PALOPEGTERIPARATIDE for Chronic hypoparathyroidism

Inclusion criteria

• {"criterion_text":"- Males and females, 12 to less than 18 years of age at the time of screening (defined as the date of signing informed consent).\n- Participants with postsurgical chronic hypoparathyroidism, or auto-immune, genetic, or idiopathic hypoparathyroidism for at least 26 weeks. Diagnosis of hypoparathyroidism is established based on historic hypocalcemia in the setting of inappropriately low serum PTH levels (Hypocalcemia is defined as a value below the reference range for normal at the performing laboratory. Inappropriately low serum PTH levels are defined as at or below the median value of the reference range for normal at the performing laboratory while the concomitant serum calcium is low. If specific lab results at the time of original diagnosis are not available, as historical diagnosis affirming these two components is adequate for inclusion).\n- Prior to randomization, achieve normal levels of serum 25(OH) vitamin D and magnesium.\n- Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 prior to randomization utilizing the 2009 Schwartz equation: eGFR (mL/min/1.73m^2)= 36.5*((Height (cm))/(Serum Creatinine (µmol/L)))\n- Body mass index (BMI) Z-score greater than -2 SDS and below + 3 SDS at Screening.\n- Written, signed informed consent provided by parent(s) or legally acceptable representative(s) of the participant, and by the participant if in accordance to local health authority/ethics requirements. Assent from participant obtained in accordance with applicable regulatory requirements.\n- Written, signed informed consent provided by parent(s) or legally acceptable representative(s) of the participant, and by the participant if in accordance to local health authority/ethics requirements. Assent from participant obtained in accordance with applicable regulatory requirements."}

Exclusion criteria

• {"criterion_text":"- Impaired responsiveness to PTH which is characterized as PTH-resistance, with elevated PTH levels in the setting of hypocalcemia.\n- Female participants who are pregnant, intend to become pregnant, or are lactating. Note: Acceptable, effective contraception is required for sexually active women of childbearing potential during the trial and for 2 weeks after the last dose of investigational product.\n- Diagnosed drug or alcohol dependence within 3 years prior to Screening.\n- Symptomatic or severe cardiac disease within 26 weeks prior to Screening including but not limited to congestive heart failure, symptomatic or severe valvular disease, myocardial infarction, severe or uncontrolled arrhythmias, bradycardia, symptomatic hypotension, abnormal systolic BP, or poorly controlled hypertension based on age and sex-specific reference ranges.\n- Cerebrovascular accident within 5 years prior to Screening.\n- Within 26 weeks prior to Screening: acute colic due to nephrolithiasis or acute gout. Note: Participants with asymptomatic renal stones are permitted.\n- Participation in any other interventional trial in which receipt of investigational product or device occurred within 8 weeks (or within 5.5 times the half-life of the investigational product) (whichever comes first) prior to Screening.\n- Known allergy or sensitivity to PTH or any of the excipients [metacresol, mannitol, succinic acid, NaOH/(HCl)] of the investigational product.\n- Any other reason that in the opinion of the investigator would prevent the participant from completing participation or following the trial schedule.\n- Any disease that might affect calcium metabolism or calcium-phosphate homeostasis or PTH levels other than hypoparathyroidism, such as active hyperthyroidism; Paget disease of bone; severe hypomagnesemia; type 1 diabetes mellitus or poorly controlled type 2 diabetes mellitus (HbA1C >9%, documented HbA1C result drawn within 12 weeks prior to Screening is acceptable); severe and chronic liver, or renal disease; Cushing syndrome; multiple myeloma; active pancreatitis; malnutrition; rickets; recent prolonged immobility; active malignancy (other than low-risk well differentiated thyroid cancer or non-melanoma skin cancer); active hyperparathyroidism; parathyroid carcinoma within 5 years prior to Screening; acromegaly; or multiple endocrine neoplasia types 1 and 2.\n- 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.\n- Use of thiazide diuretic within 4 weeks prior to the 24-hour urine collection scheduled to occur within 1 week prior to Visit 1.\n- Use of PTH-like drugs (whether commercially available or through participation in an investigational trial), including PTH(1-84), PTH(1-34), or other N-terminal fragments or analogs of PTH or PTH-related protein, within 4 weeks prior to Screening.\n- 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.\n- 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.\n- Non-hypocalcemic seizure disorder with occurrence of a seizure within 26 weeks prior to Screening.Note: History of seizures that occur in the setting of hypocalcemia is not exclusionary.\n- Increased risk for osteosarcoma, such as those with Paget's disease of bone or unexplained elevations of alkaline phosphatase, hereditary disorders predisposing to osteosarcoma, or with a prior history of substantial external beam or implant radiation therapy involving the skeleton."}

Primary endpoints

• {"endpoint_text":"- At Week 26, proportion of participants with: Albumin-adjusted serum calcium within the normal range measured within 4 weeks prior to and on the Week 26 visit; and","definition_or_measurement_approach":"Albumin-adjusted serum calcium measured within 4 weeks prior to and on the Week 26 visit; proportion of participants meeting normal range at Week 26."}
• {"endpoint_text":"- Independence from active vitamin D and","definition_or_measurement_approach":"Assessment of whether participants are independent from active vitamin D therapy (no active vitamin D use) by the Week 26 timepoint."}
• {"endpoint_text":"- Independence from therapeutic doses of calcium. Calcium ≤600 mg/day (in the form of tablets, powder, liquid suspension, or transdermal patch) is considered as “supplemental” to meet recommended daily intake for general health, as opposed to a “therapeutic” dose to treat hypoparathyroidism","definition_or_measurement_approach":"Daily elemental calcium intake recorded; independence defined as therapeutic calcium not required and calcium intake ≤600 mg/day considered supplemental rather than therapeutic."}

France

Earliest CTIS Part Ii Submission Date

23-01-2026

Latest Decision Or Authorization Date

10-03-2026

Processing Time Days

46

Number Of Sites

2

Number Of Participants

3

Germany

Earliest CTIS Part Ii Submission Date

17-02-2026

Latest Decision Or Authorization Date

09-03-2026

Processing Time Days

20

Number Of Sites

2

Number Of Participants

3

Romania

Earliest CTIS Part Ii Submission Date

13-03-2026

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

10

Number Of Sites

2

Number Of Participants

2

Poland

Earliest CTIS Part Ii Submission Date

20-02-2026

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

31

Number Of Sites

1

Number Of Participants

3

Primary sponsor

Full Name

Ascendis Pharma Bone Diseases A/S

Organisation Type

Pharmaceutical company

Country Of Registered Address

Denmark

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Site platform solutions; other duties codes: 1, 12, 4, 5, 8

Name

Imperial Clinical Research Services International Limited

Responsibilities

Printing and supplies

Name

Scout Clinical

Responsibilities

Travel/accomodation arrangement and patient reinbursment

Name

4G Clinical B.V.

Responsibilities

IRT

Name

Clario

Responsibilities

Medical image analysis/ review - X-ray, MRI, ultrasound, ECG, etc.

Name

Cognizant Technology Solutions India Private Limited

Responsibilities

PV vendor

Third parties

• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Site platform solutions; other duties codes: 1, 12, 4, 5, 8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Clario","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, ECG, etc.","organisation_type":"Industry"}
• {"country":"India","full_name":"Cognizant Technology Solutions India Private Limited","duties_or_roles":"PV vendor","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"code 6","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"Printing and supplies","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Netherlands","full_name":"ICON Bioanalytical Laboratories","duties_or_roles":"ICON Bioanalytical Laboratories","organisation_type":"Health care"}
• {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"IRT","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel/accomodation arrangement and patient reinbursment","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Immunogenicity assessments","organisation_type":"Pharmaceutical company"}