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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects were recruited from Mayo Clinic, Rochester between October 2006 and September 2008.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Fifteen subjects were enrolled, but 2 subjects withdrew prior to group assignment.

Reporting Groups
  Description
Betaine First, Then Placebo 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.
Placebo First, Then Betaine 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.

Participant Flow for 3 periods

Period 1:   First Intervention
    Betaine First, Then Placebo   Placebo First, Then Betaine
STARTED   6   7 
COMPLETED   5   5 
NOT COMPLETED   1   2 
Subject non-compliant                1                1 
Withdrawal by Subject                0                1 

Period 2:   Washout Period of 2 Months
    Betaine First, Then Placebo   Placebo First, Then Betaine
STARTED   5   5 
COMPLETED   5   5 
NOT COMPLETED   0   0 

Period 3:   Second Intervention
    Betaine First, Then Placebo   Placebo First, Then Betaine
STARTED   5   5 
COMPLETED   5   5 
NOT COMPLETED   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Entire Study Population Includes groups randomized to receive placebo first and betaine first.

Baseline Measures
   Entire Study Population 
Overall Participants Analyzed 
[Units: Participants]
 13 
Age 
[Units: Years]
Mean (Full Range)
 20.1 
 (6 to 43) 
Gender 
[Units: Participants]
 
Female   7 
Male   6 
Region of Enrollment 
[Units: Participants]
 
United States   13 
Glomerular Filtration Rate (GFR) 
[Units: Ml/min/1.73 m^2]
Mean (Full Range)
 79 
 (39 to 134) 
Number of Subjects on Concomitant Medications [1] 
[Units: Participants]
 
Vitamin B6   9 
Neutral Phosphate   7 
Citrate   3 
[1] 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.


  Outcome Measures

1.  Primary:   Urinary Oxalate Excretion   [ Time Frame: baseline, 2 months, 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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
e-mail: milliner.dawn@mayo.edu


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