SUB09246MIG ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
18脊髄小脳変性症(多系統萎縮症を除く。)1

18. 脊髄小脳変性症(多系統萎縮症を除く。)


臨床試験数 : 71 薬物数 : 101 - (DrugBank : 30) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 65
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2017-002163-17-GB
(EUCTR)
21/01/202031/10/2019Use of Nicotinamide for the Treatment of Patients with Friedreich's Ataxia.A RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICH´S ATAXIA (NICOFA) - NICOFA Friedreich's ataxia.Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia. It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene (FXN) and results in decreased levels of FXN protein. FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty. Patients gradually lose coordination, become dysarthric and are frequently wheelchair-bound as adolescents.
MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Nicotinamide
Product Name: Nicotinamide
INN or Proposed INN: Nicotinamide
Other descriptive name: SUB09246MIG
RWTH Aachen University, Center for Translational & Clinical Research Aachen (CTC-A)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
225Phase 2;Phase 3Spain;Austria;Germany;United Kingdom