A Cohort Study on the Safety and Efficacy of XH-02 in Treating Hypoparathyroidism
ConditionHypoparathyroidism
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-90
SponsorPeking Union Medical College Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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;
Disqualifiers
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;
Trial design
Design model
Parallel
Treatments tested in this trial
Single subcutaneous injection
DrugParticipants will receive a single dose of XH-02 through subcutaneous injection.
Multiple subcutaneous injection
DrugParticipants will receive XH-02 via subcutaneous injection daily or every other day, for a total of 5 doses.
Treatment groups
60 Participants
are divided into 10 treatment groups10
Treatment groupsSee each treatment group below.
Group A: Single dose: 40 μgExperimental treatment 1 intervention
Group B: Single dose: 60 μgExperimental treatment 1 intervention
Group C: Single dose: 80 μgExperimental treatment 1 intervention
Group D: Single dose: 120 μgExperimental treatment 1 intervention
Group E: Single dose: 160 μgExperimental treatment 1 intervention
Group F: Multiple dose: 40 μgExperimental treatment 1 intervention
Group G: Multiple dose: 60 μgExperimental treatment 1 intervention
Group H: Multiple dose: 80 μgExperimental treatment 1 intervention
Group I: Multiple dose: 120 μgExperimental treatment 1 intervention
Group J: Multiple dose: 160 μgExperimental treatment 1 intervention
Trial outcomes
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.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.Sponsors and contacts
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Source ClinicalTrials.gov