Phase 2
Completed N=20
Pharmacological Treatment of Rett Syndrome With Statins
Source: ClinicalTrials.gov NCT02563860 ↗Enrolled (actual)
20
Serious AEs
0.0%
Results posted
May 2019
Primary outcomePrimary: Gait Velocity as Measured by GAITRite System — 58.6 cm/sec
Summary
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
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 | — |
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. Informational only — not medical advice.