X-linked hypophosphoric rickets
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1. Aged between 1-12 years; 2. A definite diagnosis of X-linked hypophosphatemic rickets (PHEX gene mutation) through pathogenic gene testing; 3. Rickets severity score of at least 2 points; 4. Fasting serum phosphorus lower than 0.97 mmol/L (3 mg/dL).
Exclusion criteria
Exclusion criteria: 1. The height is 50% higher than the average height of children of the same age and sex; 2. The application of growth hormone within 1 year before the test screening; 3. Brain disease, abnormal judgment ability; mental illness unable to cooperate; 4. Those who have participated in other clinical trials within the first three months of this trial; 5. Malignant tumor, active tuberculosis or taking glucocorticoid drugs; 6. Combined with other diseases, and the researchers believe that it affects the evaluation of efficacy or poor compliance; 7. White blood cell count <3.0x10^9/L, or platelets <100x10^9/L, or hemoglobin <90g/L; 8. For any other reason, the investigator considers it inappropriate to participate in the trial; 9. Those who are allergic to all the drugs in this test or their ingredients, or have allergies to the current symptoms, or those who have hypersensitivity constitution.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Imaging global impression change score;Thacher rickets severity score; | — |
Secondary
| Measure | Time frame |
|---|---|
| Height;Maximum tubular phosphorus reabsorption rate;Fractional urinary phosphate excretion; | — |
Countries
China