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

Tracking Information
First Received Date ^ICMJE	January 20, 2011
Last Updated Date	October 26, 2012
Start Date ^ICMJE	December 2010
Primary Completion Date	September 2012 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: January 21, 2011)	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 ]
Original Primary Outcome Measures ^ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01281878 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE	Not Provided
Original Secondary Outcome Measures ^ICMJE	Not Provided
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	Not Provided

Descriptive Information
Brief Title ^ICMJE	Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
Official Title ^ICMJE	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
Brief Summary	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.
Detailed Description	Not Provided
Study Type ^ICMJE	Interventional
Study Phase	Phase 2
Study Design ^ICMJE	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Condition ^ICMJE	Primary Hyperoxaluria Type I
Intervention ^ICMJE	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.
Study Arm (s)	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
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Enrollment ^ICMJE	12
Completion Date	September 2012
Primary Completion Date	September 2012 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	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
Gender	Both
Ages	5 Years to 60 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	Germany

Administrative Information
NCT Number ^ICMJE	NCT01281878
Other Study ID Numbers ^ICMJE	Uni-Koeln-1251
Has Data Monitoring Committee	No
Responsible Party	Prof. Dr. B. Hoppe, University of Cologne
Study Sponsor ^ICMJE	University of Cologne
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	University of Cologne
Verification Date	October 2012
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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