Share
Save
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Study details:
ADH1 is a rare genetic form of hypoparathyroidism. ADH1 may be passed down from affected parents to their children. The main portion of the study is divided into a Screening Period and 3 Periods followed by an optional Long-Term Extension (LTE).
The estimated duration of this main portion of the study is approximately 12 months. The duration of the LTE is approximately 48 months. Participants will enter an up-to-6-week Screening period and once confirmation of all Inclusion/Exclusion criteria transition into an up-to-15-week standard of care (SoC) optimization phase.
The eligible participants will enter Period 1 after completing the SoC optimization phase. Period 1 is the 4-week SoC Maintenance period of the study during which the SoC dose will only be adjusted to address potential safety concerns such as hypocalcemia or hypercalcemia. After completion of Period 1, eligible participants will enter Period 2 and will be randomized to receive either encaleret or SoC treatment for 20 weeks.
Both the investigator and participant will know whether the participant was randomized to the encaleret treatment arm or SoC treatment arm. During Period 2, encaleret or SoC will be adjusted based on blood calcium levels. After completion of Period 2, participants will proceed to Period 3, the 4-week dose maintenance period.
Following completion of Period 3, participants from CLTX-305-302 may enter the LTE. Those who completed CLTX-305-201 (NCT04581629) are also eligible to continue in the LTE. Participants will receive encaleret treatment for approximately 48 months, or 72 months for participants who transitioned from CLTX-305-201, or until a participant has access to commercial encaleret, or the Sponsor decides to end the study, whichever occurs first.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 16 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2023-01-06
Primary completion: 2025-04-01
Study completion finish: 2028-09-01
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT05680818
Intervention or treatment
DRUG: Encaleret
DIETARY_SUPPLEMENT: Standard of Care
Conditions
- • Autosomal Dominant Hypocalcemia (ADH)
Find a site
Closest Location:
Royal North Shore Hospital
Research sites nearby
Select from list below to view details:
Royal North Shore Hospital
Saint Leonards, New South Wales, Australia
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: Encaleret
| DRUG: Encaleret
|
OTHER: Standard of Care (SoC)
| DIETARY_SUPPLEMENT: Standard of Care
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range | * cCa within 8.3-10.7 mg/dL (2.1-2.7 millimoles per liter \[mmol/L\]) * 24-hr UCa within the reference range (\< 300 mg/day for men \[7.5 mmol/day\], \< 250 mg/day for women \[6.25 mmol/day\]) | Up to Week 24 |
Secondary outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| Number of Participants With Intact Parathyroid Hormone (iPTH) Within or Greater than the Reference Range | Not Specified | Up to Week 24 |
| Number of Participants who Achieve Blood Magnesium Within the Reference Range | Not Specified | Up to Week 24 |
| Number of Participants who Achieve Blood Phosphate Within the Reference Range | Not Specified | Up to Week 24 |
| Change From Baseline in Blood 1,25-(OH)2 Vitamin D | Not Specified | Baseline to Week 24 |
| Change From Baseline in cCa | Not Specified | Baseline to Week 24 |
| Change From Baseline in 24-hour UCa | Not Specified | Baseline to Week 24 |
| Change From Baseline in iPTH | Not Specified | Baseline to Week 24 |
| Change From Baseline in Blood Phosphate and Blood Magnesium | Not Specified | Baseline to Week 24 |
| Change From Baseline in Urine Magnesium, Phosphate, Sodium, and Citrate Handling | Not Specified | Baseline to Week 24 |
| Change From Baseline in QT Interval Corrected for Changes in the Heart Rate With Fridericia Formula (QTcF) as Assessed by Electrocardiogram (ECG) | Not Specified | Baseline to Week 24 |
| Change from Baseline in 36-Item Short Form Health Survey (SF-36) Physical Component Score and Mental Component Score and Each of the Sub-Domains | Not Specified | Baseline to Week 24 |
| Number of Participants in the Encaleret Arm Receiving Calcium and/or Vitamin D Supplements | Not Specified | Up to Week 24 |
| Steady State Encaleret Trough Concentration (Ctrough) | Not Specified | Up to Week 24 |
Frequently Asked Questions
Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol
No questions submitted. Be the first to ask a question!