Rett SyndromePhase 2StatinsNCT02563860
Pharmacological Treatment of Rett Syndrome With Statins
A Phase 2 study testing Lovastatin in people with Rett Syndrome, run by Montefiore Medical Center at 1 site in United States.
Trial summary
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome. Read more about this study
- Who is it for?
- Ages 3 years and older, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Lovastatin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2015to Jul 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 years and older
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Females patients,
- Genetically confirmed RTT,
- Ambulatory.
Who cannot join
- Presence of co morbid non-Rett related disease,
- History of adverse reaction/hypersensitivity to statins,
- Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,
- Active liver disease,
- Concomitant use of strong CYP3A4 inhibitors,
- Condition predis-posing to renal failure secondary to rhabdomyolysis (scoliosis surgery within 6 months),
- Oral contraceptives use.
Trial locations and contacts
1 site in 1 country.
United States1 site
Montefiore Medical center
The Bronx, New York, 10467
About this study
Official title: Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
Read the detailed description
Excess neuronal cholesterol plays a role in the pathophysiology of Rett syndrome(RTT) and the investigators hypothesise that inhibition of cholesterol synthesis in the CNS will reduce neuronal cholesterol and lead to improvement in Rett syndrome related symptoms.
Goal: To determine the optimal dosing regiment of Lovastatin for patients with RTT and to identify the most appropriate primary outcome measure for the subsequent Phase 3 study.
Phase 2, dose escalation study.
Primary outcome: Gait speed Secondary outcomes: respiratory function, cognition, EEG (encephalopathy), Rett syndrome severity scale, neurodevelopmental assessment (QOL)
20 ambulatory female patients with genetically confirmed.
- Conditions
- Rett Syndrome
- Being tested
- Lovastatin
- Keywords
- Treatment trial, Lovastatin
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LovastatinDrug
Also called Mevacor
dose escallating
| Study group | Role | What they receive |
|---|---|---|
| Open label | Experimental | Treatment with Lovastatin, dose escalating trial according to the following schedule:
10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks. Receives: Drug: Lovastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gait Velocity as Measured by GAITRite System
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
Measured: During final week of treatment, week 32
Secondary outcomes (1)
Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System
Novelty score is considered a composite measure of visual memory.Test is a standardized neuropsychological test( "Rose test") adapted for eye tracking system.
The present study made use of a well-established battery of "visual paired comparison" problems .There were nine problems: five using achromatic photos of faces and four using multicolored abstract patterns paired stimuli , separated by 23°. For each problem, two identical stimuli are briefly presented side by side for familiarization, then the familiar and a new one are paired on test
Recognition(memory) is indexed by a novelty score (percentage of looking to the novel target on test).
Higher novelty scores were related to better recognition (index of better memory) Scores range from 0.0-1.0 (0-100%) and indicate 1) more overall looking at novel target and (2) more fixations to novel targets.
Score of .5 (50%) indicates looking "by change" only with no recognition.
Measured: final week of treatment, Week 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2015 (estimated)
- Study startJul 2015
- Primary completionJul 2016
- Study completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- More trials run by Montefiore Medical CenterThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Rett Syndrome Research Trust
- Responsible party
- Principal investigator, Aleksandra Djukic, Montefiore Medical Center
- Investigators
- Aleksandra Djukic, MD PhD, Principal investigator, Montefiore Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02563860
- Sponsor's protocol ID
- 2014-4253
- Acronym
- Statins
- Status last verified
- Jul 2019
- First submitted
- 20 Jul 2015
- Last updated
- 12 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)
- ClinicalTrials.gov ID
- NCT02563860
- Acronym / sponsor ID
- Statins / 2014-4253
- Conditions
- Rett Syndrome
- Keywords
- Treatment trial, Lovastatin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- Collaborators
- Rett Syndrome Research Trust
- Study start
- Jul 2015
- Primary completion
- Jul 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 30 Sep 2015 (estimated)
- Results
- Posted 7 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
Detailed description
Excess neuronal cholesterol plays a role in the pathophysiology of Rett syndrome(RTT) and the investigators hypothesise that inhibition of cholesterol synthesis in the CNS will reduce neuronal cholesterol and lead to improvement in Rett syndrome related symptoms.
Goal: To determine the optimal dosing regiment of Lovastatin for patients with RTT and to identify the most appropriate primary outcome measure for the subsequent Phase 3 study.
Phase 2, dose escalation study.
Primary outcome: Gait speed Secondary outcomes: respiratory function, cognition, EEG (encephalopathy), Rett syndrome severity scale, neurodevelopmental assessment (QOL)
20 ambulatory female patients with genetically confirmed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Experimental | Drug: LovastatinTreatment with Lovastatin, dose escalating trial according to the following schedule: 10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks. |
Who may qualify
- Ages
- 3 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Females patients,
- Genetically confirmed RTT,
- Ambulatory.
Key exclusion criteria (7 listed)
- Presence of co morbid non-Rett related disease,
- History of adverse reaction/hypersensitivity to statins,
- Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,
- Active liver disease,
- Concomitant use of strong CYP3A4 inhibitors,
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 |
|---|---|---|
| LovastatinAlso: Mevacordose escallating | Drug | Open label |
Primary outcome
Gait Velocity as Measured by GAITRite System
Time frame: During final week of treatment, week 32
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
1 secondary outcome
Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System
Time frame: final week of treatment, Week 32
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Aleksandra Djukic, MD PhD principal investigator
Montefiore Medical Center
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 Lovastatin in people with Rett Syndrome, run by Montefiore Medical Center at 1 site in United States.
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 3 inclusion and 7 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 12 Jul 2019. 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 United States. 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. 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?
Yes. Results are posted in the registry (first posted 7 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Gait Velocity as Measured by GAITRite System (time frame: During final week of treatment, week 32). 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 12 Jul 2019.