Trial Outcomes & Findings for A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH in Adults With Hypoparathyroidism (NCT NCT04009291)
NCT ID: NCT04009291
Last Updated: 2025-05-08
Results Overview
The percentage of subjects with albumin-adjusted serum calcium within the normal range, and spot morning fractional excretion of calcium (spot AM FECa) within normal range (≤2%) or a reduction by at least 50% from baseline, and not taking active vitamin D supplements, and taking ≤1000 mg/day of calcium supplements
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
59 participants
Primary outcome timeframe
Week 4
Results posted on
2025-05-08
Participant Flow
A total of 104 subjects were screened and 59 of these met eligibility criteria and were enrolled into the study. The four-week blinded period is completed and the study is ongoing with all participants receiving TransCon PTH in the open-label period.
Participant milestones
| Measure |
TransCon PTH 15 mcg
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
Open-Label TransCon PTH
Subjects who completed the four-week blinded period were assigned to open-label treatment with TransCon PTH for up to 262 weeks, with up to an initial 14 weeks of TransCon PTH titration and standard of care optimization, followed by approximately 248 weeks of individualized dosing.
|
|---|---|---|---|---|---|
|
Blinded Period (Weeks 0 to 4)
STARTED
|
14
|
15
|
15
|
15
|
0
|
|
Blinded Period (Weeks 0 to 4)
COMPLETED
|
14
|
15
|
15
|
15
|
0
|
|
Blinded Period (Weeks 0 to 4)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Open-Label Period (Weeks 4 to 266)
STARTED
|
0
|
0
|
0
|
0
|
59
|
|
Open-Label Period (Weeks 4 to 266)
COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Open-Label Period (Weeks 4 to 266)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
59
|
Reasons for withdrawal
| Measure |
TransCon PTH 15 mcg
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
Open-Label TransCon PTH
Subjects who completed the four-week blinded period were assigned to open-label treatment with TransCon PTH for up to 262 weeks, with up to an initial 14 weeks of TransCon PTH titration and standard of care optimization, followed by approximately 248 weeks of individualized dosing.
|
|---|---|---|---|---|---|
|
Open-Label Period (Weeks 4 to 266)
Withdrawal by Subject
|
0
|
0
|
0
|
0
|
1
|
|
Open-Label Period (Weeks 4 to 266)
Study Ongoing
|
0
|
0
|
0
|
0
|
58
|
Baseline Characteristics
A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH in Adults With Hypoparathyroidism
Baseline characteristics by cohort
| Measure |
TransCon PTH 15 mcg
n=14 Participants
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
n=15 Participants
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
n=15 Participants
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
n=15 Participants
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
Total
n=59 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Continuous
|
47.03 years
STANDARD_DEVIATION 13.230 • n=5 Participants
|
46.58 years
STANDARD_DEVIATION 11.157 • n=7 Participants
|
53.67 years
STANDARD_DEVIATION 11.287 • n=5 Participants
|
51.80 years
STANDARD_DEVIATION 12.345 • n=4 Participants
|
49.82 years
STANDARD_DEVIATION 12.094 • n=21 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
48 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
58 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
White
|
14 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
54 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Height
|
166.92 cm
STANDARD_DEVIATION 8.806 • n=5 Participants
|
166.71 cm
STANDARD_DEVIATION 8.385 • n=7 Participants
|
165.37 cm
STANDARD_DEVIATION 10.961 • n=5 Participants
|
164.07 cm
STANDARD_DEVIATION 10.368 • n=4 Participants
|
165.75 cm
STANDARD_DEVIATION 9.520 • n=21 Participants
|
|
Weight
|
76.58 kg
STANDARD_DEVIATION 22.479 • n=5 Participants
|
80.04 kg
STANDARD_DEVIATION 11.279 • n=7 Participants
|
72.26 kg
STANDARD_DEVIATION 18.621 • n=5 Participants
|
76.43 kg
STANDARD_DEVIATION 14.256 • n=4 Participants
|
76.32 kg
STANDARD_DEVIATION 16.869 • n=21 Participants
|
|
Body Mass Index
|
27.08 kg/m^2
STANDARD_DEVIATION 5.723 • n=5 Participants
|
28.76 kg/m^2
STANDARD_DEVIATION 3.148 • n=7 Participants
|
26.12 kg/m^2
STANDARD_DEVIATION 4.647 • n=5 Participants
|
28.30 kg/m^2
STANDARD_DEVIATION 3.775 • n=4 Participants
|
27.57 kg/m^2
STANDARD_DEVIATION 4.415 • n=21 Participants
|
PRIMARY outcome
Timeframe: Week 4The percentage of subjects with albumin-adjusted serum calcium within the normal range, and spot morning fractional excretion of calcium (spot AM FECa) within normal range (≤2%) or a reduction by at least 50% from baseline, and not taking active vitamin D supplements, and taking ≤1000 mg/day of calcium supplements
Outcome measures
| Measure |
TransCon PTH 15 mcg
n=14 Participants
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
n=15 Participants
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
n=15 Participants
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
n=15 Participants
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
|---|---|---|---|---|
|
Efficacy - Primary Endpoint During the Blinded Period
|
50.0 Percentage of participants
Interval 23.0 to 77.0
|
40.0 Percentage of participants
Interval 16.3 to 67.7
|
60.0 Percentage of participants
Interval 32.3 to 83.7
|
26.7 Percentage of participants
Interval 7.8 to 55.1
|
SECONDARY outcome
Timeframe: Week 4The percentage of subjects with albumin-adjusted serum calcium within the normal range, and spot AM FECa within the normal range (≤2%) or a reduction by at least 50% from baseline, and not taking active vitamin D supplements, and taking ≤ 500 mg/day of calcium supplements
Outcome measures
| Measure |
TransCon PTH 15 mcg
n=14 Participants
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
n=15 Participants
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
n=15 Participants
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
n=15 Participants
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
|---|---|---|---|---|
|
Efficacy - Key Secondary Endpoint During the Blinded Period
|
50.0 Percentage of participants
Interval 23.0 to 77.0
|
26.7 Percentage of participants
Interval 7.8 to 55.1
|
60.0 Percentage of participants
Interval 32.3 to 83.7
|
20.0 Percentage of participants
Interval 4.3 to 48.1
|
Adverse Events
TransCon PTH 15 mcg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
TransCon PTH 18 mcg
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
TransCon PTH 21 mcg
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
TransCon PTH Period
Serious events: 5 serious events
Other events: 51 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
TransCon PTH 15 mcg
n=14 participants at risk
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
n=15 participants at risk
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
n=15 participants at risk
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
n=15 participants at risk
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH Period
n=59 participants at risk
The total period of exposure to TransCon PTH. For subjects randomized to TransCon PTH at enrollment, this was the time from the first dose of blinded TransCon PTH until data cutoff in the open-label extension (OLE) period. The TransCon PTH Period for subjects randomized to placebo at enrollment was the time from first exposure to TransCon PTH at the time of cross-over from placebo, until data cutoff in the OLE.
|
|---|---|---|---|---|---|
|
Nervous system disorders
Headache
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Metabolism and nutrition disorders
Hypokalemia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary tumour of renal pelvis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Chronic sinusitis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Injury, poisoning and procedural complications
Thoracic vertebral fracture
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Invasive ductal breast carcinoma
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
1.7%
1/59 • Number of events 1 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
Other adverse events
| Measure |
TransCon PTH 15 mcg
n=14 participants at risk
TransCon PTH 15 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 18 mcg
n=15 participants at risk
TransCon PTH 18 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH 21 mcg
n=15 participants at risk
TransCon PTH 21 mcg delivered once daily by subcutaneous injection
TransCon PTH: TransCon PTH drug product is supplied as a clear solution containing TransCon PTH with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.
|
Placebo
n=15 participants at risk
Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection
Placebo for TransCon PTH: Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection.
|
TransCon PTH Period
n=59 participants at risk
The total period of exposure to TransCon PTH. For subjects randomized to TransCon PTH at enrollment, this was the time from the first dose of blinded TransCon PTH until data cutoff in the open-label extension (OLE) period. The TransCon PTH Period for subjects randomized to placebo at enrollment was the time from first exposure to TransCon PTH at the time of cross-over from placebo, until data cutoff in the OLE.
|
|---|---|---|---|---|---|
|
Nervous system disorders
Headache
|
21.4%
3/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
22.0%
13/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Nervous system disorders
Dizziness
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
13.3%
2/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Gastrointestinal disorders
Nausea
|
14.3%
2/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
10.2%
6/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
13.3%
2/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
General disorders
Fatigue
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
11.9%
7/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
General disorders
Injection site pain
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
General disorders
Thirst
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Vascular disorders
Hypertension
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
10.2%
6/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Urinary tract infection
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
11.9%
7/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
10.2%
6/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
8.5%
5/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Sinusitis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.8%
4/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
5.1%
3/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
5.1%
3/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
5.1%
3/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Cardiac disorders
Palpitations
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
5.1%
3/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
General disorders
Asthenia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
COVID-19
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Reproductive system and breast disorders
Cervical dysplasia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Ear infection
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Reproductive system and breast disorders
Heavy menstrual bleeding
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Eye disorders
Keratitis
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Reproductive system and breast disorders
Uterine haemorrhage
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Injury, poisoning and procedural complications
Limb injury
|
7.1%
1/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
General disorders
Pyrexia
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
|
Investigations
Weight increased
|
0.00%
0/14 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
6.7%
1/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
0.00%
0/15 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
3.4%
2/59 • 4 Week Blinded Period and Open-Label Period starting at Week 4 up to Week 84
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place