This trial will enroll adolescents between ages of ≥12 and \<18 years with clinically diagnosed hypoparathyroidism . The purpose of the study is to see how well treatment with once-daily palopegteriparatide works and how safe it is. At least 12 participants will receive palopegteriparatide for 234 weeks. This trial will be conducted in Europe.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Subcutaneous injection for 234 weeks
Ascendis Pharma Investigational Site
Bron, France
RECRUITINGAscendis Pharma Investigational Site
Le Kremlin-Bicêtre, France
RECRUITINGAscendis Pharma Investigational Site
Lodz, Poland
RECRUITINGPercentage of participants meeting the multicomponent efficacy endpoint at Week 26
The multi-component endpoint is the percentage of participants who met the following criteria at Week 26: 1) albumin-adjusted serum calcium within the normal range ; 2) independence from active vitamin D, and 3) independence from therapeutic doses of calcium
Time frame: 26 weeks
Percentage of participants meeting the multicomponent efficacy endpoint through Week 234
The multi-component endpoint is the percentage of participants who met the following criteria: 1) albumin-adjusted serum calcium within the normal range (8.3-10.6 mg/dL) 2) independence from active vitamin D; and 3) independence from therapeutic doses of calcium.
Time frame: 234 weeks
Serum biochemistries
Change from baseline and normalization of serum calcium, mmol/L
Time frame: 234 weeks
Serum biochemistries
Change from baseline and normalization of serum phosphate, mmol/L
Time frame: 234 weeks
Serum biochemistries
Change from baseline and normalization of serum creatinine, mL/min
Time frame: 234 weeks
Renal calcifications
Incidence of nephrolithiasis or nephrocalcinosis on renal ultrasound
Time frame: 234 weeks
Hospitalizations/emergency room (ER)/urgent care visits
Number of hospitalizations/ER/urgent care visits
Time frame: 234 weeks
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Adverse events
Percentage of participants experiencing an adverse event
Time frame: 234 weeks
Bone mineral density
Bone mineral density measured by dual-energy x-ray absorptiometry (DXA)
Time frame: 234 weeks
Bone turnover marker
Qualitative evaluation of bone turnover marker ctx (ng/L) in blood sample
Time frame: 234 weeks
Bone turnover marker
Qualitative evaluation of bone turnover marker p1np (ng/mL) in blood sample
Time frame: 234 weeks
HPES-Symptom score
Hypoparathyroidism Patient Experience Scale (HPES) Symptom score, including Physical and Cognitive Domains
Time frame: 234 weeks
HPES-Impact score
Hypoparathyroidism Patient Experience Scale (HPES) Impact score, including Physical Functioning and Daily Life Domains
Time frame: 234 weeks
Urine biochemistries
Urine biochemistries to include 24-hour urine calcium
Time frame: 234 weeks