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

告示番号

疾患名（ページ内リンク）

臨床試験数

脊髄小脳変性症（多系統萎縮症を除く。）

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	EUCTR2007-003357-85-IT (EUCTR)	10/10/2007	09/11/2007	Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND	Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND	Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation in the FXN gene, which encodes a protein named frataxin. As a result of the mutation, frataxin is quantitatively reduced but qualitatively normal. Thus, any pharmacological agent able to increase frataxin intracellular levels would have a great therapeutic relevance. MedDRA version: 6.1;Level: PT;Classification code 10003591	Trade Name: EPREX*1FL 40000UI/ML 1ML INN or Proposed INN: Erythropoietin Other descriptive name: Erythropoietin	ISTITUTO NEUROLOGICO CARLO BESTA	NULL	Not Recruiting			Female: yes Male: yes			Italy

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

EUCTR2007-003357-85-IT
(EUCTR)

10/10/2007

09/11/2007

Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND

Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation in the FXN gene, which encodes a protein named frataxin. As a result of the mutation, frataxin is quantitatively reduced but qualitatively normal. Thus, any pharmacological agent able to increase frataxin intracellular levels would have a great therapeutic relevance.
MedDRA version: 6.1;Level: PT;Classification code 10003591

Trade Name: EPREX*1FL 40000UI/ML 1ML
INN or Proposed INN: Erythropoietin
Other descriptive name: Erythropoietin

ISTITUTO NEUROLOGICO CARLO BESTA

NULL

Not Recruiting

Female: yes
Male: yes

Italy