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 - ND
- Conditions
- HomocystinuriaMedDRA version: 6.1Level: PTClassification code 10020365
- Registration Number
- EUCTR2006-000614-21-IT
- Lead Sponsor
- ZAMBON GROUP
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 22
signed informed consent (patient and countersigned by Legal responsible in case of patients below age of consent);
≥16 years of age;
classical (non-pyridoxine responsive) homocystinuria (CBS deficiency);
stable clinical condition;
willing to complete all phases and all procedures of the study;
on B6, B12 and betaine and FA treatment;
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.
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
anticipated need of multivitamin supplements, folic acid or creatine treatment throughout the duration of the study
episodes of thrombosis and/or embolism one year before inclusion in the study
history of drug and/or alcohol abuse with 6 months of baseline
serious undercurrent illness(as) 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
donation or receipt of blood or blood products (within 2 months before baseline)
pregnant or lactating female
Investigative drug trials: participation in the evaluation of any drug within 3 months prior to the start of the study
substantial changes in eating habits within the past 4 weeks.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To investigate the efficacy and safety of a 3 month 5-MTHF treatment in terms of decreasing total plasma homocysteine (tHcy) in homocystinuric patients, while co-administered with an individual established therapy as a folic acid substitute.;Secondary Objective: ;Primary end point(s): Reduction from baseline of tHcy in plasma induced by 3 months treatment with 5-MTHF 15 mg. Baseline will be defined as the tHcy levels observed after the wash-out phase, before 5-MTHF first intake.
- Secondary Outcome Measures
Name Time Method