/L\])
- Have at least 1 symptom or sign of hypoparathyroidism at screening or a documented history of symptoms or signs of hypoparathyroidism
- Be on ADH1 treatment for at least 6 months before screening for cohorts 1 to 3, or for at least 3 months before screening for cohort 4
Exclusion criteria
- History of thyroid or parathyroid surgery
- History of renal transplantation
- History of cancer (except thyroid cancer, basal cell skin cancer, or squamous cell skin cancer), skeletal malignancies, bone metastases, irradiation (radiotherapy) to the skeleton, chemotherapy with alkylating agents, Paget disease, fibrous dysplasia, chronic osteomyelitis, bone infarcts, benign bone tumors with curettage and bone grafts, retinoblastoma, or Li-Fraumeni syndrome within 5 years before screening
- Received any investigational medicinal product within 30 days or 5 half-lives before Day 1, whichever is longer, or is in follow-up for another interventional clinical study during screening
- Treatment with a strong P-glycoprotein (P-gp) inhibitor within 300 days before screening for amiodarone or within 30 days before screening for any other strong P-gp inhibitor
- Treatment with cardiac glycosides, or is being breastfed while the participant's nursing mother is treated with cardiac glycosides, within 30 days before screening
- Presence or history of any disease or condition (eg, drug or alcohol dependence) that would affect the participant's safety, treatment compliance, or ability to complete the study, in the opinion of the investigator
Other protocol defined inclusion/exclusion criteria apply.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 4 centers
- Yale University — New Haven
- Children's National Medical Center — Washington D.C.
- Nemours Children's Health — Jacksonville
- The Children's Hospital of Philadelphia — Philadelphia
United Kingdom · 2 centers
- Royal London Hospital — London
- Royal Manchester Children's Hospital — Manchester
Identifiers
NCT: NCT07080385 · CLTX-305-303