DDrare: Database of Drug Development for Rare Diseases

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

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2017-002163-17-GB (EUCTR)	21/01/2020	31/10/2019	Use 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)	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	225	Phase 2;Phase 3	Spain;Austria;Germany;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2017-002163-17-GB
(EUCTR)

21/01/2020

31/10/2019

Use 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)

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

225

Phase 2;Phase 3

Spain;Austria;Germany;United Kingdom