Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
- Registration Number
- NCT01281878
- Lead Sponsor
- University of Cologne
- Brief Summary
In this study the investigators will prospectively analyze the reduction of urinary oxalate excretion under the treatment with PLP in dosages of 5mg/kg/day up to 20 mg/kg/day and serum level response relationship with PLP as an i.v. solution used orally in 12 patients with primary hyperoxaluria type I as an inherited autosomal-recessive-disorder leading to increased endogenous oxalate production, urolithiasis and end stage renal disease.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 12
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Documentation of diagnosis of PH I by any one of the following:
- Liver biopsy confirmation of deficient liver specific peroxisomal alanine-glyoxylate aminotransferase, (AGT or mislocalization of AGT from peroxisomes to mitochondria)
- Homozygosity or compound heterozygosity for a known mutation in the causative gene (AGXT) for PH I
-
Male or female subjects between 5 years and 60 years of age
-
Renal function defined as an estimated GFR > 60 ml/min normalized to 1.73 m2 body surface area
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Subjects receiving pyridoxal-phosphate before the study must be willing to discontinue therapy with pyridoxal-phosphate for a wash out phase of at least 4 weeks but always until normalization of serum pyridoxal-phosphate levels
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Written informed consent from patients and/or legally acceptable representatives
- Pregnant or lactating women
- Women of child-bearing potential who are not using a highly effective contraception method with a pearl-index < 1. Highly effective contraception methods are oral, transdermal, injectable, or implanted contraceptives, IUD, abstinence, or sterile sexual partner and must agree to continue using such precautions during the pyridoxal-phosphate study
- Subjects post liver or kidney transplantation or combined transplantation
- Chronic diarrhoea with the risk of malabsorption
- Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator, is indicative of a disease that would compromise the safety taking pyridoxal-phosphate per os and the absorption
- Subjects participating in other clinical trials with investigational products 4 weeks prior to trial entry, during the trial and 4 weeks after the trial
- Subjects who are unable to take the trial medication
- Subjects who are unable to collect 24-hour urine samples or follow other study procedures
- Subjects who are under treatment with L-Dopa, Isoniazid, D-Penicillamine (interactions between these drugs and pyridoxal-phosphate are known and might influence serum pyridoxal-phosphate levels)
- Subjects with known allergies to substances of contents (e.g. Potassium sorbet, raspberry syrup)
- Subjects confined to an institution on judicial or official behalf
- Subjects who are in dependency to the sponsor or the PI of the trial
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Pyridoxal-phosphate Vitamin B 6 Treatment with pyridoxal-phosphate in increasing dosages every six weeks starting with 5mg/kg body weight up to 20 mg/kg body weight. treatment duration 24 weeks
- Primary Outcome Measures
Name Time Method The primary endpoint of the study is the reduction of the urinary oxalate excretion (percentage change in urinary oxalate, expressed as mmol/1.73 m2 /day) at week 24 compared to baseline. 6 month
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Children´s Hospital University of Cologne
🇩🇪Cologne, NRW, Germany