Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Completed · Phase 2 · Has a placebo group
Conditions studied: Hyperoxaluria
In brief
The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients. Hypothesis: Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
Key facts
- Study ID
- NCT00283387
- Run by
- Mayo Clinic
- People needed
- 15
- Starts
- 2007-02-01
- Expected to finish
- 2011-09-01
- Last updated by the study team
- 2013-12-16
Who can join
Age: 6 and older, up to 70. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- Mayo Clinic Hyperoxaluria Center — Rochester, Minnesota, United States
Full record on ClinicalTrials.gov
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