A single-blind study of M071754 in patients with infantile spasms.

Phase 1

Conditions: Patients diagnosed with infantile spasms
MedDRA version: 19.1Level: PTClassification code 10021750Term: Infantile spasmsSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2017-000230-62-Outside-EU/EEA

Lead Sponsor: Alfresa Pharma Corporation and Sanofi KK

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 12

Inclusion Criteria

Patients (aged >=4 weeks, <2 years) diagnosed with infantile spasms manifested by spasms and hypsarrhythmia on electroencephalography. Using classification of ILAE 1989
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with concurrent severe liver/renal disease, cardiac disease, serious gastrointestinal disorder.
- Patients who received adrenocorticotropic hormone (ACTH) products or steroids within 4 weeks (28 days prior to the formal registration of this study.
- Patients previously or currently treated with vigabatrin.
- Patients who have concurrent or a history of ophthalmological complication in whom possible aggravation of the symptoms with vigabatrin.
- Patients with concomitant Lennox-Gastaut syndrome.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate the efficacy of orally-administered vigabatrin in patients with infantile spasms, using changes in spasm frequency as an endpoint. Also to investigate safety and pharmacokinetics.;Secondary Objective: -;Primary end point(s): Changes in frequency of spasms before and after administration of the investigational drug (percentage of subjects with reduction in frequency of spasms of at least 50% from baseline on the primary spasms assessment date ;Timepoint(s) of evaluation of this end point: 2 days prior to the maintenance administration start date

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Changes in frequency of spasms<br>- Disappearance of spasms<br>- Complete disappearance of infantile spasms and hypsarrhythmia<br>- Brainwave findings<br>- Comprehensive evaluation of efficacy by the Investigators including guardians' opinion.;Timepoint(s) of evaluation of this end point: 2 days prior to the maintenance administration start date