CClinicalTrials.gg
Sign In
RecruitingNCT07540286Updated May 18, 2026

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

A Phase 2 interventional study of Single subcutaneous injection and Multiple subcutaneous injection in Hypoparathyroidism, sponsored by Peking Union Medical College Hospital. Recruiting at 1 site in China. Open to participants aged 18 Years to 90 Years. Per ClinicalTrials.gov, last updated 2026-05-18.

Sponsored by Peking Union Medical College Hospital · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
60
Allocation
Non-randomized
Ages
18 Years to 90 Years
Sex
All
01

Study 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. 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.

02

Conditions studied

  • Hypoparathyroidism

Browse trials for

Keywords

  • hypoparathyroidism
  • XH-02
  • mRNA drug
03

Who can participate

Ages eligible
18 Years to 90 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
60 participants (estimated)

Study arms

  • Experimental
    Single dose: 40 μg

    Participants will receive a single subcutaneous dose of 40 μg of XH-02

    Drug: Single subcutaneous injection

  • Experimental
    Single dose: 60 μg

    Participants will receive a single subcutaneous dose of 60 μg of XH-02

    Drug: Single subcutaneous injection

  • Experimental
    Single dose: 80 μg

    Participants will receive a single subcutaneous dose of 80 μg of XH-02

    Drug: Single subcutaneous injection

  • Experimental
    Single dose: 120 μg

    Participants will receive a single subcutaneous dose of 120 μg of XH-02

    Drug: Single subcutaneous injection

  • Experimental
    Single dose: 160 μg

    Participants will receive a single subcutaneous dose of 160 μg of XH-02

    Drug: Single 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

  • 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

  • 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

  • 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

  • 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

Interventions

  • DrugSingle subcutaneous injection

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

  • DrugMultiple subcutaneous injection

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

05

What researchers measure

Primary outcomes

  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).

    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.

Secondary outcomes

  1. PTH(1-84) (efficacy)

    Serum PTH(1-84)

    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.

  2. PTH (efficacy)

    Serum PTH

    Time frame: 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

    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.

  4. Serum phosphorus (efficacy)

    Serum phosphorus

    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.

  5. Serum magnesium (efficacy)

    Serum magnesium

    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.

  6. 1, 25-Dihydroxyvitamin D3 (efficacy)

    Serum 1, 25-Dihydroxyvitamin D3

    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.

  7. 24-hour urine calcium (efficacy)

    24-hour urine calcium (24hUCa)

    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.

  8. Fractional Excretion of calcium (efficacy)

    Fractional Excretion of calcium (FECa)

    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.

Other outcomes

  1. Procollagen type 1 N-terminal propeptide (Exploratory endpoint)

    Serum Procollagen type 1 N-terminal propeptide (P1NP)

    Time frame: Multiple dose: Predose (within one hour before each dose) and 24, 48, and 72 hours after the last dose.

  2. Beta-isomerized C-terminal telopeptide of type 1 collagen (Exploratory endpoint)

    Serum Beta-isomerized C-terminal telopeptide of type 1 collagen (β-CTX)

    Time frame: Multiple dose: Predose (within one hour before each dose); and 24, 48, and 72 hours after the last dose.

06

Study locations

1 of 1 sites recruiting
  • Peking Union Medical College Hospital
    Beijing, 100730, China
    • Sanxi Ai, Doctor · Contact · sanxiai@163.com · 18811054896
    • Yan Qin, Doctor · Principal investigator
    Recruiting
07

References and documents

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 ↗

Individual participant data

Plan to share: No — The investigators do not plan to share Individual Participant Data (IPD) due to confidentiality agreements and participant privacy commitments.

08

Registry details

Key details

Study ID
NCT07540286
Lead sponsor
Peking Union Medical College Hospital
Responsible party
Yan Qin (Professor, Peking Union Medical College Hospital) — Principal investigator
First posted
Apr 20, 2026
Start date
Apr 13, 2026
Primary completion
Apr 30, 2030 (estimated)
Completion
Jul 30, 2030 (estimated)
Last update
May 18, 2026

Study contacts

SanXi Ai, Doctor
Contact
sanxiai@163.com
18811054896
Yan Qin
principal investigator · Peking Union Medical College Hospital

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

Contact study team

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Hypoparathyroidism→

Other studies from this sponsor

Peking Union Medical College Hospital

Start the discussion