In this study we 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. MedDRA version: 12.1 Level: LLT Classification code 10037671 Term: Pyrid
Conditions
Interventions
Product Name: B6-Solution
Pharmaceutical Form: Oral solution
CAS Number: 447052
Current Sponsor code: Uni-Koeln-1251
Other descriptive name: PYRIDOXINE PHOSPHATE
Concentration unit: mg/ml milligram(s)
Sponsors
University of Cologne
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •The drug-response-relationship of pyridoxal-phosphate will be checked by repeated measurements of the serum pyridoxal-phosphate levels • Reduction of the plasma oxalate level as change in plasma oxalate concentration from baseline to week 6, 12, 18 and 24 •Reduction of the urinary oxalate excretion in patients with PH I at weeks 6, 12, 18 compared to baseline. •The safety of the oral application of an i.v. pyridoxal-phosphate solution will be determined. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • 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. Potassiumsorbet, raspberry sirupe) • Subjects confined to an institution on judicial or official behalf. • Subjects who are in dependency to the sponsor or the PI of the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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. The excess of endogenously produced oxalate is excreted via the urine in patients with primary hyperoxaluria. Because of the low solubility of oxalate as calcium salt, a high urinary oxalate excretion results in urolithiasis and/or progressive nephrolithiasis. Reduction of the urinary oxalate level is the standard measurement of treatment efficacy in patients with PH I. ;Secondary Objective: •The drug-response-relationship of pyridoxal-phosphate will be checked by repeated measurements of the serum pyridoxal-phosphate levels • Reduction of the plasma oxalate level as change in plasma oxalate concentration from baseline to week 6, 12, 18 and 24 •Reduction of the urinary oxalate excretion in patients with PH I at weeks 6, 12, 18 compared to baseline. •The safety of the oral application of an i.v. pyridoxal-phosphate solution will be determined. ;Primary end point(s): 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. The excess of endogenously produced oxalate is excreted via the urine in patients with primary hyperoxaluria. Because of the low solubility of oxalate as calcium salt, a high urinary oxalate excretion results in urolithiasis and/or progressive nephrolithiasis. Reduction of the urinary oxalate level is the standard measurement of treatment efficacy in patients with PH I. | — |
Countries
Germany
Outcome results
None listed