Actively Recruiting
Efficacy and Safety of mRNA Drug XH02 in the Treatment of Adult Hypoparathyroidism
Led by Peking Union Medical College Hospital · Updated on 2026-05-22
6
Participants Needed
1
Research Sites
2 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety and effectiveness of a new mRNA-based drug called XH02 for adults with hypoparathyroidism, a condition where the body does not produce enough parathyroid hormone (PTH). The study focuses on patients with chronic hypoparathyroidism caused by surgery, autoimmune, genetic, or unknown reasons, who have difficulty managing their condition with standard treatments. This early phase 1 trial is sponsored by Peking Union Medical College Hospital to better understand this novel therapy's potential. The investigational treatment involves intravenous administration of a drug called PTH1-84 mRNA at different doses ranging from 20 micrograms to 160 micrograms. Participants will receive one of several dosages of this mRNA drug, which is designed to produce PTH after being administered. The study is non-randomized and unblinded, meaning all participants know the treatment they receive. The treatment period includes administration and monitoring, followed by a 90-day follow-up to observe effects and safety. During the study, participants will undergo various assessments, including blood tests measuring PTH levels, calcium, magnesium, phosphorus, vitamin D, and bone turnover markers at multiple time points before and up to 72 hours after administration. Urine tests will also monitor calcium excretion. Safety is closely monitored throughout the entire study period, including the screening, treatment, and follow-up phases. Participants will be observed for adverse events and serious adverse events to evaluate the treatment's safety profile.
CONDITIONS
Brief Title
Efficacy and Safety of XH02 for the Treatment of Hypoparathyroidism
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 65 years, male or female
- Documented chronic hypoparathyroidism for at least 26 weeks due to surgery, autoimmune, genetic, or idiopathic causes
- Diagnosis confirmed by history of low calcium and low parathyroid hormone levels
- Poor control or intolerance to conventional therapy with calcium and active vitamin D
- Body Mass Index (BMI) between 17 and 40 kg/m² at screening
- For participants aged 25 years or younger, radiological evidence of closed bone growth plates by X-ray of the non-dominant hand
You will not qualify if you...
- Pseudohypoparathyroidism with elevated PTH despite low calcium
- Known allergy to the investigational drug or polyethylene glycol (PEG)-containing medications
- Other diseases affecting calcium or parathyroid hormone levels, including active hyperthyroidism, Paget's disease, severe hypomagnesemia, uncontrolled diabetes, severe liver or kidney disease, Cushing's syndrome, multiple myeloma, pancreatitis, malnutrition, rickets, recent immobilization, active malignancy (except low-risk thyroid or skin cancer), active hyperparathyroidism, recent parathyroid carcinoma, acromegaly, or multiple endocrine neoplasia
- Vaccination within 4 weeks before enrollment or planned during the study
- Pregnant or breastfeeding women
- High-risk thyroid cancer needing TSH suppression or history of malignancy
- Long-term use of certain medications including diuretics, phosphate binders (except calcium), digoxin, lithium, methotrexate, high-dose biotin, or systemic corticosteroids (except replacement therapy)
- Use of PTH-like drugs within 4 weeks before screening
- Participation in another interventional trial within 8 weeks before screening
- Uncontrolled hypertension or serious cardiovascular and cerebrovascular diseases
- Increased risk of osteosarcoma or history of extensive bone radiation
- Abnormal lab results including low neutrophils, low platelets, low hemoglobin, high eosinophils, abnormal liver or kidney function
- Any condition that may interfere with study conduct or safety as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration with assessments up to 72 hours after dosing
Participants receive intravenous administration of PTH1-84 mRNA at varying doses to treat hypoparathyroidism.
1 baseline visit and multiple assessments within 72 hours post-administration
Duration - Up to 90 days after administration
Participants are monitored for safety and adverse events for up to 90 days after treatment.
Approximately 3 follow-up visits
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, China, 100730
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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