Trial Outcomes & Findings for A Study to Investigate the Safety and Tolerability of NPSP558, for the Treatment of Adults With Hypoparathyroidism - A Clinical Extension Study in Hungary (NCT NCT01455181)

NCT ID: NCT01455181

Last Updated: 2021-06-11

Results Overview

A ≥ 50% reduction from baseline in dose of oral calcium or an oral calcium dose of ≤ 500 mg and a ≥ 50% reduction from baseline in dose of oral active vitamin D (calcitriol dose of ≤ 0.25 μg/day or alphacalcidol dose of ≤ 0.50 μg/day) and a total serum calcium concentration that was normalized or maintained compared to the baseline value and did not exceed the ULN of the central laboratory.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

24 participants

Primary outcome timeframe

24 Weeks

Results posted on

2021-06-11

Participant Flow

24 Subjects were enrolled between 8/2011 to 5/2012 at 3 Clinical sites in Hungary.

Subjects previously completed 24 weeks of therapy and 4 weeks of follow-up in the REPLACE study, or enrolled in REPLACE and dropped out during optimization, but currently met inclusion/exclusion criteria for REPLACE.

Participant milestones

Participant milestones
Measure
NPSP558
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Overall Study
STARTED
24
Overall Study
COMPLETED
24
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Investigate the Safety and Tolerability of NPSP558, for the Treatment of Adults With Hypoparathyroidism - A Clinical Extension Study in Hungary

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Age, Customized
< 45 years
6 Participants
n=5 Participants
Age, Customized
45 to 64 years
17 Participants
n=5 Participants
Age, Customized
≥ 65 years
1 Participants
n=5 Participants
Sex: Female, Male
Female
21 Participants
n=5 Participants
Sex: Female, Male
Male
3 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
24 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
24 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
Hungary
24 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 24 Weeks

A ≥ 50% reduction from baseline in dose of oral calcium or an oral calcium dose of ≤ 500 mg and a ≥ 50% reduction from baseline in dose of oral active vitamin D (calcitriol dose of ≤ 0.25 μg/day or alphacalcidol dose of ≤ 0.50 μg/day) and a total serum calcium concentration that was normalized or maintained compared to the baseline value and did not exceed the ULN of the central laboratory.

Outcome measures

Outcome measures
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Percentage of Subjects Who Achieved the Primary Triple Endpoint at Week 24, Based on Investigator Prescribed Data.
75.0 percentage of participants
Interval 53.3 to 90.2

SECONDARY outcome

Timeframe: 24 Weeks

Outcome measures

Outcome measures
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit
Week 8
-96.70 percentage of change
Standard Deviation 7.865
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit
Week 16
-96.18 percentage of change
Standard Deviation 11.521
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit
Week 24
-97.92 percentage of change
Standard Deviation 10.206
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit
Week 2
-47.08 percentage of change
Standard Deviation 31.566
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit
Week 4
-80.38 percentage of change
Standard Deviation 16.688

SECONDARY outcome

Timeframe: 24 Weeks

Population: The Intent-to-treat population, which includes all subjects who received at least one dose of study drug and had at least one efficacy measurement.

Outcome measures

Outcome measures
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit
Week 2
-8.3 percentage of change
Standard Deviation 36.20
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit
Week 4
-46.4 percentage of change
Standard Deviation 35.57
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit
Week 8
-71.5 percentage of change
Standard Deviation 33.14
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit
Week 16
-71.7 percentage of change
Standard Deviation 38.60
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit
Week 24
-76.0 percentage of change
Standard Deviation 40.48

SECONDARY outcome

Timeframe: 24 Weeks

Population: The Intent-to-treat population, which includes all subjects who received at least one dose of study drug and had at least one efficacy measurement .

A ≥ 50% reduction from baseline in dose of oral calcium or an oral calcium dose of ≤ 500 mg and a ≥ 50% reduction from baseline in dose of oral active vitamin D (calcitriol dose of ≤ 0.25 μg/day or alphacalcidol dose of ≤ 0.50 μg/day) and a total serum calcium concentration that was normalized or maintained compared to the baseline value and did not exceed the ULN of the central laboratory.

Outcome measures

Outcome measures
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Proportion of Patients Achieving the Primary Endpoint at Each Visit
Week 2
20.8 percentage of participants
Proportion of Patients Achieving the Primary Endpoint at Each Visit
Week 4
50.0 percentage of participants
Proportion of Patients Achieving the Primary Endpoint at Each Visit
Week 8
70.8 percentage of participants
Proportion of Patients Achieving the Primary Endpoint at Each Visit
Week 16
83.3 percentage of participants
Proportion of Patients Achieving the Primary Endpoint at Each Visit
Week 24
83.3 percentage of participants

SECONDARY outcome

Timeframe: 24 Weeks

Population: The Intent-to-treat population, which includes all subjects who received at least one dose of study drug and had at least one efficacy measurement.

Outcome measures

Outcome measures
Measure
NPSP558
n=24 Participants
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Mean Change From Baseline in 24-hour Urine Calcium Excretion
Week 8
-64.58 mg/24 hour
Standard Deviation 188.42
Mean Change From Baseline in 24-hour Urine Calcium Excretion
Week 24
-51.53 mg/24 hour
Standard Deviation 183.41

Adverse Events

NPSP558

Serious events: 0 serious events
Other events: 22 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
NPSP558
n=24 participants at risk
NPSP558: Recombinant Human Parathyroid hormone (rhPTH\[1-84\]) 50, 75, or 100 mcg subcutaneously daily.
Nervous system disorders
Hypoaesthesia
50.0%
12/24 • Number of events 23 • 24 Weeks
Nervous system disorders
Headache
16.7%
4/24 • Number of events 5 • 24 Weeks
Nervous system disorders
Paraesthesia
8.3%
2/24 • Number of events 3 • 24 Weeks
Metabolism and nutrition disorders
Hypercalcaemia
20.8%
5/24 • Number of events 7 • 24 Weeks
Metabolism and nutrition disorders
Hypocalcaemia
16.7%
4/24 • Number of events 4 • 24 Weeks
Metabolism and nutrition disorders
Tetany
12.5%
3/24 • Number of events 3 • 24 Weeks
Musculoskeletal and connective tissue disorders
Muscle spasms
25.0%
6/24 • Number of events 8 • 24 Weeks
Musculoskeletal and connective tissue disorders
Arthralgia
12.5%
3/24 • Number of events 3 • 24 Weeks
Musculoskeletal and connective tissue disorders
Pain in extremity
8.3%
2/24 • Number of events 2 • 24 Weeks
Gastrointestinal disorders
Hypoaesthesia oral
12.5%
3/24 • Number of events 4 • 24 Weeks
Gastrointestinal disorders
Abdominal pain upper
8.3%
2/24 • Number of events 3 • 24 Weeks
Gastrointestinal disorders
Nausea
8.3%
2/24 • Number of events 2 • 24 Weeks
Investigations
Vitamin D decreased
25.0%
6/24 • Number of events 6 • 24 Weeks
General disorders
Fatigue
16.7%
4/24 • Number of events 6 • 24 Weeks
Renal and urinary disorders
Polyuria
8.3%
2/24 • Number of events 3 • 24 Weeks
Respiratory, thoracic and mediastinal disorders
Dyspnoea
8.3%
2/24 • Number of events 2 • 24 Weeks
Skin and subcutaneous tissue disorders
Hypoaesthesia facial
8.3%
2/24 • Number of events 3 • 24 Weeks

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: OTHER