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Application of compound in preparation of medicine for treating Alport syndrome

A compound and syndrome technology, applied in the field of medicine, can solve the problems of reducing urinary protein excretion, unable to change the final outcome of kidney disease, etc., and achieve the effects of reducing urinary protein excretion, lowering serum creatinine level, and prolonging survival time.

Pending Publication Date: 2022-06-24
SHANGHAI CHILDRENS HOSPITAL
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

The purpose of treatment is mainly to reduce urinary protein excretion and delay the chronic progression of renal disease, but it cannot change the final outcome of end-stage renal disease
There are currently no other effective treatments

Method used

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  • Application of compound in preparation of medicine for treating Alport syndrome
  • Application of compound in preparation of medicine for treating Alport syndrome
  • Application of compound in preparation of medicine for treating Alport syndrome

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0066] Example 1 Efficacy of HCQ in the treatment of X-linked Alport syndrome (XLAS) mice

[0067] The experiment set up 2 mouse models of X-linked Alport syndrome (XLAS). The first XLAS mouse model is the classical mouse model (COL4A5 p.G5X), purchased from Jackson Laboratory (JAX STOCK#3376), genetic background: C57BL / 6J; gene name: COL4A5; mutation type: nonsense mutation ( c.G213T, p.Gly5X), genotype: X G5X / X w (female heterozygote), X G5X / X G5X or X G5X / Y (male hemizygote) has been widely used in many studies at home and abroad. The second XLAS mouse model is the model mouse constructed as described in the first part of the study, referring to the suspected pathogenic gene variant found in the clinic and using CRISPR / Cas9 gene editing technology to construct a new frameshift mutation mouse model of COL4A5, and complete the phenotype Evaluate. Genetic background: C57BL / 6; Gene name: COL4A5; Mutation type: Frameshift mutation (c.2440delG, p.Gly814fs); Genotype: X ...

Embodiment 2

[0084] Example 2 HCQ treats children with X-linked Alport syndrome (XLAS)

[0085] This study is a single-center, concurrent, non-randomized, comparative study to evaluate the efficacy and safety of hydroxychloroquine combined with benazepril hydrochloride tablets and benazepril hydrochloride tablets alone (current first-line treatment regimen) in the treatment of children with AS sex. Subjects were included in the study group in a 1:1 ratio.

[0086] This clinical study was approved by the ethics committee of our hospital (ethics batch number: 2019R063-E02), and all the children included in this clinical study and their legal guardians signed the informed consent.

[0087] Drug usage and dosage:

[0088] HCQ treatment group: basic treatment combined with hydroxychloroquine sulfate treatment. The details are as follows: Hydroxychloroquine Sulfate Tablets (6.5mg / kg / day, taken in divided doses) + Benazepril Hydrochloride Tablets (5-10mg / time, 1 time / day), orally 15-30 minutes...

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Abstract

The invention relates to the technical field of medicines, in particular to application of a compound in preparation of a medicine for treating Alport syndrome. The compound is selected from one or more of the following compounds: hydroxychloroquine; a hydroxychloroquine derivative; a pharmaceutically acceptable salt of the aforementioned compound. Experimental results of the invention indicate that the hydroxychloroquine or the derivative and the pharmaceutically acceptable salt thereof have a therapeutic effect on the Alport syndrome.

Description

technical field [0001] The invention relates to the technical field of medicine, in particular to the application of a compound in the preparation of a medicine for treating Alport syndrome. Background technique [0002] Alport Syndrome (AS), also known as hereditary nephritis, is caused by COL4A3 / COL4A4 / COL4A5 encoding the glomerular basement membrane (GBM) collagen type IV (COLIV) α3 / α4 / α5 chain Caused by genetic mutation. The main clinical manifestations of children with Alport syndrome are hematuria, proteinuria and progressive renal dysfunction. Some children may be combined with extrarenal symptoms such as ocular lens lesions and sensorineural deafness. It is a single gene that seriously affects the physical and mental health of patients. Inherited kidney disease. X-linked Alport syndrome accounts for about 80% of cases, with the remainder due to autosomal recessive and autosomal dominant mutations. As kidney damage worsens, people with Alport syndrome often develop...

Claims

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Application Information

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IPC IPC(8): A61K31/4706A61K45/06A61P13/12A61P13/00
CPCA61K31/4706A61K45/06A61P13/12A61P13/00
Inventor 黄文彦孙蕾匡新宇
Owner SHANGHAI CHILDRENS HOSPITAL
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