Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Hyperoxaluria
About this trial
This is an interventional treatment trial for Hyperoxaluria focused on measuring Primary Hyperoxaluria
Eligibility Criteria
Inclusion Criteria: A definitive diagnosis of Type 1 Primary Hyperoxaluria (PHI) as confirmed by hepatic angiotensinogen (AGT) deficiency, biochemical criteria (marked hyperoxaluria and hyperglycolic aciduria) or mutation analysis (having a known PHI mutation) Alanine-glyoxylate aminotransferase (AGXT) genotype known Hyperoxaluria not fully corrected by 3 months of continuous Vitamin B6 (VB6) at doses of 8 mg/kg/d or more Males or females, 6-70 years of age, inclusive Preserved renal function, as defined by measured glomerular filtration rate (GFR) > 30 ml/min/1.73 m^2 Sexually active female patients of childbearing potential must practice adequate contraception during the treatment period and for 6 months after discontinuation of therapy. A pregnancy test obtained at entry prior to the initiation of treatment must be negative. Female patients must not be breast-feeding. Sexually active male patients must practice acceptable methods of contraception during the treatment period and for 6 months after discontinuation of therapy. Written informed consent for participation in this study. Exclusion Criteria: Patients who are fully VB6 responsive (i.e., G170R homozygotes). Prior recipients of liver transplantation performed for correction of AGT deficiency. Pregnancy or breastfeeding Unwillingness of patient and/or partner to use contraception during treatment. Malignant disease (other than non-melanoma skin cancer) in the previous two years. Markedly reduced renal function (Stage IV Chronic Kidney Disease or measured or estimated GFR < 30 ml/min/1.73 m^2) Allergy to betaine or related compounds History of papilledema or increased intracranial pressure.
Sites / Locations
- Mayo Clinic Hyperoxaluria Center
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Betaine
Placebo
Subjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral lactose placebo, in two doses daily, for 2 months.
Subjects were randomly assigned to receive oral lactose placebo, in two doses daily, for 2 months. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses, for 2 months.