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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Palopegteriparatide, once daily | Experimental | Participants will receive palopegteriparatide by subcutaneous injection for 234 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Palopegteriparatide | Combination Product | Subcutaneous injection for 234 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage 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 | 26 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 234 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ascendis Registry Inquiries | Contact | +45 61242484 | asnd_registryinquiries@ascendispharma.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | Ascendis Pharma A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ascendis Pharma Investigational Site | Recruiting | Lodz | 93-338 | Poland |
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| ID | Term |
|---|---|
| D007011 | Hypoparathyroidism |
| ID | Term |
|---|---|
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
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Open-label single arm multi center
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| Serum biochemistries | Change from baseline and normalization of serum calcium, mmol/L | 234 weeks |
| Serum biochemistries | Change from baseline and normalization of serum phosphate, mmol/L | 234 weeks |
| Serum biochemistries | Change from baseline and normalization of serum creatinine, mL/min | 234 weeks |
| Renal calcifications | Incidence of nephrolithiasis or nephrocalcinosis on renal ultrasound | 234 weeks |
| Hospitalizations/emergency room (ER)/urgent care visits | Number of hospitalizations/ER/urgent care visits | 234 weeks |
| Adverse events | Percentage of participants experiencing an adverse event | 234 weeks |
| Bone mineral density | Bone mineral density measured by dual-energy x-ray absorptiometry (DXA) | 234 weeks |
| Bone turnover marker | Qualitative evaluation of bone turnover marker ctx (ng/L) in blood sample | 234 weeks |
| Bone turnover marker | Qualitative evaluation of bone turnover marker p1np (ng/mL) in blood sample | 234 weeks |
| HPES-Symptom score | Hypoparathyroidism Patient Experience Scale (HPES) Symptom score, including Physical and Cognitive Domains | 234 weeks |
| HPES-Impact score | Hypoparathyroidism Patient Experience Scale (HPES) Impact score, including Physical Functioning and Daily Life Domains | 234 weeks |
| Urine biochemistries | Urine biochemistries to include 24-hour urine calcium | 234 weeks |