HyperoxaluriaPhase 2NCT00283387
Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
A Phase 2 study testing Betaine in people with Hyperoxaluria, run by Mayo Clinic at 1 site in United States.
Trial summary
The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients. Hypothesis: Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Hyperoxaluria
- What is being tested?
- Betaine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2007to Sep 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A definitive diagnosis of Type 1 Primary Hyperoxaluria (PHI) as confirmed by hepatic angiotensinogen (AGT) deficiency, biochemical criteria (marked hyperoxaluria and hyperglycolic aciduria) or mutation analysis (having a known PHI mutation)
- Alanine-glyoxylate aminotransferase (AGXT) genotype known
- Hyperoxaluria not fully corrected by 3 months of continuous Vitamin B6 (VB6) at doses of 8 mg/kg/d or more
- Males or females, 6-70 years of age, inclusive
- Preserved renal function, as defined by measured glomerular filtration rate (GFR) > 30 ml/min/1.73 m^2
- Sexually active female patients of childbearing potential must practice adequate contraception during the treatment period and for 6 months after discontinuation of therapy. A pregnancy test obtained at entry prior to the initiation of treatment must be negative. Female patients must not be breast-feeding. Sexually active male patients must practice acceptable methods of contraception during the treatment period and for 6 months after discontinuation of therapy.
- Written informed consent for participation in this study.
Who cannot join
- Patients who are fully VB6 responsive (i.e., G170R homozygotes).
- Prior recipients of liver transplantation performed for correction of AGT deficiency.
- Pregnancy or breastfeeding
- Unwillingness of patient and/or partner to use contraception during treatment.
- Malignant disease (other than non-melanoma skin cancer) in the previous two years.
- Markedly reduced renal function (Stage IV Chronic Kidney Disease or measured or estimated GFR < 30 ml/min/1.73 m^2)
- Allergy to betaine or related compounds
- History of papilledema or increased intracranial pressure.
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic Hyperoxaluria Center
Rochester, Minnesota, 55905
About this study
The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients.
Hypothesis:
Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
Read the detailed description
Our prior genotyping results have shown an association between the G170R allele and the clinical response to VB6. Patients homozygous for this change show a complete response and heterozygous patients a partial response. Since VB6 is a safe and completely effective treatment for patients homozygous for G170R, we will not study betaine in this group. Instead, 20 participants older than 6 years of age who are G170R compound heterozygous, non-G170R missense or truncating sequence change homozygous or heterozygous, will be selected for enrollment. Participants in whom VB6 provides a partial reduction in urine oxalate excretion (compound heterozygotes for the G170R mutation) will be maintained on a stable dose of VB6 (8 mg/kg/d) for two months before and throughout betaine treatment. Those who have demonstrated no response to VB6 will receive betaine alone.
Participants will be randomized to receive either betaine or placebo for the first 2 month arm of the study. Following 2 months of treatment and 2 months of washout, each participant will cross over to the other arm of the study. The other arm will consist of the participant being on 2 months of treatment of whatever they were not taking in the first arm (betaine vs. placebo). Neither the study staff nor the participant will know whether the participant is taking betaine for the first or second arm of the study, or the placebo for the first or second arm of the study. Only the pharmacy will know this.
Prior to the study, a complete history and physical examination, and baseline laboratory studies pertinent to the routine care of primary hyperoxaluria patients will be performed (Complete Blood Count (CBC) with differential, chemistry group, electrolytes, plasma oxalate and creatinine clearance, urinary supersaturation). All women capable of reproduction will receive a pregnancy test prior to enrollment.
Participant will complete two 24-hour urine collections for calcium oxalate super-saturation (includes 24-hour urine oxalate excretion) at baseline, inclusive of creatinine determination for assessment of completeness. They will then begin Cystadane anhydrous solution (12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses). These doses of betaine have been shown to effectively treat pediatric patients with VB6-resistant homocystinuria and reverse Nonalcoholic Steatohepatitis (NASH) in adult patients, so we expect they will achieve sufficient intra-hepatocyte levels to have an effect in PHI.
A sample of each 24-hour urine will be stored frozen (-80ºC) to allow determination of indicators of oxidant stress, should urinary oxalate fall.
If effective, betaine could represent a new and safe treatment option for a subset of PHI patients, particularly those with either partially VB6 responsive or VB6 refractory hyperoxaluria, or those with adverse effects such as peripheral neuropathy from large doses of VB6. We do not anticipate any adverse medication effects specific to primary hyperoxaluria. However, as an extra safeguard for children with PHI, ten subjects older than 15 years of age will be tested first and if the agent is well tolerated in PHI patients, pediatric subjects older than 6 years of age will then be recruited for participation.
- Conditions
- Hyperoxaluria
- Being tested
- Betaine, Placebo
- Keywords
- Primary Hyperoxaluria
- Medical subject headings
- Hyperoxaluria, Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BetaineDrug
Also called Cystadane
Subjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses, for 2 months.
PlaceboDrug
Also called Lactose
Subjects received oral lactose placebo, in two doses daily, for 2 months.
| Study group | Role | What they receive |
|---|---|---|
| Betaine | Experimental | Subjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral lactose placebo, in two doses daily, for 2 months. Receives: Drug: Betaine; Drug: Placebo |
| Placebo | Placebo comparator | Subjects were randomly assigned to receive oral lactose placebo, in two doses daily, for 2 months. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses, for 2 months. Receives: Drug: Betaine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Urinary Oxalate Excretion
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.
Measured: baseline, 2 months, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2006 (estimated)
- Study startFeb 2007
- Primary completionSep 2011
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (32)Papers the sponsor has linked to this study.
- Other trials for HyperoxaluriaStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Orphan Europe
- Investigators
- Carla G Monico, M.D., Principal investigator, Mayo Clinic Hyperoxaluria Center, Rochester MN
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00283387
- Sponsor's protocol ID
- 2147-05
- Other IDs
- 5R01DK073354-04
- Status last verified
- Nov 2013
- First submitted
- 26 Jan 2006
- Last updated
- 16 Dec 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Abdelmalek MF, Angulo P, Jorgensen RA, Sylvestre PB, Lindor KD. Betaine, a promising new agent for patients with nonalcoholic steatohepatitis: results of a pilot study. Am J Gastroenterol. 2001 Sep;96(9):2711-7. doi: 10.1111/j.1572-0241.2001.04129.x.
PubMed 11569700Adamzik M, Schmermund A, Reed JE, Adamzik S, Behrenbeck T, Sheedy PF 2nd. Comparison of two different software systems for electron-beam CT-derived quantification of coronary calcification. Invest Radiol. 1999 Dec;34(12):767-73. doi: 10.1097/00004424-199912000-00006.
PubMed 10587873ARCHER HE, DORMER AE, SCOWEN EF, WATTS RW. Primary hyperoxaluria. Lancet. 1957 Aug 17;273(6990):320-2. doi: 10.1016/s0140-6736(57)92210-9. No abstract available.
PubMed 13464052Barak AJ, Beckenhauer HC, Badakhsh S, Tuma DJ. The effect of betaine in reversing alcoholic steatosis. Alcohol Clin Exp Res. 1997 Sep;21(6):1100-2.
PubMed 9309323Bourot S, Sire O, Trautwetter A, Touze T, Wu LF, Blanco C, Bernard T. Glycine betaine-assisted protein folding in a lysA mutant of Escherichia coli. J Biol Chem. 2000 Jan 14;275(2):1050-6. doi: 10.1074/jbc.275.2.1050.
PubMed 10625645Cochat P, Deloraine A, Rotily M, Olive F, Liponski I, Deries N. Epidemiology of primary hyperoxaluria type 1. Societe de Nephrologie and the Societe de Nephrologie Pediatrique. Nephrol Dial Transplant. 1995;10 Suppl 8:3-7. doi: 10.1093/ndt/10.supp8.3.
PubMed 8592622Danpure 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.
Diamant S, Eliahu N, Rosenthal D, Goloubinoff P. Chemical chaperones regulate molecular chaperones in vitro and in cells under combined salt and heat stresses. J Biol Chem. 2001 Oct 26;276(43):39586-91. doi: 10.1074/jbc.M103081200. Epub 2001 Aug 21.
PubMed 11517217Diamant S, Rosenthal D, Azem A, Eliahu N, Ben-Zvi AP, Goloubinoff P. Dicarboxylic amino acids and glycine-betaine regulate chaperone-mediated protein-disaggregation under stress. Mol Microbiol. 2003 Jul;49(2):401-10. doi: 10.1046/j.1365-2958.2003.03553.x.
PubMed 12828638Levine DZ, Nash LA, Chan T, Dubrovskis AH. Proximal bicarbonate reabsorption during Ringer and albumin infusions in the rat. J Clin Invest. 1976 Jun;57(6):1490-7. doi: 10.1172/JCI108419.
PubMed 932191Lumb MJ, Birdsey GM, Danpure CJ. Correction of an enzyme trafficking defect in hereditary kidney stone disease in vitro. Biochem J. 2003 Aug 15;374(Pt 1):79-87. doi: 10.1042/BJ20030371.
PubMed 12737622Lumb MJ, Danpure CJ. Functional synergism between the most common polymorphism in human alanine:glyoxylate aminotransferase and four of the most common disease-causing mutations. J Biol Chem. 2000 Nov 17;275(46):36415-22. doi: 10.1074/jbc.M006693200.
PubMed 10960483Monico CG, Rossetti S, Olson JB, Milliner DS. Pyridoxine effect in type I primary hyperoxaluria is associated with the most common mutant allele. Kidney Int. 2005 May;67(5):1704-9. doi: 10.1111/j.1523-1755.2005.00267.x.
PubMed 15840016Purdue PE, Takada Y, Danpure CJ. Identification of mutations associated with peroxisome-to-mitochondrion mistargeting of alanine/glyoxylate aminotransferase in primary hyperoxaluria type 1. J Cell Biol. 1990 Dec;111(6 Pt 1):2341-51. doi: 10.1083/jcb.111.6.2341.
PubMed 1703535Rantanen I, Nicander I, Jutila K, Ollmar S, Tenovuo J, Soderling E. Betaine reduces the irritating effect of sodium lauryl sulfate on human oral mucosa in vivo. Acta Odontol Scand. 2002 Oct;60(5):306-10. doi: 10.1080/00016350260248292.
PubMed 12418722
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT00283387
- Acronym / sponsor ID
- 2147-05
- Conditions
- Hyperoxaluria
- Keywords
- Primary Hyperoxaluria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Orphan Europe
- Study start
- Feb 2007
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 27 Jan 2006 (estimated)
- Results
- Posted 1 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients.
Hypothesis:
Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
Detailed description
Our prior genotyping results have shown an association between the G170R allele and the clinical response to VB6. Patients homozygous for this change show a complete response and heterozygous patients a partial response. Since VB6 is a safe and completely effective treatment for patients homozygous for G170R, we will not study betaine in this group. Instead, 20 participants older than 6 years of age who are G170R compound heterozygous, non-G170R missense or truncating sequence change homozygous or heterozygous, will be selected for enrollment. Participants in whom VB6 provides a partial reduction in urine oxalate excretion (compound heterozygotes for the G170R mutation) will be maintained on a stable dose of VB6 (8 mg/kg/d) for two months before and throughout betaine treatment. Those who have demonstrated no response to VB6 will receive betaine alone.
Participants will be randomized to receive either betaine or placebo for the first 2 month arm of the study. Following 2 months of treatment and 2 months of washout, each participant will cross over to the other arm of the study. The other arm will consist of the participant being on 2 months of treatment of whatever they were not taking in the first arm (betaine vs. placebo). Neither the study staff nor the participant will know whether the participant is taking betaine for the first or second arm of the study, or the placebo for the first or second arm of the study. Only the pharmacy will know this.
Prior to the study, a complete history and physical examination, and baseline laboratory studies pertinent to the routine care of primary hyperoxaluria patients will be performed (Complete Blood Count (CBC) with differential, chemistry group, electrolytes, plasma oxalate and creatinine clearance, urinary supersaturation). All women capable of reproduction will receive a pregnancy test prior to enrollment.
Participant will complete two 24-hour urine collections for calcium oxalate super-saturation (includes 24-hour urine oxalate excretion) at baseline, inclusive of creatinine determination for assessment of completeness. They will then begin Cystadane anhydrous solution (12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses). These doses of betaine have been shown to effectively treat pediatric patients with VB6-resistant homocystinuria and reverse Nonalcoholic Steatohepatitis (NASH) in adult patients, so we expect they will achieve sufficient intra-hepatocyte levels to have an effect in PHI.
A sample of each 24-hour urine will be stored frozen (-80ºC) to allow determination of indicators of oxidant stress, should urinary oxalate fall.
If effective, betaine could represent a new and safe treatment option for a subset of PHI patients, particularly those with either partially VB6 responsive or VB6 refractory hyperoxaluria, or those with adverse effects such as peripheral neuropathy from large doses of VB6. We do not anticipate any adverse medication effects specific to primary hyperoxaluria. However, as an extra safeguard for children with PHI, ten subjects older than 15 years of age will be tested first and if the agent is well tolerated in PHI patients, pediatric subjects older than 6 years of age will then be recruited for participation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Betaine | Experimental | Drug: Betaine, Drug: PlaceboSubjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral lactose placebo, in two doses daily, for 2 months. |
| Placebo | Placebo comparator | Drug: Betaine, Drug: PlaceboSubjects were randomly assigned to receive oral lactose placebo, in two doses daily, for 2 months. This was followed by a 2 month washout period. Subjects then received the alternative study medication, oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses, for 2 months. |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- A definitive diagnosis of Type 1 Primary Hyperoxaluria (PHI) as confirmed by hepatic angiotensinogen (AGT) deficiency, biochemical criteria (marked hyperoxaluria and hyperglycolic aciduria) or mutation analysis (having a known PHI mutation)
- Alanine-glyoxylate aminotransferase (AGXT) genotype known
- Hyperoxaluria not fully corrected by 3 months of continuous Vitamin B6 (VB6) at doses of 8 mg/kg/d or more
- Males or females, 6-70 years of age, inclusive
- Preserved renal function, as defined by measured glomerular filtration rate (GFR) > 30 ml/min/1.73 m^2
Key exclusion criteria (8 listed)
- Patients who are fully VB6 responsive (i.e., G170R homozygotes).
- Prior recipients of liver transplantation performed for correction of AGT deficiency.
- Pregnancy or breastfeeding
- Unwillingness of patient and/or partner to use contraception during treatment.
- Malignant disease (other than non-melanoma skin cancer) in the previous two years.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BetaineAlso: CystadaneSubjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses, for 2 months. | Drug | Betaine, Placebo |
| PlaceboAlso: LactoseSubjects received oral lactose placebo, in two doses daily, for 2 months. | Drug | Betaine, Placebo |
Primary outcome
Urinary Oxalate Excretion
Time frame: baseline, 2 months, 6 months
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 11569700Abdelmalek MF, Angulo P, Jorgensen RA, Sylvestre PB, Lindor KD. Betaine, a promising new agent for patients with nonalcoholic steatohepatitis: results of a pilot study. Am J Gastroenterol. 2001 Sep;96(9):2711-7. doi: 10.1111/j.1572-0241.2001.04129.x.
- Background: PubMed 10587873Adamzik M, Schmermund A, Reed JE, Adamzik S, Behrenbeck T, Sheedy PF 2nd. Comparison of two different software systems for electron-beam CT-derived quantification of coronary calcification. Invest Radiol. 1999 Dec;34(12):767-73. doi: 10.1097/00004424-199912000-00006.
- Background: PubMed 13464052ARCHER HE, DORMER AE, SCOWEN EF, WATTS RW. Primary hyperoxaluria. Lancet. 1957 Aug 17;273(6990):320-2. doi: 10.1016/s0140-6736(57)92210-9. No abstract available.
- Background: PubMed 9309323Barak AJ, Beckenhauer HC, Badakhsh S, Tuma DJ. The effect of betaine in reversing alcoholic steatosis. Alcohol Clin Exp Res. 1997 Sep;21(6):1100-2.
- Background: PubMed 10625645Bourot S, Sire O, Trautwetter A, Touze T, Wu LF, Blanco C, Bernard T. Glycine betaine-assisted protein folding in a lysA mutant of Escherichia coli. J Biol Chem. 2000 Jan 14;275(2):1050-6. doi: 10.1074/jbc.275.2.1050.
- Background: PubMed 8592622Cochat P, Deloraine A, Rotily M, Olive F, Liponski I, Deries N. Epidemiology of primary hyperoxaluria type 1. Societe de Nephrologie and the Societe de Nephrologie Pediatrique. Nephrol Dial Transplant. 1995;10 Suppl 8:3-7. doi: 10.1093/ndt/10.supp8.3.
- BackgroundDanpure 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.
- Background: PubMed 11517217Diamant S, Eliahu N, Rosenthal D, Goloubinoff P. Chemical chaperones regulate molecular chaperones in vitro and in cells under combined salt and heat stresses. J Biol Chem. 2001 Oct 26;276(43):39586-91. doi: 10.1074/jbc.M103081200. Epub 2001 Aug 21.
- Background: PubMed 12828638Diamant S, Rosenthal D, Azem A, Eliahu N, Ben-Zvi AP, Goloubinoff P. Dicarboxylic amino acids and glycine-betaine regulate chaperone-mediated protein-disaggregation under stress. Mol Microbiol. 2003 Jul;49(2):401-10. doi: 10.1046/j.1365-2958.2003.03553.x.
- Background: PubMed 932191Levine DZ, Nash LA, Chan T, Dubrovskis AH. Proximal bicarbonate reabsorption during Ringer and albumin infusions in the rat. J Clin Invest. 1976 Jun;57(6):1490-7. doi: 10.1172/JCI108419.
- Background: PubMed 12737622Lumb MJ, Birdsey GM, Danpure CJ. Correction of an enzyme trafficking defect in hereditary kidney stone disease in vitro. Biochem J. 2003 Aug 15;374(Pt 1):79-87. doi: 10.1042/BJ20030371.
- Background: PubMed 10960483Lumb MJ, Danpure CJ. Functional synergism between the most common polymorphism in human alanine:glyoxylate aminotransferase and four of the most common disease-causing mutations. J Biol Chem. 2000 Nov 17;275(46):36415-22. doi: 10.1074/jbc.M006693200.
- Oxalosis and Hyperoxaluria FoundationLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carla G Monico, M.D. principal investigator
Mayo Clinic Hyperoxaluria Center, Rochester MN
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Betaine in people with Hyperoxaluria, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Jan 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Urinary Oxalate Excretion (time frame: baseline, 2 months, 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00283387, which also lists 32 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2013.