A multicentre, open-label study to evaluate the efficacy and safety of 5-MTHF administration, added on to the individual established therapy, on plasma homocysteine levels in patients with congenital homocystinuria

• Conditions: homocystinuria; MedDRA version: 8.1Level: prefClassification code 10020365

• Registration Number: EUCTR2006-000614-21-AT
• Lead Sponsor: Zambon Group S.p.A

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2006-06-16
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1.signed informed consent (patient or Legal responsible)
2.>16 years of age
3.classical (non-pyridoxine responsive) homocystinuria (CBS deficiency)
4.stable clinical condition
5.willing to complete all phases and all procedures of the study
6.on B6, B12, betaine and folic acid treatment
7.ability to comprehend the full nature and purpose of the study, including possible risks and side effects; ability to co-operate with the Investigator and to comply with the requirements of the entire study; signed written informed consent prior to inclusion in the study

Are the trial subjects under 18? yes
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

1.patients taking multivitamin supplements, folic acid or creatine treatment at inclusion and throughout the duration of the study
2.episodes of thrombosis and/or embolism one year before inclusion in the study
3.history of drug and/or alcohol abuse during the 6 months before the beginning of the study
4.patients with serious illness(es) or diseases (e.g., haematological, renal, hepatic, respiratory, endocrine, psychiatric) that may interfere with, or put patients at additional risk for their ability to receive the treatment outlined in the protocol
5.donation or receipt of blood or blood products (within 2 months before baseline)
6.pregnant or lactating female

Study & Design

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