A Phase 3 Clinical Trial to Investigate the Safety and Efficacy of Palopegteriparatide at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism

NCT ID: NCT07081997

Last Updated: 2025-10-27

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-11-30
• Study Completion Date: 2027-12-31

Brief Summary

This trial has a duration of 78 weeks and will include adult participants already on treatment with palopegteriparatide at doses at or greater than 30 mcg/day. All participants will receive subcutaneous palopegteriparatide during the trial and will be individually and progressively titrated to an optimal dose at pre-specified dose levels. The primary purpose of the trial is to provide additional evidence of treatment effect and safety of palopegteriparatide at doses greater than 30 mcg/day in adults with hypoparathyroidism. The trial will be conducted in the US.

Conditions

Hypoparathyroidism; Endocrine System Diseases; Parathyroid Diseases

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Palopegteriparatide

Palopegteriparatide at doses at or greater than 30 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose

Group Type: EXPERIMENTAL

Palopegteriparatide

Intervention Type: COMBINATION_PRODUCT

Palopegteriparatide is supplied as a solution with a concentration of 0.3 mg/mL in a single-patient-use prefilled pen intended for subcutaneous injection.

Interventions

Palopegteriparatide

Palopegteriparatide is supplied as a solution with a concentration of 0.3 mg/mL in a single-patient-use prefilled pen intended for subcutaneous injection.

Intervention Type: COMBINATION_PRODUCT

Other Intervention Names

Yorvipath

Eligibility Criteria

Inclusion Criteria

1. Males and females, ≥18 years of age at the time of providing informed consent

2. Participants with postsurgical chronic hypoparathyroidism (HP), or auto-immune, genetic, or idiopathic HP, for at least 26 weeks

3. Receiving doses of palopegteriparatide at or above 30 µg/day

For individuals receiving 30 µg/day: evidence that dose is insufficient to keep serum calcium in the normal range, defined as:

Documented hypocalcemia within 12 weeks prior to Screening; and/or Standing dose of calcitriol ≥0.25 μg/day, and / or (elemental) calcium ≥1500 mg/day (e.g., calcium citrate, calcium carbonate etc.) for at least 4 weeks prior to Screening

For individuals receiving 33 µg/day or greater: no requirement for documented hypocalcemia or minimum doses of calcitriol or elemental calcium

4. Confirmation of laboratory parameters (Central and Local) within 2 weeks of screening visit:

25(OH) vitamin D levels of 20 - 80 ng/mL (49 - 200 nmol/L) and Magnesium level in the normal range, or just below the normal range i.e.: ≥1.3 mg/dL (≥0.53 mmol/L) and Albumin-adjusted or ionized sCa level in the normal range or just below the normal range

• Albumin-adjusted sCa 7.8 - 10.6 mg/dL (or 1.95 - 2.64 mmol/L)
• Ionized sCa 4.40 - 5.29 mg/dL (1.10 - 1.32 mmol/L)

5. BMI 17- 40 kg/m2 at Screening

6. If ≤25 years of age, radiological evidence of epiphyseal closure based on locally interpreted X-ray of non-dominant wrist and hand

7. eGFR ≥30 mL/min/1.73 m2 during Screening using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula

Exclusion Criteria

1. Impaired responsiveness to PTH (pseudohypoparathyroidism), 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 HP

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)

4. Use of thiazide diuretic within 4 weeks prior to the 24-hour urine collection scheduled to occur within 1 week prior to Visit 1

5. Use of PTH-like drugs other than palopegteriparatide (whether commercially available or through participation in an investigational trial), including PTH(1-34), or other N-terminal fragments, analogs of PTH or PTH-related protein, or PTH1R biased agonists within 4 weeks prior to Screening

6. Use of drugs known to influence calcium and bone metabolism within 12 weeks prior to Screening

7. Use of osteoporosis therapies other than bisphosponate known to influence calcium and bone metabolism within 2 years prior to Screening. Note: use of bisphosphonate (oral or intravenous [IV] is not exclusionary

8. Non-hypocalcemic seizure disorder with occurrence of a seizure within 26 weeks prior to Screening.

9. Increased risk for osteosarcoma

10. Women who are pregnant, intend to become pregnant, or are lactating

11. Male who has a female partner who intends to become pregnant or is of childbearing potential and is unwilling to use adequate contraceptive methods during the trial

12. Diagnosed drug or alcohol dependence within 3 years prior to Screening

13. Chronic or severe cardiac disease within 26 weeks prior to Screening

14. Cerebrovascular accident within 5 years prior to Screening.

15. Within 26 weeks prior to Screening: acute colic due to nephrolithiasis, or acute gout

16. Participation in any other interventional trial in which receipt of investigational drug or device other than palopegteriparatide occurred within 8 weeks (or within 5.5 times the half-life of the investigational drug) (whichever comes first) prior to Screening.

17. Known allergy or sensitivity to PTH or any of the excipients [metacresol, mannitol, succinic acid, NaOH/(HCl)] of the investigational product

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ascendis Pharma Bone Diseases A/S

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Claus Strange

Role: STUDY_DIRECTOR

Ascendis Pharma A/S

Central Contacts

Ascendis Registry Inquiries

Role: CONTACT

asnd_registryinquiries@ascendispharma.com

+4561161658

Other Identifiers

ASND0052

Identifier Type: -

Identifier Source: org_study_id