During the first 26 weeks of the trial, participants were randomly assigned to one of two groups: one group received TransCon PTH and one group received placebo. All participants started with study drug at a dose of 18 mcg/day and were individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo were administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors knew who had been assigned to each group. After the 26 weeks, participants continued in the trial as part of a long-term extension study. During the extension, all participants received TransCon PTH, with the dose adjusted to their individual needs. This was a global trial that was conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
84
TransCon PTH drug product is supplied as a solution with a concentration of 0.3 mg PTH(1-34)/mL in a single-patient-use prefilled pen intended for subcutaneous injection.
Placebo is supplied as a solution containing the formulation buffer for TransCon PTH in a single-patient-use prefilled pen intended for subcutaneous injection.
Ascendis Pharma Investigational Site
San Francisco, California, United States
Ascendis Pharma Investigational Site
Chicago, Illinois, United States
Ascendis Pharma Investigational Site
Rochester, Minnesota, United States
Ascendis Pharma Investigational Site
Reno, Nevada, United States
Ascendis Pharma Investigational Site
New York, New York, United States
Ascendis Pharma Investigational Site
Greenville, North Carolina, United States
Ascendis Pharma Investigational Site
Austin, Texas, United States
Ascendis Pharma Investigational Site
Fort Worth, Texas, United States
Ascendis Pharma Investigational Site
Spokane, Washington, United States
Ascendis Pharma Investigational Site
Halifax, Nova Scotia, Canada
...and 11 more locations
Efficacy - Primary Endpoint During the Blinded Period
The primary endpoint was a multi-component endpoint that included the percentage of participants who met the following criteria at 26 weeks of blinded treatment: 1) albumin-adjusted serum calcium measured within 4 weeks prior to and on Week 26 visit within the normal range (8.3 to 10.6 mg/dL), and 2) independence from active vitamin D within 4 weeks prior to Week 26 visit (i.e., all daily standing dose of active vitamin D equal to zero AND use of PRN ≤7 days during the 4 weeks), and 3) independence from therapeutic doses of calcium within 4 weeks prior to Week 26 visit (i.e., average daily standing dose of elemental calcium ≤600 mg AND use of PRN doses on ≤7 days during the 4 weeks), and 4) no increase in prescribed study drug within 4 weeks prior to Week 26 visit.
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Symptom - Physical Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Symptom - Physical Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in hypoparathyroidism disease related physical symptoms.
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Symptom - Cognitive Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Symptom - Cognitive Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in hypoparathyroidism disease related cognitive symptoms.
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Physical Functioning Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Impact - Physical Functioning Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in physical functioning health-related quality of life.
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Daily Life Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Impact - Daily Life Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in daily health-related quality of life.
Time frame: 26 weeks
Change From Baseline to Week 26 in SF-36 Physical Functioning Subscale Score
Change from baseline in the 36-item Short Form Survey (SF-36) Physical Functioning subscale score, a generic health survey, at 26 weeks of treatment. The Physical Functioning subscale uses a range of 19-57.6 and values represent the change in scores from baseline. An increase in SF-36 score denotes an improvement in physical functioning health-related quality of life.
Time frame: 26 weeks
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