Study on parathyroid hormone for use in hypoparathyroidism in children 12 to less than 18 years old.

• Register: EUCTR
• Last refreshed on: 27 July 2020
• Main ID: EUCTR2015-003108-22-GB
• Date of registration: 26/10/2015
• Prospective Registration: Yes
• Primary sponsor: NPS Pharmaceuticals, Inc.
• Public title: Study on parathyroid hormone for use in hypoparathyroidism in children 12 to less than 18 years old.
• Scientific title: An Open-label, Single-dose Study to Determine the Pharmacokinetic/Pharmacodynamic Profile of Parathyroid Hormone (rDNA) Administered Subcutaneously at a Dose of 50 µg in Subjects with Hypoparathyroidism Who Are 12 to Less Than 18 Years of Age
• Date of first enrolment: 08/12/2015
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-003108-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

United Kingdom

Contacts

• Name: Hjalmar Lagast
• Affiliation: NPS Pharmaceuticals, Inc.

• Name: Hjalmar Lagast
• Affiliation: NPS Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
Parent/guardian or emancipated minor willing and able to provide informed consent, and minor (non-emancipated) subject willing and able to provide informed assent prior to any study-related procedures

Subjects aged 12 to less than 18 years inclusive at the final visit (Study Day 3)

Completed the screening process within 90 days prior to dosing

Body weight > 30 kg

Hemoglobin level = 11 g/dL

History of hypoparathyroidism for at least 6 months post-diagnosis, inclusive of historical biochemical evidence of hypocalcemia with concomitant serum intact parathyroid hormone (PTH) concentrations below the lower limit of the laboratory normal range

Subjects who are treated with both oral calcium and active vitamin D must require oral calcium treatment over and above normal dietary calcium intake = 500 mg/day and active vitamin D therapy at a dose of = 0.5 µg/day for calcitriol or = 1 µg/day for alphacalcidol.

If a subject does not take oral calcium, the active vitamin D dosage must be
= 1 µg/day for calcitriol or = 2 µg/day for alphacalcidol.

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Participation in any other investigational drug study in which receipt of investigational drug occurred within 6 months prior to study entry.

Presence or history of a clinically significant disorder involving the cardiovascular, respiratory, renal, gastrointestinal, immunologic, hematologic, endocrine or neurologic system(s) or psychiatric disease as determined by the clinical investigator(s)

Known history of hypoparathyroidism resulting from an activating mutation in the calcium-sensing receptor (CaSR) gene or impaired responsiveness to PTH (pseudohypoparathyroidism)

Any disease that might affect calcium metabolism or calcium-phosphate homeostasis other than hypoparathyroidism such as active hyperthyroidism; Paget's disease; poorly controlled insulin-dependent diabetes mellitus; severe and chronic cardiac, liver or renal disease; Cushing's syndrome; neuromuscular disease such as rheumatoid arthritis; myeloma; pancreatitis; malnutrition; rickets; recent prolonged immobility; active malignancy, bone metastases or history of skeletal malignancies; primary or secondary hyperparathyroidism; a history of parathyroid carcinoma; hypopituitarism; acromegaly; or multiple endocrine neoplasia types I and II

Subjects who required parenteral calcium infusions (eg, calcium gluconate) to maintain calcium homeostasis within 3 months prior to enrollment




Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Chronic hypoparathyroidism

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Product Name: Parathyroid Hormone (rDNA)
Product Code: NPSP 558
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: INN Parathyroid Hormone (1-84) human
CAS Number: 68893-82-3
Current Sponsor code: NPSP 558
Other descriptive name: Parathyroid Hormone (1-84) Human Recombinant
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 50-

Primary Outcome(s)

Main Objective: The primary objective of this study is to assess the pharmacokinetic (PK)
profile of parathyroid hormone (1-84), after administration of a single
subcutaneous (SC) dose of 50 µg parathyroid hormone (rDNA) in
subjects with hypoparathyroidism aged
12 to less than 18 years.

Primary end point(s): 1) Pharmacokinetics parameters of PTH (1-84), including, e.g. Cmax and AUC
2) Serum calcium.

Secondary Objective: The secondary objective of this study is to assess safety and tolerability
of parathyroid hormone (rDNA) administration in this population.

Timepoint(s) of evaluation of this end point: 1) 5 minutes, 1.5 and 4 hours after injection
2) 1.5, 4, 8, 12 and 24 hours after single PTH (1-84) injection.

Secondary Outcome(s)

Secondary end point(s): Pharmacodynamics responses to recombinant parathyroid hormone including albumen, albumen corrected total serum calcium, magnesium, phosphate, 1, 25-dihydroxyvitamin D and creatinine. Safety and tolerability assessments.

Timepoint(s) of evaluation of this end point: Pharmacodynamics responses will be determined at selected timepoints from: 0, 1.5, 4, 8, 12 and 24 hours after dosing.

Secondary ID(s)

PAR-C14-007

Source(s) of Monetary Support

NPS Pharmaceuticals, Inc.

Ethics review

Status: Approved
Approval date: 08/12/2015
Contact: