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
  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:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

ClinicalTrials.gov processed this record on October 01, 2014