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HypoparathyroidismPhase 2NCT07540286

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

A Phase 2 study testing Single subcutaneous injection and Multiple subcutaneous injection in people with Hypoparathyroidism, run by Peking Union Medical College Hospital at 1 site in China.

Suspended

Paused. Enrolment is on hold for now and may resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 90 years
Conditions the study is for
Hypoparathyroidism
What is being tested?
Single subcutaneous injection and Multiple subcutaneous injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
1 siteChina
Study dates
13 Apr 2026to 30 Jul 2030
Phase
Phase 2
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 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years, both males and females eligible;
  • 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.
  • Poorly controlled or intolerant to conventional treatment (calcium and vitamin D) for hypoparathyroidism;
  • BMI 17-40 kg/m² (inclusive) at screening;
  • If age ≤25 years, radiographic evidence of epiphyseal closure based on X-ray results of the wrist and palm of the non-dominant hand.

Who cannot join

  • Impaired PTH response (pseudohypoparathyroidism), characterized by PTH resistance and elevated PTH levels in the presence of hypocalcemia;
  • Allergic constitution, or allergy to the investigational drug or polyethylene glycol (PEG)-based drugs;
  • 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;
  • Pregnant or breastfeeding women;
  • 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;
  • Patients with high-risk thyroid cancer requiring TSH suppression to <0.2 mIU/L within 2 years, or those with a history of tumors;
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  • Use of loop diuretics, phosphate binders (except calcium supplements), digoxin, lithium, methotrexate, biotin >30 mcg/day, or systemic corticosteroids (except as replacement therapy);
  • 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;
  • 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;
  • 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;
  • 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;
  • 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;
  • 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.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Peking Union Medical College Hospital

    Beijing, 100730

Other trials in China

About this study

Official title: 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.

Being tested
Single subcutaneous injection, Multiple subcutaneous injection
Keywords
hypoparathyroidism, XH-02, mRNA drug
Medical subject headings
Hypoparathyroidism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Single subcutaneous injectionDrug

    Participants will receive a single dose of XH-02 through subcutaneous injection.

  • Multiple subcutaneous injectionDrug

    Participants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.

Study groups in NCT07540286
Study groupRoleWhat they receive
Single dose: 40 μgExperimentalParticipants will receive a single subcutaneous dose of 40 μg of XH-02
Receives: Drug: Single subcutaneous injection
Single dose: 60 μgExperimentalParticipants will receive a single subcutaneous dose of 60 μg of XH-02
Receives: Drug: Single subcutaneous injection
Single dose: 80 μgExperimentalParticipants will receive a single subcutaneous dose of 80 μg of XH-02
Receives: Drug: Single subcutaneous injection
Single dose: 120 μgExperimentalParticipants will receive a single subcutaneous dose of 120 μg of XH-02
Receives: Drug: Single subcutaneous injection
Single dose: 160 μgExperimentalParticipants will receive a single subcutaneous dose of 160 μg of XH-02
Receives: Drug: Single subcutaneous injection
Multiple dose: 40 μgExperimentalParticipants will receive 40 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Receives: Drug: Multiple subcutaneous injection
Multiple dose: 60 μgExperimentalParticipants will receive 60 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Receives: Drug: Multiple subcutaneous injection
Multiple dose: 80 μgExperimentalParticipants will receive 80 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Receives: Drug: Multiple subcutaneous injection
Multiple dose: 120 μgExperimentalParticipants will receive 120 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Receives: Drug: Multiple subcutaneous injection
Multiple dose: 160 μgExperimentalParticipants will receive 160 μg of XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Receives: Drug: Multiple subcutaneous injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse events (safety)

    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).

    Measured: 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 outcomes (8)

  1. PTH(1-84) (efficacy)

    Serum PTH(1-84)

    Measured: 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.

  2. PTH (efficacy)

    Serum PTH

    Measured: Single dose: Predose (within one hour before dosing) and 4, 8, 12, 18, 24, 30, 36, 48, 60, and 72 hours postdose.

  3. Serum calcium (efficacy)

    Serum calcium

    Measured: 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.

  4. Serum phosphorus (efficacy)

    Serum phosphorus

    Measured: 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.

  5. Serum magnesium (efficacy)

    Serum magnesium

    Measured: 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.

  6. 1, 25-Dihydroxyvitamin D3 (efficacy)

    Serum 1, 25-Dihydroxyvitamin D3

    Measured: 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.

  7. 24-hour urine calcium (efficacy)

    24-hour urine calcium (24hUCa)

    Measured: 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.

  8. Fractional Excretion of calcium (efficacy)

    Fractional Excretion of calcium (FECa)

    Measured: 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.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Apr 2026
  2. Study start13 Apr 2026
  3. Primary completion30 Apr 2030 (estimated)
  4. Study completion30 Jul 2030 (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

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
Single dose: 5 dose groups (40, 60, 80, 120, 160 μg). Multiple doses: 5 dose groups (40, 60, 80, 120, 160 μg), daily or alternate-day dosing.
Masking
None (open label)
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07540286
Sponsor's protocol ID
K10348
Status last verified
Sep 2026
First submitted
13 Apr 2026
Last updated
28 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: The investigators do not plan to share Individual Participant Data (IPD) due to confidentiality agreements and participant privacy commitments.

Publications

  • Shoback DM, Bilezikian JP, Costa AG, Dempster D, Dralle H, Khan AA, Peacock M, Raffaelli M, Silva BC, Thakker RV, Vokes T, Bouillon R. Presentation of Hypoparathyroidism: Etiologies and Clinical Features. J Clin Endocrinol Metab. 2016 Jun;101(6):2300-12. doi: 10.1210/jc.2015-3909. Epub 2016 Mar 4.

    PubMed 26943721

  • Khan AA, Bilezikian JP, Brandi ML, Clarke BL, Gittoes NJ, Pasieka JL, Rejnmark L, Shoback DM, Potts JT, Guyatt GH, Mannstadt M. Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop. J Bone Miner Res. 2022 Dec;37(12):2568-2585. doi: 10.1002/jbmr.4691. Epub 2022 Nov 14.

    PubMed 36054621

  • Gosmanova EO, Houillier P, Rejnmark L, Marelli C, Bilezikian JP. Renal complications in patients with chronic hypoparathyroidism on conventional therapy: a systematic literature review : Renal disease in chronic hypoparathyroidism. Rev Endocr Metab Disord. 2021 Jun;22(2):297-316. doi: 10.1007/s11154-020-09613-1. Epub 2021 Feb 18.

    PubMed 33599907

  • Gosmanova EO, Chen K, Rejnmark L, Mu F, Swallow E, Briggs A, Ayodele O, Sherry N, Ketteler M. Risk of Chronic Kidney Disease and Estimated Glomerular Filtration Rate Decline in Patients with Chronic Hypoparathyroidism: A Retrospective Cohort Study. Adv Ther. 2021 Apr;38(4):1876-1888. doi: 10.1007/s12325-021-01658-1. Epub 2021 Mar 9.

    PubMed 33687651

  • Gosmanova EO, Ayodele O, Chen K, Cook EE, Mu F, Young JA, Rejnmark L. Association of Calcium and Phosphate Levels with Incident Chronic Kidney Disease in Patients with Hypoparathyroidism: A Retrospective Case-Control Study. Int J Endocrinol. 2022 Nov 2;2022:6078881. doi: 10.1155/2022/6078881. eCollection 2022.

    PubMed 36389126

  • Karpf DB, Pihl S, Mourya S, Mortensen E, Kovoor E, Markova D, Leff JA. A Randomized Double-Blind Placebo-Controlled First-In-Human Phase 1 Trial of TransCon PTH in Healthy Adults. J Bone Miner Res. 2020 Aug;35(8):1430-1440. doi: 10.1002/jbmr.4016. Epub 2020 Apr 16.

    PubMed 32212275

  • Khan AA, Rubin MR, Schwarz P, Vokes T, Shoback DM, Gagnon C, Palermo A, Marcocci C, Clarke BL, Abbott LG, Hofbauer LC, Kohlmeier L, Pihl S, An X, Eng WF, Smith AR, Ukena J, Sibley CT, Shu AD, Rejnmark L. Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial. J Bone Miner Res. 2023 Jan;38(1):14-25. doi: 10.1002/jbmr.4726. Epub 2022 Nov 12.

    PubMed 36271471

  • Khan AA, Koch CA, Van Uum S, Baillargeon JP, Bollerslev J, Brandi ML, Marcocci C, Rejnmark L, Rizzoli R, Shrayyef MZ, Thakker R, Yildiz BO, Clarke B. Standards of care for hypoparathyroidism in adults: a Canadian and International Consensus. Eur J Endocrinol. 2019 Mar;180(3):P1-P22. doi: 10.1530/EJE-18-0609.

    PubMed 30540559

  • Chen KS, Gosmanova EO, Curhan GC, Ketteler M, Rubin M, Swallow E, Zhao J, Wang J, Sherry N, Krasner A, Bilezikian JP. Five-year Estimated Glomerular Filtration Rate in Patients With Hypoparathyroidism Treated With and Without rhPTH(1-84). J Clin Endocrinol Metab. 2020 Oct 1;105(10):e3557-65. doi: 10.1210/clinem/dgaa490.

    PubMed 32738041

  • Rejnmark L, Ayodele O, Lax A, Mu F, Swallow E, Gosmanova EO. The risk of chronic kidney disease development in adult patients with chronic hypoparathyroidism treated with rhPTH(1-84): A retrospective cohort study. Clin Endocrinol (Oxf). 2023 Apr;98(4):496-504. doi: 10.1111/cen.14813. Epub 2022 Aug 28.

    PubMed 35974422

  • Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2.

    PubMed 34035281

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Single subcutaneous injection and Multiple subcutaneous injection 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 5 inclusion and 13 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 28 Sep 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?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

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.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07540286, which also lists 11 linked publications.

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 28 Sep 2026.

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