ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    12777626 [PUBMED-IDS]
Previous Study | Return to List | Next Study

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

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.
ClinicalTrials.gov Identifier: NCT00283387
Recruitment Status : Completed
First Posted : January 27, 2006
Results First Posted : January 1, 2013
Last Update Posted : December 16, 2013
Sponsor:
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Orphan Europe
Information provided by:
Mayo Clinic

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Hyperoxaluria
Interventions Drug: Betaine
Drug: Placebo
Enrollment 15

Recruitment Details Subjects were recruited from Mayo Clinic, Rochester between October 2006 and September 2008.
Pre-assignment Details Fifteen subjects were enrolled, but 2 subjects withdrew prior to group assignment.
Arm/Group Title Betaine First, Then Placebo Placebo First, Then Betaine
Hide Arm/Group Description Subjects received oral betaine 10 gm (subjects >10 yrs old) or 6 gm (subjects <10 yrs old) divided in two doses daily, for 2 months, followed by a 2 month washout period. Subjects then received oral lactose placebo divided in two doses daily, for 2 months. Subjects received oral lactose placebo divided in two doses daily, for 2 months, followed by a 2 month washout period. Subjects then received oral betaine 10 gm (subjects >10 yrs old) or 6 gm (subjects <10 yrs old), divided in two doses daily, for 2 months.
Period Title: First Intervention
Started 6 7
Completed 5 5
Not Completed 1 2
Reason Not Completed
Subject non-compliant             1             1
Withdrawal by Subject             0             1
Period Title: Washout Period of 2 Months
Started 5 5
Completed 5 5
Not Completed 0 0
Period Title: Second Intervention
Started 5 5
Completed 5 5
Not Completed 0 0
Arm/Group Title Entire Study Population
Hide Arm/Group Description Includes groups randomized to receive placebo first and betaine first.
Overall Number of Baseline Participants 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 13 participants
20.1
(6 to 43)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants
Female
7
  53.8%
Male
6
  46.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 13 participants
13
Glomerular Filtration Rate (GFR)  
Mean (Full Range)
Unit of measure:  Ml/min/1.73 m^2
Number Analyzed 13 participants
79
(39 to 134)
Number of Subjects on Concomitant Medications   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 13 participants
Vitamin B6 9
Neutral Phosphate 7
Citrate 3
[1]
Measure Description: Usual medications for primary hyperoxaluria were continued throughout the study. Subjects could have been prescribed more than one medication, so the sum of participants will be greater than the subject population.
1.Primary Outcome
Title Urinary Oxalate Excretion
Hide Description

The patients were randomly assigned oral betaine or placebo for 2 months, followed by a 2 month washout. Each patient then received the alternate study medication for 2 months.

Urinary Oxalate Excretion was measured by oxalate oxidase. Two 24 hour urine collections were obtained at baseline, and during the eighth week of each study period.

Time Frame baseline, 2 months, 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Per protocol analysis: 10 of 15 enrolled PHI subjects completed the study: 2 withdrew before initiation, 2 were noncompliant, in 1 symptoms led to withdrawal.
Arm/Group Title Betaine Placebo
Hide Arm/Group Description:
Subjects received oral betaine 10 gm (subjects >10 yrs old) or 6 gm (subjects <10 yrs old), divided in two doses daily, for 2 months.
Subjects received oral lactose placebo divided in two doses daily, for 2 months.
Overall Number of Participants Analyzed 10 10
Mean (Standard Deviation)
Unit of Measure: umol/mg
1.43  (0.97) 1.04  (0.71)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Betaine, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.007
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Time Frame Subjects will be followed for adverse events for the 6 months while on study.
Adverse Event Reporting Description Subjects unable to tolerate the initial dose due to gastrointestinal symptoms will be offered a dose reduction of 3 - 5 g/day. Monitoring for symptoms and signs of cerebral edema will be 1) weekly telephone calls from the study coordinator with a list of questions re: increased intracranial pressure and 2) full eye exam before and after treatment.
 
Arm/Group Title Betaine Placebo
Hide Arm/Group Description Subjects received oral betaine 10 gm (subjects >10 yrs old) or 6 gm (subjects <10 yrs old), divided in two doses daily, for 2 months. Subjects received oral lactose placebo divided in two doses daily, for 2 months.
All-Cause Mortality
Betaine Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Betaine Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/13 (0.00%)      0/13 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Betaine Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/13 (84.62%)      8/13 (61.54%)    
Gastrointestinal disorders     
Dyspepsia   10/13 (76.92%)  10 7/13 (53.85%)  7
Diarrhea/loose stools   6/13 (46.15%)  6 6/13 (46.15%)  6
Constipation   2/13 (15.38%)  2 1/13 (7.69%)  1
General disorders     
Headache   4/13 (30.77%)  4 1/13 (7.69%)  1
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Dawn S. Milliner
Organization: Mayo Clinic
Phone: 507-284-7431
Publications:
Danpure CJ and Rumsby G. Enzymology and molecular genetics of primary hyperoxaluria type 1. Consequences for clinical management. In: Calcium Oxalate in Biological Systems, edited by Khan SR. Boca Raton, FL: CRC Press, 1995, p. 189-205.
Micromedex Healthcare Series (http://wwwmicromed.mayo.edu).
Berlow S, Bachman RP, Berry GT, Donnell GN, Grix A, Levitsky LL, Hoganson G, Levy HL. Betaine therapy in homocystinemia. Brain Dysfunct 2:10-24, 1989.
Responsible Party: Dr. Carla G. Monico, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00283387     History of Changes
Other Study ID Numbers: 2147-05
5R01DK073354-04 ( U.S. NIH Grant/Contract )
First Submitted: January 26, 2006
First Posted: January 27, 2006
Results First Submitted: November 1, 2012
Results First Posted: January 1, 2013
Last Update Posted: December 16, 2013