Rett SyndromePhase 2NCT01822249
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
A Phase 2 study testing EPI-743 in people with Rett Syndrome, run by Edison Pharmaceuticals Inc at 1 site in Italy.
Trial summary
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress. Read more about this study
- Who is it for?
- Ages Up to 18 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- EPI-743
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteItaly
- Study dates
- Jan 2013to Jan 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of Rett syndrome with disease stage 1-2
- Abnormality of at least two disease biomarker levels
- Confirmed MeCP2 mutation
- Patient or patient's guardian able to consent and comply with protocol requirements
- Abstention from use of Coenzyme Q10, vitamin E and Idebenone two weeks prior to enrollment into the study
Who cannot join
- Any condition, which in the opinion of the investigator could compromise the subject's safety or adherence to treatment with EPI-743.
- Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).
- Clinically significant allergy or hypersensitivity to Vitamin E
- Lack of confirmation of MeCP2 mutation
- Clinical history of bleeding or abnormal baseline PT/PTT
- Diagnosis of any other concurrent inborn error of metabolism
Show all 10Show fewer
- Hepatic insufficiency with LFTs greater than 3 times upper limit of normal
- Renal insufficiency requiring dialysis
- End stage cardiac failure
- Fat malabsorption syndromes precluding drug absorption
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress. EPI-743 is a novel therapeutic with demonstrated efficacy and safety in the treatment of disorders characterized by oxidative stress. The purpose of this study is to examine the safety and efficacy of EPI-743 in a population of children with Rett syndrome.
- Conditions
- Rett Syndrome
- Being tested
- EPI-743, Placebo
- Keywords
- Rett syndrome, EPI-743, Edison
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPI-743Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| EPI-743 15 mg/kg | Active comparator | Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily Receives: Drug: EPI-743 |
| Placebo | Placebo comparator | Subjects in this arm will receive placebo at a volume equivalent to the volume of EPI-743 they would receive if in active group based on their weight Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rett Syndrome Clinical Severity Sore
Measure of disease progression
Measured: Change at six months from baseline
Secondary outcomes (6)
Oxidative Stress Biomarkers
Measured: Change at six months from baseline
Head circumference
Measured: Change at six months from baseline
Rett syndrome behavioral questionnaire
Measured: Change at six months from baseline
PedsQL
Measured: Change at six months from baseline
Number of Drug-related adverse and serious adverse events
Measured: Six months
Respiratory Disturbance Index (RDI)
RDI will be determined on polysomnography study
Measured: Change at six months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2013 (estimated)
- Study startJan 2013
- Primary completionDec 2013
- Study completionJan 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edison Pharmaceuticals Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01822249
- Sponsor's protocol ID
- OPBGC&RS_12_003
- Status last verified
- Jul 2018
- First submitted
- 25 Mar 2013
- Last updated
- 26 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
- ClinicalTrials.gov ID
- NCT01822249
- Acronym / sponsor ID
- OPBGC&RS_12_003
- Conditions
- Rett Syndrome
- Keywords
- Rett syndrome, EPI-743, Edison
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Edison Pharmaceuticals IncIndustry (pharma, biotech, devices)
- Study start
- Jan 2013
- Primary completion
- Dec 2013 (actual)
- Study completion
- Jan 2014 (actual)
- First posted
- 2 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress. EPI-743 is a novel therapeutic with demonstrated efficacy and safety in the treatment of disorders characterized by oxidative stress. The purpose of this study is to examine the safety and efficacy of EPI-743 in a population of children with Rett syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EPI-743 15 mg/kg | Active comparator | Drug: EPI-743Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily |
| Placebo | Placebo comparator | Drug: PlaceboSubjects in this arm will receive placebo at a volume equivalent to the volume of EPI-743 they would receive if in active group based on their weight |
Who may qualify
- Ages
- Up to 18 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Diagnosis of Rett syndrome with disease stage 1-2
- Abnormality of at least two disease biomarker levels
- Confirmed MeCP2 mutation
- Patient or patient's guardian able to consent and comply with protocol requirements
- Abstention from use of Coenzyme Q10, vitamin E and Idebenone two weeks prior to enrollment into the study
Key exclusion criteria (10 listed)
- Any condition, which in the opinion of the investigator could compromise the subject's safety or adherence to treatment with EPI-743.
- Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).
- Clinically significant allergy or hypersensitivity to Vitamin E
- Lack of confirmation of MeCP2 mutation
- Clinical history of bleeding or abnormal baseline PT/PTT
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EPI-743 | Drug | EPI-743 15 mg/kg |
| Placebo | Drug | Placebo |
Primary outcome
Rett Syndrome Clinical Severity Sore
Time frame: Change at six months from baseline
Measure of disease progression
6 secondary outcomes
Oxidative Stress Biomarkers
Time frame: Change at six months from baseline
Head circumference
Time frame: Change at six months from baseline
Rett syndrome behavioral questionnaire
Time frame: Change at six months from baseline
PedsQL
Time frame: Change at six months from baseline
Number of Drug-related adverse and serious adverse events
Time frame: Six months
Respiratory Disturbance Index (RDI)
Time frame: Change at six months from baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EPI-743 in people with Rett Syndrome, run by Edison Pharmaceuticals Inc at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rett Syndrome Clinical Severity Sore (time frame: Change at six months from baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2018.