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
114	非ジストロフィー性ミオトニー症候群	2

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	EUCTR2010-024026-38-NL (EUCTR)	10/01/2012	09/02/2011	Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs	Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs	Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene encoding for the skeletal muscle sodium (SCN4A) or chloride (CLCN1) channel. In this study patients with NDMs with a genetically confirmed mutation in the SCN4A-gene or the CLCN1-gene as registred in the Netherlands database of NDMs will be included.	Trade Name: Mexitil Product Name: mexitil INN or Proposed INN: MEXILETINE Other descriptive name: Mexitil	Radboud University Nijmegen Medical Center	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30		Netherlands
2	EUCTR2009-011184-36-IT (EUCTR)	25/06/2010	05/09/2011	Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND	Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND	Myotonic disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias (NDM). The dystrophic myotonias are associated with significant progressive muscular weakness and other systemic organ involvement. On the other hand, NDM usually presents with muscle stiffness as the primary symptom, and severe weakness is not considered a major feature, especially in myotonia congenita. MedDRA version: 14.0;Level: LLT;Classification code 10032487;Term: Other specific muscle disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: MEXITIL*20CPS 200MG INN or Proposed INN: Mexiletine	Dept. of Neurology - Univ. of Kansas Medical Center	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 2	United Kingdom;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

EUCTR2010-024026-38-NL
(EUCTR)

10/01/2012

09/02/2011

Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs

Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene encoding for the skeletal muscle sodium (SCN4A) or chloride (CLCN1) channel. In this study patients with NDMs with a genetically confirmed mutation in the SCN4A-gene or the CLCN1-gene as registred in the Netherlands database of NDMs will be included.

Trade Name: Mexitil
Product Name: mexitil
INN or Proposed INN: MEXILETINE
Other descriptive name: Mexitil

Radboud University Nijmegen Medical Center

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Netherlands

EUCTR2009-011184-36-IT
(EUCTR)

25/06/2010

05/09/2011

Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND

Myotonic disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias (NDM). The dystrophic myotonias are associated with significant progressive muscular weakness and other systemic organ involvement. On the other hand, NDM usually presents with muscle stiffness as the primary symptom, and severe weakness is not considered a major feature, especially in myotonia congenita.
MedDRA version: 14.0;Level: LLT;Classification code 10032487;Term: Other specific muscle disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: MEXITIL*20CPS 200MG
INN or Proposed INN: Mexiletine

Dept. of Neurology - Univ. of Kansas Medical Center

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United Kingdom;Italy