Phase 2 N=13 Randomized Triple-blind Treatment

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

Hyperoxaluria

Bottom Line

View on ClinicalTrials.gov: NCT00283387 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jan 2013

Primary outcome: Primary: Urinary Oxalate Excretion — 1.43; 1.04 umol/mg — p=0.007

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Betaine (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 6+ yrs
Sex: All
Sponsor: Mayo Clinic
Primary completion: Sep 2011

Outcome Measures

Outcome	Result	p-value
PRIMARY Urinary Oxalate Excretion	1.43; 1.04	0.007 sig

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 urine oxalate excretion in Primary Hyperoxaluria Type I patients.

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00283387). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.