Hypoparathyroidism
Conditions
Brief summary
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.
Interventions
COMBINATION_PRODUCTPalopegteriparatide
Subcutaneous injection for 234 weeks
Sponsors
Ascendis Pharma A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label single arm multi center
Eligibility
Sex/Gender
ALL
Age
12 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* 1\. Males and females, 12 to less than 18 years of age * 2\. Participants with postsurgical chronic hypoparathyroidism, or auto-immune, genetic, or idiopathic hypoparathyroidism for at least 26 weeks * 3\. Normal levels of serum 25(OH) vitamin D and magnesium * 4\. Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 * 5\. Able to perform daily subcutaneous self-injections of palopegteriparatide (or have a caregiver to perform injections) * 6\. Body mass index (BMI) Z-score greater than -2 SDS and below + 3 SDS * 7\. Written, signed informed consent
Exclusion criteria
* 1\. Impaired responsiveness to PTH which is characterized as PTH-resistance, with elevated PTH levels in the setting of hypocalcemia * 2\. Any disease that might affect calcium metabolism or calcium-phosphate homeostasis or PTH levels other than hypoparathyroidism, such as active hyperthyroidism * 3\. Use of loop diuretics, phosphate binders (other than calcium supplements), digoxin, lithium, methotrexate, biotin \>30 µg/day, or systemic corticosteroids (other than as replacement therapy). Short course use of steroids (≤2 weeks/year) equivalent to prednisone ≤60 mg/day is permitted * 4\. Use of thiazide diuretic * 5\. Use of PTH-like drugs * 6\. Use of other drugs known to influence calcium and bone metabolism, such as calcitonin, fluoride tablets (\>0.5 mg/day), strontium, or cinacalcet hydrochloride, within 12 weeks prior to Screening * 7\. Use of osteoporosis therapies known to influence calcium and bone metabolism, i.e., bisphosphonate (oral or intravenous \[IV\]), denosumab, raloxifene, or romosozumab therapies within 2 years prior to Screening * 8\. Non-hypocalcemic seizure disorder with occurrence of a seizure within 26 weeks prior to Screening * 9\. Increased risk for osteosarcoma * 10\. Female participants who are pregnant, intend to become pregnant, or are lactating * 11\. Diagnosed drug or alcohol dependence within 3 years prior to Screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants meeting the multicomponent efficacy endpoint at Week 26 | 26 weeks | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants meeting the multicomponent efficacy endpoint through Week 234 | 234 weeks | 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. |
| Serum biochemistries | 234 weeks | Change from baseline and normalization of serum calcium, mmol/L |
| Renal calcifications | 234 weeks | Incidence of nephrolithiasis or nephrocalcinosis on renal ultrasound |
| Hospitalizations/emergency room (ER)/urgent care visits | 234 weeks | Number of hospitalizations/ER/urgent care visits |
| Adverse events | 234 weeks | Percentage of participants experiencing an adverse event |
| Bone mineral density | 234 weeks | Bone mineral density measured by dual-energy x-ray absorptiometry (DXA) |
| Bone turnover marker | 234 weeks | Qualitative evaluation of bone turnover marker ctx (ng/L) in blood sample |
| HPES-Symptom score | 234 weeks | Hypoparathyroidism Patient Experience Scale (HPES) Symptom score, including Physical and Cognitive Domains |
| HPES-Impact score | 234 weeks | Hypoparathyroidism Patient Experience Scale (HPES) Impact score, including Physical Functioning and Daily Life Domains |
| Urine biochemistries | 234 weeks | Urine biochemistries to include 24-hour urine calcium |
Countries
France, Poland
Contacts
CONTACTAscendis Registry Inquiries
STUDY_DIRECTORMedical Director, MD
Ascendis Pharma A/S
Outcome results
None listed