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

NCT ID: NCT01281878

Last Updated: 2012-10-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-12-31
• Study Completion Date: 2012-09-30

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.

Conditions

Primary Hyperoxaluria Type I

Keywords

primary hyperoxaluria; Pyridoxal-phosphate

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

Vitamin B 6

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Cologne

OTHER

Sponsor Role: lead

Responsible Party

Prof. Dr. B. Hoppe

Prof. Dr. med. Bernd Hoppe

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Children´s Hospital University of Cologne

Cologne, North Rhine-Westphalia, Germany

Countries

Germany

References

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.

Reference Type: DERIVED

PMID: 24385516 (View on PubMed)

Other Identifiers

Uni-Koeln-1251

Identifier Type: -

Identifier Source: org_study_id