Trial Outcomes & Findings for Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria (NCT NCT00283387)
NCT ID: NCT00283387
Last Updated: 2013-12-16
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
baseline, 2 months, 6 months
Results posted on
2013-12-16
Participant Flow
Subjects were recruited from Mayo Clinic, Rochester between October 2006 and September 2008.
Fifteen subjects were enrolled, but 2 subjects withdrew prior to group assignment.
Participant milestones
| Measure |
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.
|
|---|---|---|
|
First Intervention
STARTED
|
6
|
7
|
|
First Intervention
COMPLETED
|
5
|
5
|
|
First Intervention
NOT COMPLETED
|
1
|
2
|
|
Washout Period of 2 Months
STARTED
|
5
|
5
|
|
Washout Period of 2 Months
COMPLETED
|
5
|
5
|
|
Washout Period of 2 Months
NOT COMPLETED
|
0
|
0
|
|
Second Intervention
STARTED
|
5
|
5
|
|
Second Intervention
COMPLETED
|
5
|
5
|
|
Second Intervention
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|---|
|
First Intervention
Subject non-compliant
|
1
|
1
|
|
First Intervention
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Baseline characteristics by cohort
| Measure |
Entire Study Population
n=13 Participants
Includes groups randomized to receive placebo first and betaine first.
|
|---|---|
|
Age, Continuous
|
20.1 years
n=93 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=93 Participants
|
|
Glomerular Filtration Rate (GFR)
|
79 ml/min/1.73 m^2
n=93 Participants
|
|
Number of Subjects on Concomitant Medications
Vitamin B6
|
9 participants
n=93 Participants
|
|
Number of Subjects on Concomitant Medications
Neutral Phosphate
|
7 participants
n=93 Participants
|
|
Number of Subjects on Concomitant Medications
Citrate
|
3 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: baseline, 2 months, 6 monthsPopulation: 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.
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.
Outcome measures
| Measure |
Betaine
n=10 Participants
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.
|
Placebo
n=10 Participants
Subjects received oral lactose placebo divided in two doses daily, for 2 months.
|
|---|---|---|
|
Urinary Oxalate Excretion
|
1.43 umol/mg
Standard Deviation 0.97
|
1.04 umol/mg
Standard Deviation 0.71
|
Adverse Events
Betaine
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Betaine
n=13 participants at risk
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.
|
Placebo
n=13 participants at risk
Subjects received oral lactose placebo divided in two doses daily, for 2 months.
|
|---|---|---|
|
Gastrointestinal disorders
Dyspepsia
|
76.9%
10/13 • Number of events 10 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
53.8%
7/13 • Number of events 7 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
|
Gastrointestinal disorders
Diarrhea/loose stools
|
46.2%
6/13 • Number of events 6 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
46.2%
6/13 • Number of events 6 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13 • Number of events 2 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
7.7%
1/13 • Number of events 1 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
|
General disorders
Headache
|
30.8%
4/13 • Number of events 4 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
7.7%
1/13 • Number of events 1 • Subjects will be followed for adverse events for the 6 months while on study.
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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place