Primary HyperoxaluriaPhase 2/3NCT01037231
Phase 2/3 Oxabact Study
A Phase 2/3 study testing Oxalobacter formigenes in people with Primary Hyperoxaluria, run by OxThera at 3 sites in 3 countries.
Trial summary
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Primary Hyperoxaluria
- What is being tested?
- Oxalobacter formigenes
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 3 sitesin 3 countries
- Study dates
- Dec 2009to Jan 2011
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent (as applicable for the age of the subject)
- Male or female subjects ≥ 2 years of age
- A mean urinary oxalate excretion of > 1.0 mmol/1.73m2/day from eligible urine collections performed during screening.
- A diagnosis of PH I or PH II by one of the following:
- Liver biopsy confirmation of deficient liver specific peroxisomal alanine-glyoxylate aminotransferase, (AGT) or mislocalization of AGT from peroxisomes to mitochondria (PH I) or deficient glyoxylate reductase/hydroxypyruvate reductase (GRHPR) activity (PH II)
- Homozygosity or compound heterozygosity for a known mutation in the causative genes for PH I and PH II.
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- Increased glycolate excretion for PH I or increased L-glycerate excretion for PH II.
- Subjects receiving pyridoxine must be receiving a stable dose for at least 3 months prior to entry into the study and must remain on the stable dose during the study. Subjects not receiving pyridoxine at study entry must be willing to refrain from initiating pyridoxine during study participation.
- Renal function defined as an estimated GFR ≥ 40 ml/min normalized to 1.73m2 body surface area, or a creatinine clearance of ≥ 40 ml/min normalized to 1.73m2 body surface area.
Who cannot join
- Inability to collect two complete 24-hour urine samples. Each urine collection will be evaluated for completeness based on the urine acceptance criteria outlined in section 11.1.
- Subjects diagnosed as PH I who are pyridoxine naïve.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- The existence of secondary hyperoxaluria, e.g. chronic gastrointestinal diseases such as cystic fibrosis, chronic inflammatory bowel disease and short-bowel syndrome.
- Current systemic (oral, IM, IV) antibiotic use or received systemic antibiotics within 14 days of study enrolment.
- History of a recurrent infection requiring >2 courses of systemic antibiotics in the past 6 months, or chronic antimicrobial suppression.
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- Subjects who require immune suppressive therapy (including prednisone > 10mg daily for more than 2 weeks).
- Current treatment with a separate ascorbic acid preparation. Ascorbic acid up to 250mg/day as a component of a multivitamin formulation is not excluded.
- Known hypersensitivity to esomeprazol (or any of the other ingredients of this medicine), or to any other proton pump inhibitor medicine. (Nexium contraindication)
- Concomitant treatment with atazanavir. (Nexium contraindication)
- Pregnancy.
- Women of child-bearing potential who are not using adequate contraceptive precautions. Sexually active females, unless surgically sterile or at least 2 years post-menopausal, must be using a highly effective contraception (including oral, transdermal, injectable, or implanted contraceptives, IUD, abstinence, use of a condom by the sexual partner or sterile sexual partner) for 30 days prior to the first dose of OxabactTM and must agree to continue using such precautions during the clinical study.
- Presence of a medical condition that the Principal Investigator considers likely to make the subject susceptible to adverse effect of study treatment or unable to follow study procedures. Note: Subjects from correctional facilities or asylums and subjects who are mentally handicapped are not to be included in the study.
- Participation in any study of an investigational product, biologic, device, or other agent within 30 days prior to randomization or not willing to forego other forms of investigational treatment during this study.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Mayo Clinic (Department of Pediatric Nephrology)
Rochester, Minnesota, 55905
Germany1 site
University Children's Hospital (Division of Pediatric Nephrology)
Cologne
Netherlands1 site
Academy Medical Center, University of Amsterdam
Amsterdam
About this study
Official title: A Phase 2/3, Double-blind, Randomized, Placebo-controlled, Multi-center Study to Evaluate the Efficacy and Safety of OxabactTM to Reduce Urinary Oxalate in Subjects With Primary Hyperoxaluria.
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Conditions
- Primary Hyperoxaluria
- Being tested
- Oxalobacter formigenes, Placebo
- Keywords
- hyperoxaluria, oxalate, PH
- Medical subject headings
- Hyperoxaluria, Primary, Hyperoxaluria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oxalobacter formigenesBiological
Also called Oxabact, OC3
NLT (not less than) 10^7 CFU oxalobacter formigenes twice daily for 24 weeks
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| Oxabact (tm) | Experimental | Not described Receives: Biological: Oxalobacter formigenes |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24
Measured: 24 weeks
Secondary outcomes (11)
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 8
Measured: 8 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by baseline urinary oxalate level, above and below median at screening
Measured: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by concomitant vitamin B6 therapy and no vitamin B6 therapy, in PH type I
Measured: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by eGFR of ≥90 mL/min/1.73m2 and < 90 mL/min/1.73m2
Measured: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by PH Type I and PH Type II
Measured: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by age below 18 and age 18 or above
Measured: 24 weeks
Percentage of subjects who have ≥20% reduction from Baseline urinary oxalate at Week 24
Measured: 24 weeks
Percentage change in plasma oxalate levels
Measured: 24 weeks
Frequency of Stone Events (i.e. nephrolithiasis or markers thereof)
Measured: 24 weeks
Correlation between percentage change in plasma oxalate levels and percentage change in urinary oxalate levels, from Baseline to Week 24
Measured: 24 weeks
Adverse events (AEs), hematology, clinical chemistry, urinalysis.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2009 (estimated)
- Study startDec 2009
- Primary completionJan 2011
- Study completionJan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary HyperoxaluriaStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- OxThera
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dawn Milliner, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01037231
- Sponsor's protocol ID
- OC3-DB-02
- Status last verified
- May 2012
- First submitted
- 18 Dec 2009
- Last updated
- 15 May 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3, Double-blind, Randomized, Placebo-controlled, Multi-center Study to Evaluate the Efficacy and Safety of OxabactTM to Reduce Urinary Oxalate in Subjects With Primary Hyperoxaluria.
- ClinicalTrials.gov ID
- NCT01037231
- Acronym / sponsor ID
- OC3-DB-02
- Conditions
- Primary Hyperoxaluria
- Keywords
- hyperoxaluria, oxalate, PH
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- OxTheraIndustry (pharma, biotech, devices)
- Study start
- Dec 2009
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 22 Dec 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Oxabact (tm) | Experimental | Biological: Oxalobacter formigenes |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Signed informed consent (as applicable for the age of the subject)
- Male or female subjects ≥ 2 years of age
- A mean urinary oxalate excretion of > 1.0 mmol/1.73m2/day from eligible urine collections performed during screening.
- A diagnosis of PH I or PH II by one of the following:
- Subjects receiving pyridoxine must be receiving a stable dose for at least 3 months prior to entry into the study and must remain on the stable dose during the study. Subjects not receiving pyridoxine at study entry must be willing to refrain from initiating pyridoxine during study participation.
Key exclusion criteria (14 listed)
- Inability to collect two complete 24-hour urine samples. Each urine collection will be evaluated for completeness based on the urine acceptance criteria outlined in section 11.1.
- Subjects diagnosed as PH I who are pyridoxine naïve.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- The existence of secondary hyperoxaluria, e.g. chronic gastrointestinal diseases such as cystic fibrosis, chronic inflammatory bowel disease and short-bowel syndrome.
- Current systemic (oral, IM, IV) antibiotic use or received systemic antibiotics within 14 days of study enrolment.
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 |
|---|---|---|
| Oxalobacter formigenesAlso: Oxabact, OC3NLT (not less than) 10^7 CFU oxalobacter formigenes twice daily for 24 weeks | Biological | Oxabact (tm) |
| Placeboplacebo | Drug | Placebo |
Primary outcome
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24
Time frame: 24 weeks
11 secondary outcomes
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 8
Time frame: 8 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by baseline urinary oxalate level, above and below median at screening
Time frame: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by concomitant vitamin B6 therapy and no vitamin B6 therapy, in PH type I
Time frame: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by eGFR of ≥90 mL/min/1.73m2 and < 90 mL/min/1.73m2
Time frame: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by PH Type I and PH Type II
Time frame: 24 weeks
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 in subsets of subjects defined by age below 18 and age 18 or above
Time frame: 24 weeks
Percentage of subjects who have ≥20% reduction from Baseline urinary oxalate at Week 24
Time frame: 24 weeks
Percentage change in plasma oxalate levels
Time frame: 24 weeks
Frequency of Stone Events (i.e. nephrolithiasis or markers thereof)
Time frame: 24 weeks
Correlation between percentage change in plasma oxalate levels and percentage change in urinary oxalate levels, from Baseline to Week 24
Time frame: 24 weeks
Adverse events (AEs), hematology, clinical chemistry, urinalysis.
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2013.
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Enrolling by invitationObservational study
Follow-up of Pediatric Patients Affected by Primary Hyperoxaluria Type 1 (PH1) Treated With Lumasiran
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Active, not recruitingPhase 3
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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.
Dawn Milliner, M.D. principal investigator
Mayo Clinic
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Oxalobacter formigenes in people with Primary Hyperoxaluria, run by OxThera at 3 sites in 3 countries.
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 9 inclusion and 14 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 15 May 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Germany, Netherlands. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Percentage change in urinary oxalate levels (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24 (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 May 2013.