A Study to Test the Effects and Safety of Palopegteriparatide in Adolescents With Long-term Hypoparathyroidism
10. juli 2026 opdateret af: Ascendis Pharma A/S
A Phase 3, Multicenter, Open-Label Single-Arm Clinical Trial to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of Palopegteriparatide Administered Subcutaneously Daily in the Adolescent Population (12 Years to Less Than 18 Years of Age) With Chronic Hypoparathyroidism
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.
Studieoversigt
Status
Rekruttering
Betingelser
Intervention / Behandling
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
12
Fase
- Fase 3
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: Ascendis Registry Inquiries
- Telefonnummer: +45 61242484
- E-mail: asnd_registryinquiries@ascendispharma.com
Studiesteder
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-
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Lodz, Polen, 93-338
- Rekruttering
- Ascendis Pharma Investigational Site
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Barn
- Voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
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
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
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Eksperimentel: Palopegteriparatide, once daily
Participants will receive palopegteriparatide by subcutaneous injection for 234 weeks
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Subcutaneous injection for 234 weeks
Andre navne:
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Percentage of participants meeting the multicomponent efficacy endpoint at Week 26
Tidsramme: 26 weeks
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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
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26 weeks
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Percentage of participants meeting the multicomponent efficacy endpoint through Week 234
Tidsramme: 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.
|
234 weeks
|
|
Serum biochemistries
Tidsramme: 234 weeks
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Change from baseline and normalization of serum calcium, mmol/L
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234 weeks
|
|
Serum biochemistries
Tidsramme: 234 weeks
|
Change from baseline and normalization of serum phosphate, mmol/L
|
234 weeks
|
|
Serum biochemistries
Tidsramme: 234 weeks
|
Change from baseline and normalization of serum creatinine, mL/min
|
234 weeks
|
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Renal calcifications
Tidsramme: 234 weeks
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Incidence of nephrolithiasis or nephrocalcinosis on renal ultrasound
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234 weeks
|
|
Hospitalizations/emergency room (ER)/urgent care visits
Tidsramme: 234 weeks
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Number of hospitalizations/ER/urgent care visits
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234 weeks
|
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Adverse events
Tidsramme: 234 weeks
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Percentage of participants experiencing an adverse event
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234 weeks
|
|
Bone mineral density
Tidsramme: 234 weeks
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Bone mineral density measured by dual-energy x-ray absorptiometry (DXA)
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234 weeks
|
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Bone turnover marker
Tidsramme: 234 weeks
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Qualitative evaluation of bone turnover marker ctx (ng/L) in blood sample
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234 weeks
|
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Bone turnover marker
Tidsramme: 234 weeks
|
Qualitative evaluation of bone turnover marker p1np (ng/mL) in blood sample
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234 weeks
|
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HPES-Symptom score
Tidsramme: 234 weeks
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Hypoparathyroidism Patient Experience Scale (HPES) Symptom score, including Physical and Cognitive Domains
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234 weeks
|
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HPES-Impact score
Tidsramme: 234 weeks
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Hypoparathyroidism Patient Experience Scale (HPES) Impact score, including Physical Functioning and Daily Life Domains
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234 weeks
|
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Urine biochemistries
Tidsramme: 234 weeks
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Urine biochemistries to include 24-hour urine calcium
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234 weeks
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Efterforskere
- Studieleder: Medical Director, MD, Ascendis Pharma A/S
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
22. april 2026
Primær færdiggørelse (Anslået)
1. august 2027
Studieafslutning (Anslået)
1. august 2031
Datoer for studieregistrering
Først indsendt
23. juni 2026
Først indsendt, der opfyldte QC-kriterier
10. juli 2026
Først opslået (Faktiske)
16. juli 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
16. juli 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
10. juli 2026
Sidst verificeret
1. juli 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- ASND0035
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