A Cohort Study on the Safety and Efficacy of XH-02 in Treating Hypoparathyroidism

May 13, 2026 updated by: Yan Qin, Peking Union Medical College Hospital

An Expanded Cohort Study on the Safety and Efficacy of mRNA Nucleic Acid Drug XH-02 in Treating Adult Hypoparathyroidism

XH-02 is an mRNA nucleic acid drug that expresses PTH in the body following intravenous or subcutaneous injection, providing PTH replacement therapy for patients with hypoparathyroidism. Previous clinical studies have demonstrated the safety of subcutaneously administered XH-02 in several patients with hypoparathyroidism and have yielded clear efficacy results. This study aims to further validate the safety and efficacy of subcutaneously injected XH-02 in the treatment of hypoparathyroidism in a expanded cohort.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Beijing, China, 100730
        • Recruiting
        • Peking Union Medical College Hospital
        • Contact:
        • Principal Investigator:
          • Yan Qin, Doctor

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥18 years, both males and females eligible;
  2. History of postoperative chronic HP or autoimmune, genetic, or idiopathic HP for at least 26 weeks. The diagnosis of HP is established based on the presence of inappropriately low serum PTH levels concurrent with hypocalcemia in the past.
  3. Poorly controlled or intolerant to conventional treatment (calcium and vitamin D) for hypoparathyroidism;
  4. BMI 17-40 kg/m² (inclusive) at screening;
  5. If age ≤25 years, radiographic evidence of epiphyseal closure based on X-ray results of the wrist and palm of the non-dominant hand.

Exclusion Criteria:

  1. Impaired PTH response (pseudohypoparathyroidism), characterized by PTH resistance and elevated PTH levels in the presence of hypocalcemia;
  2. Allergic constitution, or allergy to the investigational drug or polyethylene glycol (PEG)-based drugs;
  3. Any disease other than HP that may affect calcium metabolism, calcium-phosphorus homeostasis, or PTH levels, such as active hyperthyroidism; Paget's disease of bone; severe hypomagnesemia; type 1 diabetes mellitus or poorly controlled type 2 diabetes mellitus (HbA1C >9%; HbA1C test results from blood samples collected within 12 weeks prior to screening are acceptable); severe and chronic liver or kidney disease; Cushing's syndrome; multiple myeloma; active pancreatitis; malnutrition; rickets; recent prolonged immobilization; active malignancy (except for low-risk well-differentiated thyroid cancer or non-melanoma skin cancer); active hyperparathyroidism; parathyroid carcinoma occurring within 5 years prior to screening; acromegaly; or multiple endocrine neoplasia;
  4. Pregnant or breastfeeding women;
  5. Male partners with female partners planning to become pregnant, or partners of childbearing potential who are unwilling to use adequate contraceptive methods during the study period;
  6. Patients with high-risk thyroid cancer requiring TSH suppression to <0.2 mIU/L within 2 years, or those with a history of tumors;
  7. Use of loop diuretics, phosphate binders (except calcium supplements), digoxin, lithium, methotrexate, biotin >30 mcg/day, or systemic corticosteroids (except as replacement therapy);
  8. Use of PTH-like drugs (whether commercially available or obtained through participation in clinical trials), 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;
  9. Participation in any other interventional trial receiving investigational drugs or devices within 8 weeks prior to screening, or still within 5.5 half-lives of the investigational drug from the trial in which they participated;
  10. Presence of uncontrolled hypertension at baseline, or a history of the following cardiovascular or cerebrovascular diseases, including: (1) unstable angina; (2) cardiac arrhythmias requiring medication or severe arrhythmias; (3) myocardial infarction; (4) heart failure class III or higher (NYHA classification), second-degree or higher atrioventricular block; (5) cerebral infarction (excluding lacunar infarction), cerebral hemorrhage, or other such diseases;
  11. Increased risk of osteosarcoma, such as having Paget's disease of bone or unexplained elevated alkaline phosphatase, having genetic disorders predisposing to osteosarcoma, or having a prior history of extensive external beam or implant radiation therapy involving bone;
  12. Disease processes that adversely affect gastrointestinal absorption, including but not limited to short bowel syndrome, significant small bowel resection, gastric bypass surgery, tropical sprue, active celiac disease, active ulcerative colitis, active Crohn's disease, gastroparesis, and autoimmune regulator gene mutations associated with malabsorption;
  13. Any medical or other condition that, in the investigator's judgment, may affect the conduct of the study, interfere with the study results, or increase the risk to the subject/study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single dose: 40 μg
Participants will receive a single subcutaneous dose of 40 μg of XH-02
Drug: Single subcutaneous injection
Participants will receive a single dose of XH-02 through subcutaneous injection.
Experimental: Single dose: 60 μg
Participants will receive a single subcutaneous dose of 60 μg of XH-02
Drug: Single subcutaneous injection
Participants will receive a single dose of XH-02 through subcutaneous injection.
Experimental: Single dose: 80 μg
Participants will receive a single subcutaneous dose of 80 μg of XH-02
Drug: Single subcutaneous injection
Participants will receive a single dose of XH-02 through subcutaneous injection.
Experimental: Single dose: 120 μg
Participants will receive a single subcutaneous dose of 120 μg of XH-02
Drug: Single subcutaneous injection
Participants will receive a single dose of XH-02 through subcutaneous injection.
Experimental: Single dose: 160 μg
Participants will receive a single subcutaneous dose of 160 μg of XH-02
Drug: Single subcutaneous injection
Participants will receive a single dose of XH-02 through subcutaneous injection.
Experimental: Multiple dose: 40 μg
Participants will receive 40 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Drug: Multiple subcutaneous injection
Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Experimental: Multiple dose: 60 μg
Participants will receive 60 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Drug: Multiple subcutaneous injection
Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Experimental: Multiple dose: 80 μg
Participants will receive 80 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Drug: Multiple subcutaneous injection
Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Experimental: Multiple dose: 120 μg
Participants will receive 120 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Drug: Multiple subcutaneous injection
Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Experimental: Multiple dose: 160 μg
Participants will receive 160 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Drug: Multiple subcutaneous injection
Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse events (safety)
Time Frame: From the first dose through 30 days after the last dose for non-serious adverse events, and through 3 months after the last dose for severe adverse events.
Adverse events, including serious adverse events. Assessment methods: spontaneous reports; scheduled laboratory tests (hematology, chemistry, C-reactive protein, urinalysis); vital signs (blood pressure, heart rate, respiratory rate, temparature), physical examinations, and electrocardiogram (QTc interval, arrhythmia).
From the first dose through 30 days after the last dose for non-serious adverse events, and through 3 months after the last dose for severe adverse events.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
PTH(1-84) (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 4, 8, 12, 18, 24, 30, 36, 48, 60, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 4, 8, 12, 24, 30, 36, 48, 60, and 72 hours after the last dose.
Serum PTH(1-84)
Single dose: Predose (within one hour before dosing) and 4, 8, 12, 18, 24, 30, 36, 48, 60, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 4, 8, 12, 24, 30, 36, 48, 60, and 72 hours after the last dose.
PTH (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 4, 8, 12, 18, 24, 30, 36, 48, 60, and 72 hours postdose.
Serum PTH
Single dose: Predose (within one hour before dosing) and 4, 8, 12, 18, 24, 30, 36, 48, 60, and 72 hours postdose.
Serum calcium (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing), and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum calcium
Single dose: Predose (within one hour before dosing), and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum phosphorus (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum phosphorus
Single dose: Predose (within one hour before dosing) and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum magnesium (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum magnesium
Single dose: Predose (within one hour before dosing) and 4, 8, 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
1, 25-Dihydroxyvitamin D3 (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 24, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before the 1st, 3rd, and 5th dose) and 48 hours and 72 hours after the last dose.
Serum 1, 25-Dihydroxyvitamin D3
Single dose: Predose (within one hour before dosing) and 24, 48, and 72 hours postdose. Multiple dose: Predose (within one hour before the 1st, 3rd, and 5th dose) and 48 hours and 72 hours after the last dose.
24-hour urine calcium (efficacy)
Time Frame: Single dose: Predose (within 3 days before dosing), and 24, 48, and 72 hours postdose. Multiple dose: Predose (before each dose) and 24, 48, and 72 hours after the last dose.
24-hour urine calcium (24hUCa)
Single dose: Predose (within 3 days before dosing), and 24, 48, and 72 hours postdose. Multiple dose: Predose (before each dose) and 24, 48, and 72 hours after the last dose.
Fractional Excretion of calcium (efficacy)
Time Frame: Single dose: Predose (within one hour before dosing) and 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (before each dose) and 24, 48, and 72 hours after the last dose.
Fractional Excretion of calcium (FECa)
Single dose: Predose (within one hour before dosing) and 12, 24, 36, 48, and 72 hours postdose. Multiple dose: Predose (before each dose) and 24, 48, and 72 hours after the last dose.

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Procollagen type 1 N-terminal propeptide (Exploratory endpoint)
Time Frame: Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Serum Procollagen type 1 N-terminal propeptide (P1NP)
Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.
Beta-isomerized C-terminal telopeptide of type 1 collagen (Exploratory endpoint)
Time Frame: Multiple dose: Predose (within one hour before each dose); and 24, 48, and 72 hours after the last dose.
Serum Beta-isomerized C-terminal telopeptide of type 1 collagen (β-CTX)
Multiple dose: Predose (within one hour before each dose); and 24, 48, and 72 hours after the last dose.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Yan Qin, Peking Union Medical College Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 13, 2026

Primary Completion (Estimated)

April 30, 2030

Study Completion (Estimated)

July 30, 2030

Study Registration Dates

First Submitted

April 13, 2026

First Submitted That Met QC Criteria

April 13, 2026

First Posted (Actual)

April 20, 2026

Study Record Updates

Last Update Posted (Actual)

May 18, 2026

Last Update Submitted That Met QC Criteria

May 13, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • K10348

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

The investigators do not plan to share Individual Participant Data (IPD) due to confidentiality agreements and participant privacy commitments.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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