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Deutetrabenazine for the treatment of dyskinesia in cerebral palsy

A movement disorder, cerebral palsy technology, applied in pharmaceutical formulations, medical preparations containing active ingredients, nervous system diseases, etc., can solve problems such as failure to prove patient function improvement

Pending Publication Date: 2021-09-14
AUSPEX PHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Dalfampridine (a small molecule potassium channel blocker thought to restore conduction in centrally demyelinated axons) has been approved for multiple sclerosis but has not been demonstrated in patients with CP improved functionality of

Method used

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  • Deutetrabenazine for the treatment of dyskinesia in cerebral palsy
  • Deutetrabenazine for the treatment of dyskinesia in cerebral palsy
  • Deutetrabenazine for the treatment of dyskinesia in cerebral palsy

Examples

Experimental program
Comparison scheme
Effect test

example 1

[0227] Example 1: Efficacy Study

[0228] A phase 3, 21-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of deutetrabenazine was conducted as follows.

[0229] Approximately 185 patients were randomized in a 2:1 ratio to deutetrabenazine versus placebo (approximately 124 in the deutetrabenazine group; approximately 61 in the placebo group), based on Stratified by age (6 to <12 years; 12 to 18 years, inclusive) and region (United States [US]; non-US). Sample size was reestimated at interim analysis (IA) and adjustments could be made for up to a total of approximately 230 patients.

[0230] The study population consisted of male and female patients diagnosed with DCP aged 6 to 18 years (inclusive).

[0231] A patient can be included in this study only if he or she meets all of the following criteria:

[0232] 1. Patients were 6 to 18 years old (inclusive) at baseline.

[0233] 2. The patient weighed at ...

example 2

[0390] Example 2: Open Label Extension Study

[0391] A Phase 3, 55-week, open-label, single-arm, long-term safety, tolerability, and efficacy study of deutetrabenazine for the treatment of movement disorders in children and adolescents with cerebral palsy was conducted as follows:

[0392] General study design: Phase 3, 21-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study ("parent study") to evaluate the efficacy and safety of deutetrabenazine has been successfully completed Patients may be eligible to participate in the study after completing a 1-week washout period and a final assessment at week 16. The study will include children and adolescents who are between the ages of 6 and 18 when they participate in the maternal study.

[0393] Screening: Screening assessments for this open-label study will be performed as part of baseline follow-up. Any adverse events initiated after the end of the maternal study and recorded after informed con...

example 3

[0440] Example 3: Efficacy Study in Adults

[0441] A phase 3 study was conducted to evaluate the efficacy and safety of deutetrabenazine in a cohort of male and female patients diagnosed with DCP. A patient can be included in this study only if he or she meets all of the following criteria:

[0442] 1. The patient is older than 18 years at baseline.

[0443] 2. The patient has symptoms of cerebral palsy (CP) from infancy (≤2 years).

[0444] 3. According to the European cerebral palsy surveillance standards, the patient was diagnosed as DCP.

[0445] 4. Based on the investigator's score of chorea, the patient's total score on the MD-CRS Part II item at the baseline visit is ≥10.

[0446] 5. The patient's symptoms lead to functional problems as determined by a Clinical Global Impression of Severity (CGI-S) score of 4 or higher based on the investigator score.

[0447] 6. Chorea is the major movement disorder as assessed by EAB at Screening.

[0448] Patients will receive ...

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Abstract

The disclosure is directed to methods of treating dyskinesia in cerebral palsy in human patients using deutetrabenazine and its active metabolites.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of U.S. Provisional Application No. 62 / 779,232, filed December 13, 2018, and U.S. Provisional Application No. 62 / 801,450, filed February 5, 2019, the entire contents of which are incorporated herein by reference. technical field [0003] The present disclosure relates to methods of using deutetrabenazine and its active metabolites to treat movement disorders in cerebral palsy in human patients, including pediatric patients. Background technique [0004] Deuterated tetrabenazine ((RR,SS)-1,3,4,6,7,11b-hexahydro-9,10-di(methoxy-d3)-3-(2-methylpropyl )-2H-benzo[a]quinazin-2-one) is a type 2 vesicular monoamine transporter (VMAT2). Bioactive metabolites formed from deuterated tetrabenazine (α-dihydrotetrabenazine [α-HTBZ] and β-dihydrotetrabenazine [β-HTBZ]) are potent inhibitors of VMAT2 binding , where the values ​​of the inhibition constants were 3.8 nM and 22 nM, respectively. De...

Claims

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Application Information

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IPC IPC(8): A61K31/4745A61P25/02
CPCA61P25/02A61K31/4745A61K31/4375A61P25/14
Inventor 尤哈-马蒂·萨沃纳马克·福里斯特·戈登弗兰克·施耐德
Owner AUSPEX PHARMA INC
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