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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
intravenous administration of PTH1-84 mRNA
Drugintravenous administration of PTH1-84 mRNA
Treatment groups
6 Participants
are divided into 5 treatment groupsGroup A: 20ugExperimental treatment 1 intervention
Group B: 40ugExperimental treatment 1 intervention
Group C: 80ugExperimental treatment 1 intervention
Group D: 120ugExperimental treatment 1 intervention
Group E: 160ugExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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).
Time frame
the whole study period including the screening, treatment, and follow-up period. Follow-up period extends to 90 days after administration.Secondary outcomes
1
PTH1-84
serum PTH1-84
Time frame
Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration2
PTH
serum PTH
Time frame
Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration3
serum calcium
serum calcium
Time frame
Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administration4
Serum magnesium
Serum magnesium
Time frame
Before (within 1 hour) and 4 hours, 8 hours, 12 hours, 24 hours, 36 hours, 48 hours, 72 hours after administrationSponsors and contacts
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Source ClinicalTrials.gov