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NCT Number: NCT01281878

Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate

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.

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Key information

Conditions

Primary Hyperoxaluria Type I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hyperoxaluria Hyperoxaluria, Primary Kidney Diseases Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Urogenital Diseases Urologic Diseases

Age range

5 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Children´s Hospital University of Cologne

Cologne, North Rhine-Westphalia, 50931, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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
  • 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
  • Written informed consent from patients and/or legally acceptable representatives

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. 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

Treatment and study plan

Vitamin B 6

Drug

Oral solution of pyridoxal phosphate start with 5mg per kg body weight per day in two dosages over 6 weeks, increase stepwise by 5mg/kg body weight every 6 weeks up to 20 mg/kg body weight/d.

Primary outcomes

  1. 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.

    Time frame: 6 month

Sponsors and collaborators

Lead sponsor

University of Cologne

Other

Registry information

Official study title

PILOTSTUDIE ZUR PYRIDOXALPHOSPHATTHERAPIE BEI PATIENTEN MIT PRIMÄRER HYPEROXALURIE TYP I (PHOX-B6-PILOT) Pilot Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate

Acronym: PHOX-B6-Pilot

Important dates

Study start
2010
Primary completion
2012
Study completion
2012
First posted
Jan 24, 2011
Registry last updated
Oct 29, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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