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Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate (PHOX-B6-Pilot)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Prof. Dr. B. Hoppe, University of Cologne
ClinicalTrials.gov Identifier:
NCT01281878
First received: January 20, 2011
Last updated: October 26, 2012
Last verified: October 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Primary Hyperoxaluria Type I
 Drug: Vitamin B 6
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:

MedlinePlus related topics: B Vitamins
U.S. FDA Resources

Further study details as provided by University of Cologne:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 12
Study Start Date: December 2010
Study Completion Date: September 2012
Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pyridoxal-phosphate
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
 Drug: Vitamin B 6
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.

  Eligibility

Ages Eligible for Study:   5 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01281878

Locations
Germany
Children´s Hospital University of Cologne
Cologne, NRW, Germany, 50931
Sponsors and Collaborators
University of Cologne
  More Information

No publications provided

Responsible Party: Prof. Dr. B. Hoppe, Prof. Dr. med. Bernd Hoppe, University of Cologne
ClinicalTrials.gov Identifier: NCT01281878     History of Changes
Other Study ID Numbers: Uni-Koeln-1251
Study First Received: January 20, 2011
Last Updated: October 26, 2012
Health Authority: Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by University of Cologne:
primary hyperoxaluria
Pyridoxal-phosphate

Additional relevant MeSH terms:
Hyperoxaluria
Hyperoxaluria, Primary
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Pyridoxal
 Pyridoxine
Pyridoxal Phosphate
Vitamin B 6
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 23, 2013