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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 20ug | Experimental | PTH1-84 mRNA 20ug |
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| 40ug | Experimental | PTH1-84 mRNA 40ug |
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| 80ug | Experimental | PTH1-84 mRNA 80ug |
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| 120ug | Experimental | PTH1-84 mRNA 120ug |
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| 160ug | Experimental | PTH1-84 mRNA 160ug |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| intravenous administration of PTH1-84 mRNA | Drug | intravenous administration of PTH1-84 mRNA |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | the whole study period including the screening, treatment, and follow-up period. Follow-up period extends to 90 days after administration. |
| Measure | Description | Time Frame |
|---|---|---|
| PTH1-84 | serum PTH1-84 | Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration |
| PTH | serum PTH |
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Inclusion Criteria:
Exclusion Criteria:
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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Recruiting | Beijing | 100730 | China |
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| ID | Term |
|---|---|
| D007011 | Hypoparathyroidism |
| ID | Term |
|---|---|
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
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accelerated titration combined with a 3+3 design
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| 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 | 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 | 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 | 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 | 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 | 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 | 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) | 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) | Before (within 1 hour) and 24 hours, 48 hours, 72 hours after administration |