Pharmacological Treatment of Rett Syndrome With Statins
| Status: | Recruiting |
|---|---|
| Conditions: | Other Indications, Neurology |
| Therapuetic Areas: | Neurology, Other |
| Healthy: | No |
| Age Range: | 3 - Any |
| Updated: | 4/21/2016 |
| Start Date: | July 2015 |
| End Date: | February 2017 |
| Contact: | Aleksandra Djukic |
| Email: | adjukic@montefiore.org |
| Phone: | 718 920 4378 |
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.
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.
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.
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.
We found this trial at
1
site
3550 Jerome Avenue
Bronx, New York 10467
Bronx, New York 10467
(718) 920-4321
Phone: 718-920-4378
Montefiore Medical Center As the academic medical center and University Hospital for Albert Einstein College...
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