Efficacy - Primary Endpoint During the Blinded Period
| Group | Value (Percentage of participants) |
|---|---|
| TransCon PTH | 78.7 |
| Placebo | 4.8 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataShort name PaTHway
United States, Canada, Denmark, Germany, Hungary, Italy, Norway
1 trial · 1 completed · 1 with posted results
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Experimental
TransCon PTH
TransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose
Placebo comparator
Placebo
Placebo for TransCon PTH delivered once daily by subcutaneous injection
Outcomes the sponsor listed as primary.
Efficacy - Primary Endpoint During the Blinded Period
Time frame · 26 weeks
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.
Change From Baseline to Week 26 in HPES Symptom - Physical Domain Score
Time frame · 26 weeks
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.
Change From Baseline to Week 26 in HPES Symptom - Cognitive Domain Score
Time frame · 26 weeks
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.
Change From Baseline to Week 26 in HPES Impact - Physical Functioning Domain Score
Time frame · 26 weeks
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.
Change From Baseline to Week 26 in HPES Impact - Daily Life Domain Score
Time frame · 26 weeks
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.
Change From Baseline to Week 26 in SF-36 Physical Functioning Subscale Score
Time frame · 26 weeks
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.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (Percentage of participants) |
|---|---|
| TransCon PTH | 78.7 |
| Placebo | 4.8 |
| Group | Value (units on a scale) |
|---|---|
| TransCon PTH | -21.01 |
| Placebo | -4.81 |
| Group | Value (units on a scale) |
|---|---|
| TransCon PTH | -20.49 |
| Placebo | -6.16 |
| Group | Value (units on a scale) |
|---|---|
| TransCon PTH | -18.29 |
| Placebo | -1.01 |
| Group | Value (units on a scale) |
|---|---|
| TransCon PTH | -17.65 |
| Placebo | -0.36 |
| Group | Value (units on a scale) |
|---|---|
| TransCon PTH | 5.29 |
| Placebo | 0.12 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Double Blind: TransCon PTHTransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose during the blinded period TransCon PTH: 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. | 1 / 61 | 5 / 61 | 50 / 61 |
| Double Blind: PlaceboPlacebo for TransCon PTH delivered once daily by subcutaneous injection during the blinded period Placebo: Placebo is supplied as a solution containing the formulation buffer for TransCon PTH in a single-patient-use prefilled pen intended for subcutaneous injection. | 0 / 21 | 2 / 21 | 20 / 21 |
| Total TransCon PTH PeriodParticipants who completed the 26-week double blind treatment period in placebo and TransCon PTH groups, continued into the open-label extension period and received treatment with TransCon PTH up to Week 182. This period refers to total period of exposure to TransCon PTH. For participants randomized to TransCon PTH at enrollment, the "TransCon PTH Period" was the time from first dose of blinded TransCon PTH until final analysis in OLE period. The TransCon PTH Period for participants randomized to placebo at enrollment was the time from first exposure to TransCon PTH at the time of cross-over from placebo, until final analysis in the OLE period. | 1 / 80 | 20 / 80 | 72 / 80 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies that cite this trial.
2 studies on this page
Indexing hint: Randomized controlled trial · 2024
Rejnmark et al. 2024Advances in therapycited by 33
PMID 38691316PMC11133178DOI 10.1007/s12325-024-02843-8PUB-38691316
Indexing hint: Randomized controlled trial · 2024
Brod et al. 2024Journal of patient-reported outcomescited by 2
PMID 39136801PMC11322464DOI 10.1186/s41687-024-00757-1PUB-39136801
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