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

Primary Purpose

Primary Hyperoxaluria Type I

Status
Completed
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
Vitamin B 6
Sponsored by
University of Cologne
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Hyperoxaluria Type I focused on measuring primary hyperoxaluria, Pyridoxal-phosphate

Eligibility Criteria

5 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children´s Hospital University of Cologne

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Pyridoxal-phosphate

Arm Description

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

Outcomes

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.

Secondary Outcome Measures

Full Information

First Posted
January 20, 2011
Last Updated
October 26, 2012
Sponsor
University of Cologne
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1. Study Identification

Unique Protocol Identification Number
NCT01281878
Brief Title
Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
Acronym
PHOX-B6-Pilot
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2012
Overall Recruitment Status
Completed
Study Start Date
December 2010 (undefined)
Primary Completion Date
September 2012 (Actual)
Study Completion Date
September 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Cologne

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria Type I
Keywords
primary hyperoxaluria, Pyridoxal-phosphate

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pyridoxal-phosphate
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Vitamin B 6
Intervention Description
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 Outcome Measure Information:
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Children´s Hospital University of Cologne
City
Cologne
State/Province
NRW
ZIP/Postal Code
50931
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
24385516
Citation
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.
Results Reference
derived

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

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