hypophosphatemic rickets
Conditions
Interventions
Sodium Dihydrogen Phosphate and Sodium Hydrogen Phosphate Granules Combined with Calcitriol Treatment Group:Sodium Dihydrogen Phosphate and Sodium Hydrogen Phosphate Granules (Jiafuning?): Initial pho
Calcitriol Soft Capsules (Haidewei?): Initial dose of 30 ng/kg/d, administered in 2 divided doses daily, up to a maximum of 60 ng/kg/d. Continuous administration for 48 weeks.
Sponsors
West China Second University Hospital of Sichuan University (WCSUH- SCU)
Eligibility
Sex/Gender
All
Age
1 Years to 14 Years
Inclusion criteria
Inclusion criteria: 1. Age 1–14 years (inclusive), any sex; 2. Diagnosed with hypophosphatemic rickets per the Chinese Guidelines for Diagnosis and Treatment of Hypophosphatemic Rickets/Osteomalacia (2022 Edition); 3. No phosphate supplementation therapy received within the past 6 months; 4. At screening, after overnight fasting (>=8 h), serum phosphorus <2.5 mg/dL (0.80 mmol/L); one retest permitted if applicable; 5. Able to follow instructions and comply with study procedures; informed consent signed by parent(s) or legal guardian(s).
Exclusion criteria
Exclusion criteria: 1. Confirmed presence of other causes of hypophosphatemia, including renal phosphate wasting of other etiologies, such as tumor-induced osteomalacia, fibrous dysplasia, McCune-Albright syndrome (MAS), or due to primary renal diseases (e.g., Fanconi syndrome); 2. Confirmed diagnosis of other definite causes of growth retardation or short stature, including growth hormone deficiency (GHD), multiple pituitary hormone deficiency, small for gestational age (SGA), Turner syndrome, or short stature associated with other severe chronic diseases (e.g., cardiac, hepatic, or renal insufficiency, chronic inflammatory bowel disease); 3. History of traumatic fracture or orthopedic surgery within 6 months prior to screening, or planned orthopedic surgery during the study period; 4. Abnormal screening laboratory results, defined as any of the following: (1) Corrected serum calcium >= 10.8 mg/dL (2.69 mmol/L); (2) Intact parathyroid hormone (iPTH) >= 2.5 × the upper limit of normal (ULN); (3) Severe renal impairment, defined as estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m^2; 5. Known hypersensitivity to any component of the investigational product or to drugs with similar chemical structures; 6. Requirement for sodium restriction; 7. Hyperparathyroidism or hypoparathyroidism requiring clinical intervention; 8. Severe renal nephrocalcinosis confirmed by ultrasound; 9. Chronic use of medications known to affect bone metabolism or serum phosphorus that cannot be discontinued during the study, including but not limited to bisphosphonates, glucocorticoids, anticonvulsants, growth hormone, and antiviral agents; 10. Participation in another clinical trial within the 4 weeks prior to screening (excluding subjects who only signed the informed consent form [ICF] and then withdrew prior to undergoing any screening procedures); 11. History of malignancy within 5 years, excluding adequately treated basal cell or squamous cell carcinoma of the skin; 12. Any other condition that, in the opinion of the investigator, would render the patient inappropriate for participation in this study;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Radiographic Global Impression of Change (RGI-C) Score at Week 48;Change from baseline in Rickets Severity Score (RSS) at Week 48; | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in height standard deviation score (SDS) at Weeks 12, 24, 36, and 48;Change from baseline in alkaline phosphatase (ALP) at Weeks 12, 24, 36, and 48;Number and incidence of adverse events/adverse reactions;Vital signs;Renal ultrasound findings (presence or absence of calcification) at Week 24 and Week 48;Change from baseline in serum phosphorus at Weeks 12, 24, 36, and 48;physical examination;Other laboratory parameters (blood cell count, liver function, renal function, blood electrolytes, urinary electrolytes, urinary creatinine); | — |
Countries
China
Contacts
Public ContactYu Dan
West China Second University Hospital of Sichuan University (WCSUH- SCU)
Outcome results
None listed