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

ZAMBON GROUP0 个研究点目标入组 22 人开始时间: 2007年10月5日最近更新: 2012年3月19日

研究概览

暂无简介。

•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.

发起方

ZAMBON GROUP