Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.

• Register: EUCTR
• Last refreshed on: 16 December 2013
• Main ID: EUCTR2012-005021-76-IT
• Date of registration: 11/12/2012
• Prospective Registration: Yes
• Primary sponsor: EDISON PHARMACEUTICALS INC.
• Public title: Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.
• Scientific title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome
• Date of first enrolment: 31/01/2013
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-005021-76
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: Monocentre If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Italy

Contacts

• Name: Project Management
• Affiliation: OPBG Clinical & Research Services srl

• Name: Project Management
• Affiliation: OPBG Clinical & Research Services srl

Key inclusion & exclusion criteria

Inclusion criteria:
1.Diagnosis of Rett syndrome Stage 1-2 2.Abnormal disease biomarkers 3.Confirmed MeCP2 mutation 4.Patient or patient’s guardian able to consent and comply with protocol requirements 5.Abstention from use of Coenzyne Q10, vitamin E and Idebenone two weeks prior enrollment of treatment with EPI-743
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
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:
1.Any condition, which in the opinion of the investigator could compromise the subject’s safety or adherence to treatment with EPI-743 2.Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil) 3.Clinically significant allergy or hypersensitivity to VitaminE 4.Lack of confirmation of MeCP2 mutation 5.Clinical history of bleeding or abnormal baseline PT/PTT 6.Diagnosis of any other concurrent inborn error of metabolism 7.Hepatic insufficiency with LFTs greater than 3 times upper limit of normal 8.Renal insufficiency requiring dialysis 9.End stage cardiac failure 10.Fat malabsorption syndromes precluding drug absorption


Age minimum:
Age maximum:
Gender:
Female: yes
Male: no

Health Condition(s) or Problem(s) studied

Rett Syndrome
MedDRA version: 14.1 Level: HLGT Classification code 10021605 Term: Inborn errors of metabolism System Organ Class: 10027433 - Metabolism and nutrition disorders

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Intervention(s)

Product Name: EPI-743
Product Code: NA
Pharmaceutical Form: Oral solution
CAS Number: 1213269-98-7
Current Sponsor code: EPI-743
Other descriptive name: Alpha-tocotrienol quinone
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 1.5-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: At 12 weeks and at 24 weeks from baseline

Secondary Objective: Efficacy.To evaluate the efficacy of EPI-743 in patients with Rett syndrome, on: 1.Head circumference 2.Behavior using Rett syndrome behavioral questionnaire 3.Health related quality of life as measured by the PedsQL 4.Disease biomarkers 5.EEG 6.Polysomnography Safety. To evaluate the safety of EPI-743 in patients with Rett syndrome by: absence of dose-limiting AE’s through 6 months, defined as any adverse event = grade 3, per CTCAE version 4.0, that is deemed related to treatment and not related to the underlying disease or the occurrence of any exclusion criteria including the following: 1.Allergy to EPI-743 or sesame oil 2.Liver Function tests greater than 3 times ULN 3.Renal insufficiency requiring dialysis

Main Objective: To evaluate the efficacy of EPI-743 in patients with Rett syndrome, on disease progression as assessed by the Rett Syndrome clinical Severity Score (RSSS)

Primary end point(s): Change from baseline to end of treatment (Week 24, V6) in score of Rett Syndrome clinical Severity Scale (RSSS)

Secondary Outcome(s)

Secondary end point(s): Efficacy. Change from baseline to end of treatment (Week 24, V6) in: 1.Head circumference 2.Level of disease biomarkers 3.Score of Rett syndrome behavioral questionnaire 4.Score of PedsQL questionnaire 5.EEG parameters 6.Polysomnography parameters Safety. Safety will be evaluated by abnormal liver function and hepatic function laboratory tests and serious and non serious adverse event assessments over the 24 weeks of study treatment.

Timepoint(s) of evaluation of this end point: Every 4 weeks

Secondary ID(s)

OPBGC&RS_12_003

Source(s) of Monetary Support

Edison Pharmaceuticals Inc.