mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease.
Jingsong Cao, Minjung Choi, Eleonora Guadagnin...
https://pubmed.ncbi.nlm.nih.gov/34035281Actively Recruiting
Phase 1
Phase 2
Age: 18Years - 65Years
All Genders
ID07530705
Efficacy and Safety of Subcutaneous Injection of mRNA Nucleic Acid Drug XH-02 in the Treatment of Adult Hypoparathyroidism
Led by Peking Union Medical College Hospital · Updated on 2026-04-15
15
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating the safety and effectiveness of a new mRNA-based drug called XH-02 for adults with hypoparathyroidism, a condition where the body does not produce enough parathyroid hormone (PTH). Previous animal studies and an intravenous clinical trial showed promising results with favorable safety. This study focuses on evaluating XH-02 when given by subcutaneous injection to provide PTH replacement therapy for patients whose condition is not well controlled by conventional treatments. Participants will receive different doses of XH-02 through subcutaneous injections. Some will get a single dose of 40, 80, or 120 micrograms, while others will receive daily injections of 20, 40, 60, or 80 micrograms for five consecutive days. The study is non-randomized and open-label, meaning all participants know the treatment they receive. The trial is sponsored by Peking Union Medical College Hospital and includes both Phase 1 and Phase 2 stages. During the study, participants will be monitored for adverse events from the first dose through 30 days after the last dose for mild events, and up to three months for more serious events. Researchers will measure hormone levels including PTH, calcium, phosphorus, magnesium, and vitamin D metabolites at multiple time points before and after dosing. Urine calcium and calcium excretion will also be assessed. These evaluations will help determine the drug's effects and safety over the study period, which runs until June 2026.
CONDITIONS
Brief Title
Efficacy and Safety of Subcutaneous Injection of XH-02 in the Treatment of Adult Hypoparathyroidism
Who Can Participate
Age: 18Years - 65Years
All Genders
Eligibility Criteria
You may qualify if you...
- Aged between 18 and 65 years, male or female.
- History of postoperative chronic hypoparathyroidism or autoimmune, genetic, or idiopathic hypoparathyroidism for at least 26 weeks.
- Diagnosis confirmed by prior hypocalcemia with low serum parathyroid hormone (PTH) levels.
- Inadequate control of hypoparathyroidism with conventional calcium and vitamin D treatment or intolerance to such treatments.
- Body Mass Index (BMI) between 17 and 40 kg/m² at screening.
- For participants 25 years or younger, radiographic evidence of epiphyseal closure on wrist and hand X-rays.
You will not qualify if you...
- Impaired response to PTH, such as pseudohypoparathyroidism.
- Allergies or history of allergy to the investigational drug or polyethylene glycol (PEG).
- Other diseases affecting calcium metabolism or PTH levels, including active hyperthyroidism, Paget's disease, severe hypomagnesemia, diabetes with poor control, severe liver or kidney disease, Cushing's syndrome, multiple myeloma, active pancreatitis, malnutrition, rickets, recent immobilization, active malignancy (except some thyroid or skin cancers), active hyperparathyroidism, history of parathyroid cancer within 5 years, acromegaly, or multiple endocrine neoplasia.
- Recent vaccination within 4 weeks before enrollment or planned vaccination during the study.
- Pregnant or breastfeeding women.
- High-risk thyroid cancer needing TSH suppression recently or history of malignancy.
- Long-term use of certain medications including diuretics, phosphate binders (except calcium), digoxin, lithium, methotrexate, high-dose biotin, systemic corticosteroids (except replacement therapy), hormones, or immunosuppressants.
- Use of PTH-like drugs within 4 weeks before screening.
- Participation in other interventional trials with investigational drugs within 8 weeks before screening.
- Uncontrolled hypertension or significant cardiovascular or cerebrovascular diseases.
- Increased risk of osteosarcoma due to certain bone diseases or prior radiation.
- Abnormal blood tests such as low neutrophils, platelets, or hemoglobin, elevated liver enzymes, or low kidney function.
- Any other medical or personal condition that may affect study conduct or increase risk according to the investigator's judgment.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Treatment
Duration - 1 to 5 days depending on dosing regimen
Participants receive either a single subcutaneous injection of XH-02 or daily subcutaneous injections of XH-02 for 5 consecutive days.
1 to 5 visits depending on single or multiple doses
Follow-up
Duration - Up to 3 months after last dose
Participants are monitored for safety and efficacy for up to 30 days after the last dose for non-serious adverse events, and up to 3 months after the last dose for severe adverse events.
Multiple visits during 30 days to 3 months post-treatment
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
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Research Team
S
Sanxi Ai, Doctor
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
7
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Published Research Related To This Trial
The risk of chronic kidney disease development in adult patients with chronic hypoparathyroidism treated with rhPTH(1-84): A retrospective cohort study.
Lars Rejnmark, Olulade Ayodele, Angela Lax...
https://pubmed.ncbi.nlm.nih.gov/35974422Five-year Estimated Glomerular Filtration Rate in Patients With Hypoparathyroidism Treated With and Without rhPTH(1-84).
Kristina S Chen, Elvira O Gosmanova, Gary C Curhan...
https://pubmed.ncbi.nlm.nih.gov/32738041Standards of care for hypoparathyroidism in adults: a Canadian and International Consensus.
Aliya A Khan, Christian A Koch, Stan Van Uum...
https://pubmed.ncbi.nlm.nih.gov/30540559Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial.
Aliya A Khan, Mishaela R Rubin, Peter Schwarz...
https://pubmed.ncbi.nlm.nih.gov/36271471A Randomized Double-Blind Placebo-Controlled First-In-Human Phase 1 Trial of TransCon PTH in Healthy Adults.
David B Karpf, Susanne Pihl, Sanchita Mourya...
https://pubmed.ncbi.nlm.nih.gov/32212275Association of Calcium and Phosphate Levels with Incident Chronic Kidney Disease in Patients with Hypoparathyroidism: A Retrospective Case-Control Study.
Elvira O Gosmanova, Olulade Ayodele, Kristina Chen...
https://pubmed.ncbi.nlm.nih.gov/36389126Risk of Chronic Kidney Disease and Estimated Glomerular Filtration Rate Decline in Patients with Chronic Hypoparathyroidism: A Retrospective Cohort Study.
Elvira O Gosmanova, Kristina Chen, Lars Rejnmark...
https://pubmed.ncbi.nlm.nih.gov/33687651Renal complications in patients with chronic hypoparathyroidism on conventional therapy: a systematic literature review : Renal disease in chronic hypoparathyroidism.
Elvira O Gosmanova, Pascal Houillier, Lars Rejnmark...
https://pubmed.ncbi.nlm.nih.gov/33599907Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop.
Aliya A Khan, John P Bilezikian, Maria Luisa Brandi...
https://pubmed.ncbi.nlm.nih.gov/36054621