Efficacy and Safety of XH02 for the Treatment of Hypoparathyroidism
ConditionHypoparathyroidism
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorPeking Union Medical College Hospital
About this trial
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.
Eligibility criteria
Qualifiers
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).
Body Mass Index (BMI) of 17 to 40 kg/m² (inclusive) at screening.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- intravenous administration of PTH1-84 mRNA
Treatment groups
6 Participants
are divided into 5 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov