Status:
COMPLETED
Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Lead Sponsor:
Mayo Clinic
Collaborating Sponsors:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Orphan Europe
Conditions:
Hyperoxaluria
Eligibility:
All Genders
6-70 years
Phase:
PHASE2
Brief Summary
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 ...
Detailed Description
Our prior genotyping results have shown an association between the G170R allele and the clinical response to VB6. Patients homozygous for this change show a complete response and heterozygous patients...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2011
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00283387
Start Date
February 1 2007
End Date
September 1 2011
Last Update
December 16 2013
Active Locations (1)
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1
Mayo Clinic Hyperoxaluria Center
Rochester, Minnesota, United States, 55905