Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4

• Conditions: CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy), is a rare autosomal dominant disorder characterized by recurrent strokes starting in mid-adulthood and leading in some to severe motor disability with pseudobulbar palsy and dementia of the subcortical type; MedDRA version: 9.1Level: HLGTClassification code 10008804Term: Chromosomal abnormalities and abnormal gene carriers

• Registration Number: EUCTR2007-004370-55-IT
• Lead Sponsor: AZIENDA OSPEDALIERA OSPEDALE NIGUARDA CA' GRANDA (A.O. DI RILIEVO NAZIONALE)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-12-11
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

- Age > 30 and ≤ 65 years
- Diagnosis of CADASIL confirmed by the identification of a mutation in the Notch3 gene
- Informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Dementia (DSM-IV criteria) with a SIDAM score <33
-Autoimmune disorders
-Pregnancy, nursing or childbearing potential not on adequate contraception
-Myocardial infarction, cerebral vascular accident, or pulmonary embolism during the 3 months prior to Screening.
-Severe uncontrolled hypertension (BP >180 mmHg systolic and /or 110 mmHg diastolic).
-Hypotension at Screening, defined as seated resting BP values of < 100 mmHg systolic or < 55 mmHg diastolic, or as symptomatic (orthostatic) hypotension
-Serum creatinine >2.5 mg/dL or hepatic enzymes >2X normal
-Pacemakers, implantable cardioverter defibrillators or metallic implants not compatible with MRI scanning
-Concomitant treatment with methotrexate, levodopa, phosphodiesterase-3 (milrinone) or phosphodiesterase -5 inhibitors (sildenafil, taladafil, vardenafil) , pentoxyfylline, nitrate/nitrite-based vasodilators, L-arginine, or dietary supplements containing L-arginine or gingko biloba within 30 days prior to Screening, or anticipated need for treatment with any of these agents during the course of the study.
-Participation in other drug trials within the last 30 days before start for the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified