Phase 2
N=20
Pharmacological Treatment of Rett Syndrome With Statins
Rett Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02563860 ↗Enrolled (actual)
20
Serious AEs
0.0%
Results posted
May 2019
Primary outcome: Primary: Gait Velocity as Measured by GAITRite System — 58.6 cm/sec
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Lovastatin (Drug)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- Female
- Sponsor
- Montefiore Medical Center
- Primary completion
- Jul 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Gait Velocity as Measured by GAITRite System |
58.6 | — |
| SECONDARY Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System |
44.85; 58.93 | — |
Summary
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
Eligibility Criteria
Inclusion Criteria
- Females patients,
- Genetically confirmed RTT,
- Ambulatory.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT02563860). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.