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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01281878
Recruitment Status : Completed
First Posted : January 24, 2011
Last Update Posted : October 29, 2012
Information provided by (Responsible Party):
Prof. Dr. B. Hoppe, University of Cologne

January 20, 2011
January 24, 2011
October 29, 2012
December 2010
September 2012   (Final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01281878 on Archive Site
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Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
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
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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Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Primary Hyperoxaluria Type I
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.
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
Intervention: Drug: Vitamin B 6
Hoyer-Kuhn H, Kohbrok S, Volland R, Franklin J, Hero B, Beck BB, Hoppe B. Vitamin B6 in primary hyperoxaluria I: first prospective trial after 40 years of practice. Clin J Am Soc Nephrol. 2014 Mar;9(3):468-77. doi: 10.2215/CJN.06820613. Epub 2014 Jan 2.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
September 2012
September 2012   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
5 Years to 60 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
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Prof. Dr. B. Hoppe, University of Cologne
University of Cologne
Not Provided
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University of Cologne
October 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP