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

A 6-Month Open-label Study Investigating the Safety and Tolerability of NPSP558, a Recombinant Human Parathyroid Hormone (rhPTH [1-84]), for the Treatment of Adults With Hypoparathyroidism - A Clinical Extension Study

This study is designed to evaluate the effect of 6-months of treatment with NPSP558 in reducing requirements for supplemental oral calcium and active vitamin D, while maintaining stable total serum calcium levels in adult subjects with hypoparathyroidism.

Study Overview

• Status: Completed
• Conditions: Hypoparathyroidism
• Intervention / Treatment: Drug: NPSP558

Detailed Description

Subjects either must have previously completed NPSP558 Study CL1-11-040 (REPLACE) including 24 weeks of active therapy and 4 weeks of follow-up to Week 28 prior to enrolling in this study or have enrolled in REPLACE and dropped out during optimization, but currently meet inclusion/exclusion criteria for REPLACE.

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 3

Contacts and Locations

Study Locations

• Hungary
  • Budapest, Hungary
    • Semmelweis University Medical School
  • Pécs, Hungary
    • University of Pécs, School of Medicine
  • Szeged, Hungary
    • University of Szeged

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

Patients who meet all the following inclusion criteria can be enrolled into this study:

1. Signed and dated informed consent form (ICF) before any study-related procedures are performed
2. 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 meet inclusion/exclusion criteria for REPLACE
3. Able to perform daily SC self-injections of study medication (or have a designee perform injection) via a multidose injection pen into the thigh
4. Willingness and ability to understand and comply with the protocol
5. Women who are: (1) postmenopausal defined as 12 months amenorrhea with appropriate serum follicle stimulating hormone (FSH) levels (> 40 IU/L); (2) surgically sterilized; OR (3) of childbearing potential with a negative pregnancy test at screening and who consent to use two acceptable methods of contraception for the duration of the study, with pregnancy testing at every scheduled visit during the treatment period . Female partners (who are of childbearing potential) of male study patients must also use acceptable forms of contraception during their partner's participation

Exclusion Criteria:

Patients who meet any of the following exclusion criteria at baseline (Visit 1) are not eligible for enrollment in this study:

1. Any condition that, in the investigator's opinion after consultation with the sponsor, would preclude the safe use of NPSP558
2. Any disease or condition in the opinion of the investigator that has a high probability of precluding the patient from completing the study or where the patient cannot or will not appropriately comply with study requirements
3. Pregnant or lactating woman

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NPSP558	Drug: NPSP558; 50, 75, 100 μg; Other Names: REPEAT

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Subjects Who Achieved the Primary Triple Endpoint at Week 24, Based on Investigator Prescribed Data.	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.	24 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Percentage Changes From Baseline in Active Vitamin D Dosages at Each Visit		24 Weeks
Mean Percentage Changes From Baseline in Oral Calcium at Each Visit		24 Weeks
Proportion of Patients Achieving the Primary Endpoint at Each Visit	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.	24 Weeks
Mean Change From Baseline in 24-hour Urine Calcium Excretion		24 Weeks

Collaborators and Investigators

• Sponsor: Shire

Publications and helpful links

General Publications

• Lakatos P, Bajnok L, Lagast H, Valkusz Z. AN OPEN-LABEL EXTENSION STUDY OF PARATHYROID HORMONE RHPTH(1-84) IN ADULTS WITH HYPOPARATHYROIDISM. Endocr Pract. 2016 May;22(5):523-32. doi: 10.4158/EP15936.OR. Epub 2015 Dec 18.

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2011
• Primary Completion (Actual): April 26, 2012
• Study Completion (Actual): April 26, 2012

Study Registration Dates

• First Submitted: September 15, 2011
• First Submitted That Met QC Criteria: October 17, 2011
• First Posted (Estimate): October 19, 2011

Study Record Updates

• Last Update Posted (Actual): June 11, 2021
• Last Update Submitted That Met QC Criteria: May 19, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Keywords: Hypoparathyroidism; PTH 1-84; Parathyroid Hormone 1-84; NPSP558
• Additional Relevant MeSH Terms: Endocrine System Diseases; Parathyroid Diseases; Hypoparathyroidism
• Other Study ID Numbers: PAR-C10-009; 2011-001265-40 (EudraCT Number)