Primary HyperoxaluriaPhase 2/3PHOENIXNCT00638703
Study to Evaluate the Efficacy and Safety of OxabactTM on Reduction of Urinary Oxalate in Primary Hyperoxaluria Patients
A Phase 2/3 study testing Oxalobacter formigenes in people with Primary Hyperoxaluria, run by OxThera at 1 site in United States.
Trial summary
The main 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 5 years and older
- Conditions the study is for
- Primary Hyperoxaluria
- What is being tested?
- Oxalobacter formigenes
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2007to Oct 2008
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The subject (or legally acceptable representative) must give written informed consent (and assent for subjects ≥ 12 years or country specific age as appropriate). For subjects less than 18 years of age, parent or guardian will provide informed consent and the subject will provide witnessed verbal assent
- Male or female subjects ≥ 5 years of age
- Urinary oxalate excretion of > 1.0 mmol/1.73m2/day at Baseline
- Documentation of diagnosis of PH I or PH II by any 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 (GR/HPR) 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 in to the study and must remain on the stable dose during the study. Other (non-pyridoxine naïve) subjects (e.g. Pyridoxine non-responder: <30% reduction of the urine oxalate levels) not receiving pyridoxine at study entry must be willing to refrain from initiating pyridoxine during study participation. Note: There will be no pyridoxine-naïve subjects enrolled in the study.
- Renal function defined as an estimated GFR ≥ 50 ml/min normalized to 1.73m2 body surface area
Who cannot join
- Pregnant, lactating, or actively menstruating women and 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.
- Note: A highly effective method of birth control is defined as one that results in a low failure rate (i.e. less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine contraceptive devices (IUDs), sexual abstinence, or a vasectomised partner.
- Positive serum pregnancy test.
- 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.
- Subjects on hemodialysis or peritoneal dialysis.
- Subjects who have undergone transplantation (solid organ or bone marrow).
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- Chronic gastrointestinal disease associated with enteric hyperoxaluria, e.g. history of inflammatory bowel disease, colostomy. Note: For clarity, existence of Secondary Hyperoxaluria (e.g. with cystic fibrosis, chronic inflammatory bowel diseases, short bowel syndrome and/or deficiency of intestinal oxalate-degrading bacteria is included (as an exclusion criteria).
- Current systemic (oral, IM, IV) antibiotic use or received systemic antibiotics within 14 days of study enrolment.
- History of chronic, recurrent infections requiring >2 courses of antibiotics in the past 6 months.
- History of malignancy except for basal or squamous cell skin cancer that has been excised.
- Unable to collect 24-hour urine samples or follow other study procedures.
- Subjects who cannot swallow a size 2 capsule.
- 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.
- Subjects who require immune suppressive therapy (including prednisone of > 10mg daily for more than 2 weeks).
- Subjects from correctional facilities or asylums.
- Subjects who are mentally handicapped.
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic (Department of Pediatric Nephrology)
Rochester, Minnesota, 55905
About this study
Official title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multi-center, International Study to Evaluate the Efficacy and Safety of OxabactTM to Reduce Urinary Oxalate in Subjects With Primary Hyperoxaluria
The main 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, PH, oxalate
- 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(tm), - OC3
NLT (not less than) 10^7 CFU Oxalobacter formigenes twice daily for 24 weeks
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| I | Experimental | Size 2 enteric coated capsule containg lyophilized Oxalobacter formigenes Receives: Biological: Oxalobacter formigenes |
| II | Placebo comparator | Size 2 enteric coated capsule containg placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in urinary oxalate Percentage change in urinary oxalate (expressed as mmole/1.73m2 /day) from Baseline to Week 24
Measured: 24 weeks
Secondary outcomes (6)
Percentage of subjects who are responders at Week 24 where response is defined as a 20% or greater reduction from Baseline urinary oxalate to Week 24
Measured: 24 weeks
Percentage change in urinary oxalate (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24
Measured: 24 weeks
Percentage change in urinary oxalate (expressed as mmole/1.73m2/day and as molar oxalate to creatinine ratio) from Baseline to Week 12
Measured: 12 weeks
Percentage change in urinary oxalate (expressed as mmole/1.73m2/day and as molar oxalate to creatinine ratio) from Baseline to average of Weeks 12 and 24
Measured: 12 and 24 weeks
Frequency of AEs and SAEs
Measured: over 24 weeks
Laborator safety data
Measured: 12 and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2008 (estimated)
- Study startOct 2007
- Primary completionSep 2008
- Study completionOct 2008
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
- 43 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
- NCT00638703
- Sponsor's protocol ID
- OC3-DB-01
- Other IDs
- DB-01
- Acronym
- PHOENIX
- Status last verified
- Jun 2011
- First submitted
- 12 Mar 2008
- Last updated
- 15 May 2013
- Results posted
- No
- Data monitoring committee
- No
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, International Study to Evaluate the Efficacy and Safety of OxabactTM to Reduce Urinary Oxalate in Subjects With Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT00638703
- Acronym / sponsor ID
- PHOENIX / OC3-DB-01
- Conditions
- Primary Hyperoxaluria
- Keywords
- hyperoxaluria, PH, oxalate
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- OxTheraIndustry (pharma, biotech, devices)
- Study start
- Oct 2007
- Primary completion
- Sep 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 19 Mar 2008 (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 main 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 |
|---|---|---|
| I | Experimental | Biological: Oxalobacter formigenesSize 2 enteric coated capsule containg lyophilized Oxalobacter formigenes |
| II | Placebo comparator | Drug: PlaceboSize 2 enteric coated capsule containg placebo |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- The subject (or legally acceptable representative) must give written informed consent (and assent for subjects ≥ 12 years or country specific age as appropriate). For subjects less than 18 years of age, parent or guardian will provide informed consent and the subject will provide witnessed verbal assent
- Male or female subjects ≥ 5 years of age
- Urinary oxalate excretion of > 1.0 mmol/1.73m2/day at Baseline
- Documentation of diagnosis of PH I or PH II by any one of the following:
- Subjects receiving pyridoxine must be receiving a stable dose for at least 3 months prior to entry in to the study and must remain on the stable dose during the study. Other (non-pyridoxine naïve) subjects (e.g. Pyridoxine non-responder: <30% reduction of the urine oxalate levels) not receiving pyridoxine at study entry must be willing to refrain from initiating pyridoxine during study participation. Note: There will be no pyridoxine-naïve subjects enrolled in the study.
Key exclusion criteria (16 listed)
- Pregnant, lactating, or actively menstruating women and 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.
- Note: A highly effective method of birth control is defined as one that results in a low failure rate (i.e. less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine contraceptive devices (IUDs), sexual abstinence, or a vasectomised partner.
- Positive serum pregnancy test.
- 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.
- Subjects on hemodialysis or peritoneal dialysis.
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(tm), - OC3NLT (not less than) 10^7 CFU Oxalobacter formigenes twice daily for 24 weeks | Biological | I |
| Placeboplacebo | Drug | II |
Primary outcome
Reduction in urinary oxalate Percentage change in urinary oxalate (expressed as mmole/1.73m2 /day) from Baseline to Week 24
Time frame: 24 weeks
6 secondary outcomes
Percentage of subjects who are responders at Week 24 where response is defined as a 20% or greater reduction from Baseline urinary oxalate to Week 24
Time frame: 24 weeks
Percentage change in urinary oxalate (expressed as molar oxalate to creatinine ratio) from Baseline to Week 24
Time frame: 24 weeks
Percentage change in urinary oxalate (expressed as mmole/1.73m2/day and as molar oxalate to creatinine ratio) from Baseline to Week 12
Time frame: 12 weeks
Percentage change in urinary oxalate (expressed as mmole/1.73m2/day and as molar oxalate to creatinine ratio) from Baseline to average of Weeks 12 and 24
Time frame: 12 and 24 weeks
Frequency of AEs and SAEs
Time frame: over 24 weeks
Laborator safety data
Time frame: 12 and 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
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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
1 site in 1 country: 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 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 9 inclusion and 16 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 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 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?
Reduction in urinary oxalate Percentage change in urinary oxalate (expressed as mmole/1.73m2 /day) 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.