HypoparathyroidismEarly Phase 1XH02NCT07197450
Efficacy and Safety of XH02 for the Treatment of Hypoparathyroidism
A Early Phase 1 study testing intravenous administration of PTH1-84 mRNA in people with Hypoparathyroidism, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
This study aims to evaluate the safety and efficacy of a novel PTH replacement therapy drug in patients with hypoparathyroidism. The drug is an mRNA drug which will be translated into PTH after intravenous administration, to achieve the therapeutic effect. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hypoparathyroidism
- What is being tested?
- intravenous administration of PTH1-84 mRNA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 20 May 2025to 30 Aug 2027
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 to 65 years (inclusive), male or female.
- Documented history of post-surgical chronic HP or autoimmune, genetic, or idiopathic HP for at least 26 weeks. Diagnosis of HP is confirmed based on a history of hypocalcemia accompanied by an inappropriately low serum PTH level (below the upper limit of the normal range of the local laboratory). * Note: If a subject lacks documented diagnosis of chronic HP but has exhibited hypocalcemia accompanied by an inappropriately low serum PTH level for at least 26 weeks prior to screening, and is judged by the investigator to meet the diagnostic criteria for chronic HP, they will be considered eligible for this criterion.
- Poorly controlled or intolerant to conventional therapy (calcium and active vitamin D).
- Conventional therapy (including vitamin D or magnesium supplements, if applicable) can be optimized during the screening period to achieve the following target serum levels: 25(OH) Vitamin D level: 10 - 100 ng/mL (25 - 250 nmol/L, inclusive). Blood magnesium level: Within the normal range or slightly below, i.e., ≥ 1.3 mg/dL (≥ 0.53 mmol/L). Albumin-corrected serum calcium (sCa) level: Within the normal range or slightly below
- Body Mass Index (BMI) of 17 to 40 kg/m² (inclusive) at screening.
- If aged ≤ 25 years, radiological evidence of closed epiphyses based on X-ray of the non-dominant hand (wrist and palm).
Who cannot join
- Impaired PTH response (pseudohypoparathyroidism), characterized by PTH resistance and elevated PTH levels in the presence of hypocalcemia.
- History of allergic predisposition, or known allergy to the investigational drug or polyethylene glycol (PEG)-containing medications.
- Any disease other than HP that may affect calcium metabolism, calcium-phosphate 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 (HbA1c >9%; HbA1c results from 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; history of parathyroid carcinoma within 5 years prior to screening; acromegaly; or multiple endocrine neoplasia syndromes.
- History of vaccination within 4 weeks prior to enrollment, or planned vaccination during the study period.
- Pregnant or lactating women.
- Patients with high-risk thyroid cancer requiring TSH suppression <0.2 mIU/L within the past 2 years, or patients with a history of malignancy.
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- Requirement for long-term use of the following medications: diuretics, phosphate binders (except calcium supplements), digoxin, lithium, methotrexate, biotin >30 μg/day, or systemic corticosteroids (except as replacement therapy). Patients requiring long-term use of hormones or immunosuppressants (e.g., for rheumatologic/autoimmune diseases) are excluded. *Note: Subjects who can discontinue these medications for the study may be enrolled, provided the medications are stopped for at least 5.5 half-lives prior to blood sampling at Visit 1. Biotin must be stopped for at least 1 day prior to blood sampling during the screening period. These medications are prohibited throughout the entire study.*
- Use of PTH-like drugs (whether commercially available or obtained through participation in a clinical trial), including PTH(1-84), PTH(1-34), other N-terminal fragments or analogs of PTH, or PTH-related protein, within 4 weeks prior to screening.
- Participation in any other interventional trial involving an investigational drug or device within 8 weeks prior to screening, or within 5.5 half-lives of the administered drug from the previous trial (whichever is longer).
- Uncontrolled hypertension at baseline, OR a history of the following cardiovascular and cerebrovascular diseases: (1) Unstable angina; (2) Drug-requiring or severe arrhythmia; (3) Myocardial infarction; (4) Class III or higher heart failure (NYHA classification), or second-degree or higher atrioventricular block; (5) Cerebral infarction (except lacunar infarction), cerebral hemorrhage, or related diseases.
- Increased risk of osteosarcoma, such as Paget's disease of bone or unexplained elevated alkaline phosphatase; hereditary disorders predisposing to osteosarcoma; or patients who have received extensive external beam radiation therapy or implant radiation involving the skeleton.
- Clinically significant abnormal laboratory findings at screening, including any of the following:
- Hematology: Neutrophil count (NEUT#) <1.5 × 10⁹/L; Platelet count (PLT) <90 × 10⁹/L; Hemoglobin (Hb) <90 g/L; Eosinophil count (EOS#) >0.5 × 10⁹/L.
- Liver and Renal Function: Total bilirubin, Alanine Aminotransferase (ALT), or Aspartate Aminotransferase (AST) above the normal range; estimated Glomerular Filtration Rate (eGFR) <60 mL/min/1.73m².
- Any medical or other condition that, in the judgment of the Investigator, may affect the conduct of the study, interfere with the interpretation of study results, or pose an increased risk to the subject or the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety of mRNA Drug XH02 in the Treatment of Adult Hypoparathyroidism
This study aims to evaluate the safety and efficacy of a novel PTH replacement therapy drug in patients with hypoparathyroidism. The drug is an mRNA drug which will be translated into PTH after intravenous administration, to achieve the therapeutic effect.
- Conditions
- Hypoparathyroidism
- Being tested
- intravenous administration of PTH1-84 mRNA
- Keywords
- hypoparathyroidism, PTH, mRNA
- Medical subject headings
- Hypoparathyroidism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
intravenous administration of PTH1-84 mRNADrug
intravenous administration of PTH1-84 mRNA
| Study group | Role | What they receive |
|---|---|---|
| 20ug | Experimental | PTH1-84 mRNA 20ug Receives: Drug: intravenous administration of PTH1-84 mRNA |
| 40ug | Experimental | PTH1-84 mRNA 40ug Receives: Drug: intravenous administration of PTH1-84 mRNA |
| 80ug | Experimental | PTH1-84 mRNA 80ug Receives: Drug: intravenous administration of PTH1-84 mRNA |
| 120ug | Experimental | PTH1-84 mRNA 120ug Receives: Drug: intravenous administration of PTH1-84 mRNA |
| 160ug | Experimental | PTH1-84 mRNA 160ug Receives: Drug: intravenous administration of PTH1-84 mRNA |
What is being measured
The primary outcome is the main question the study is designed to answer.
AE and SAE
adverse event (AE) and severe adverse event (SAE) via regular vital sign checks， physical examinations, and safety laboratory tests (including complete blood count, blood biochemistry, coagulation profile, C-reactive protein, urinalysis, cardiac ultrasound, and electrocardiogram).
Measured: the whole study period including the screening, treatment, and follow-up period. Follow-up period extends to 90 days after administration.
Secondary outcomes (10)
PTH1-84
serum PTH1-84
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
PTH
serum PTH
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
serum calcium
serum calcium
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Serum magnesium
Serum magnesium
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Serum phosphorus
Serum phosphorus
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
1,25-dihydroxyvitamin D
serum 1,25-dihydroxyvitamin D
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Fractional excretion of calcium （FECa)
FECa (%) = (Urine Calcium × Serum Creatinine) / (Serum Calcium × Urine Creatinine) × 100
Measured: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
24-hour urinary calcium
24-hour urinary calcium
Measured: Before (within 3 days) and 24 hours, 48 hours, 72 hours after administration
Procollagen type I N-terminal propeptide (P1NP)
serum procollagen type I N-terminal propeptide (P1NP)
Measured: Before (within 1 hour) and 24 hours, 48 hours, 72 hours after administration
Type I collagen cross-linked C-telopeptide (β-CTX）
serum type I collagen cross-linked C-telopeptide (β-CTX）
Measured: Before (within 1 hour) and 24 hours, 48 hours, 72 hours after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2025
- Study start20 May 2025
- Primary completion30 May 2027 (estimated)
- Study completion30 Aug 2027 (estimated)
Why it stopped: Enrollment temporarily suspended in accordance with a newly implemented regulation, State Council Decree No. 818, on clinical research and clinical translation of new biomedical technologies; pending review and approval.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypoparathyroidismStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- accelerated titration combined with a 3+3 design
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07197450
- Sponsor's protocol ID
- K6744
- Acronym
- XH02
- Status last verified
- Dec 2025
- First submitted
- 14 Sep 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of mRNA Drug XH02 in the Treatment of Adult Hypoparathyroidism
- ClinicalTrials.gov ID
- NCT07197450
- Acronym / sponsor ID
- XH02 / K6744
- Conditions
- Hypoparathyroidism
- Keywords
- hypoparathyroidism, PTH, mRNA
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Enrollment temporarily suspended in accordance with a newly implemented regulation, State Council Decree No. 818, on clinical research and clinical translation of new biomedical technologies; pending review and approval.
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 20 May 2025 (actual)
- Primary completion
- 30 May 2027 (estimated)
- Study completion
- 30 Aug 2027 (estimated)
- First posted
- 29 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to evaluate the safety and efficacy of a novel PTH replacement therapy drug in patients with hypoparathyroidism. The drug is an mRNA drug which will be translated into PTH after intravenous administration, to achieve the therapeutic effect.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
- Model description
- accelerated titration combined with a 3+3 design
| Arm | Type | Receives |
|---|---|---|
| 20ug | Experimental | Drug: intravenous administration of PTH1-84 mRNAPTH1-84 mRNA 20ug |
| 40ug | Experimental | Drug: intravenous administration of PTH1-84 mRNAPTH1-84 mRNA 40ug |
| 80ug | Experimental | Drug: intravenous administration of PTH1-84 mRNAPTH1-84 mRNA 80ug |
| 120ug | Experimental | Drug: intravenous administration of PTH1-84 mRNAPTH1-84 mRNA 120ug |
| 160ug | Experimental | Drug: intravenous administration of PTH1-84 mRNAPTH1-84 mRNA 160ug |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 18 to 65 years (inclusive), male or female.
- Documented history of post-surgical chronic HP or autoimmune, genetic, or idiopathic HP for at least 26 weeks. Diagnosis of HP is confirmed based on a history of hypocalcemia accompanied by an inappropriately low serum PTH level (below the upper limit of the normal range of the local laboratory). * Note: If a subject lacks documented diagnosis of chronic HP but has exhibited hypocalcemia accompanied by an inappropriately low serum PTH level for at least 26 weeks prior to screening, and is judged by the investigator to meet the diagnostic criteria for chronic HP, they will be considered eligible for this criterion.
- Poorly controlled or intolerant to conventional therapy (calcium and active vitamin D).
- Conventional therapy (including vitamin D or magnesium supplements, if applicable) can be optimized during the screening period to achieve the following target serum levels: 25(OH) Vitamin D level: 10 - 100 ng/mL (25 - 250 nmol/L, inclusive). Blood magnesium level: Within the normal range or slightly below, i.e., ≥ 1.3 mg/dL (≥ 0.53 mmol/L). Albumin-corrected serum calcium (sCa) level: Within the normal range or slightly below
- Body Mass Index (BMI) of 17 to 40 kg/m² (inclusive) at screening.
Key exclusion criteria (15 listed)
- Impaired PTH response (pseudohypoparathyroidism), characterized by PTH resistance and elevated PTH levels in the presence of hypocalcemia.
- History of allergic predisposition, or known allergy to the investigational drug or polyethylene glycol (PEG)-containing medications.
- Any disease other than HP that may affect calcium metabolism, calcium-phosphate 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 (HbA1c >9%; HbA1c results from 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; history of parathyroid carcinoma within 5 years prior to screening; acromegaly; or multiple endocrine neoplasia syndromes.
- History of vaccination within 4 weeks prior to enrollment, or planned vaccination during the study period.
- Pregnant or lactating women.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| intravenous administration of PTH1-84 mRNAintravenous administration of PTH1-84 mRNA | Drug | 120ug, 160ug, 20ug, 40ug, 80ug |
Primary outcome
AE and SAE
Time frame: the whole study period including the screening, treatment, and follow-up period. Follow-up period extends to 90 days after administration.
adverse event (AE) and severe adverse event (SAE) via regular vital sign checks， physical examinations, and safety laboratory tests (including complete blood count, blood biochemistry, coagulation profile, C-reactive protein, urinalysis, cardiac ultrasound, and electrocardiogram).
10 secondary outcomes
PTH1-84
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
PTH
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
serum calcium
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Serum magnesium
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Serum phosphorus
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
1,25-dihydroxyvitamin D
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
Fractional excretion of calcium （FECa)
Time frame: Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration
24-hour urinary calcium
Time frame: Before (within 3 days) and 24 hours, 48 hours, 72 hours after administration
Procollagen type I N-terminal propeptide (P1NP)
Time frame: Before (within 1 hour) and 24 hours, 48 hours, 72 hours after administration
Type I collagen cross-linked C-telopeptide (β-CTX）
Time frame: Before (within 1 hour) and 24 hours, 48 hours, 72 hours after administration
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing intravenous administration of PTH1-84 mRNA in people with Hypoparathyroidism, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AE and SAE (time frame: the whole study period including the screening, treatment, and follow-up period. Follow-up period extends to 90 days after administration.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.