Primary HyperoxaluriaPhase 1/2NCT02012985
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria
A Phase 1/2 study testing Oxabact OC5 capsules in people with Primary Hyperoxaluria, run by OxThera at 8 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?
- Oxabact OC5 capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 8 sitesin 3 countries
- Study dates
- Dec 2013to Jan 2015
- Phase
- Phase 1/2
- 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 (Germany \& France) / Male or female subjects ≥ 5 years of age (United Kingdom)
- A diagnosis of PH type I, II or III (as determined by standard diagnostic methods).
- A mean urinary oxalate excretion of > 1.0 mmol/24h/1.73m2, based on at least three eligible urine collections performed during baseline (weeks 1-4).
- Renal function defined as an estimated GFR ≥ 40 ml/min normalised to 1.73m2 body surface area, or a creatinine clearance of ≥ 40 ml/min normalised to 1.73m2 body surface area.
- Subjects receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must not change the dose during the study. Subjects not receiving vitamin B6 at study entry must be willing to refrain from initiating pyridoxine during study participation.
Who cannot join
- Inability to collect complete 24-hour urine samples. Each urine collection will be evaluated for completeness based on urine qualitative criteria.
- Inability to swallow size 4 capsules twice daily for 8 to 10 weeks.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- The existence of secondary hyperoxaluria, e.g. hyperoxaluria due to bariatric surgery or chronic gastrointestinal diseases such as cystic fibrosis, chronic inflammatory bowel disease and short-bowel syndrome.
- Use of antibiotics to which O. formigenes is sensitive, including chronic use, a history of more than two courses of antibiotic use during the past 6 months, current antibiotic use, or antibiotics use within 14 days of initiating study medication.
- Subjects who require immune suppressive therapy.
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- Current treatment with ascorbic acid preparation.
- Pregnancy.
- Women of child-bearing potential who are not using adequate contraceptive precautions such as oral, transdermal, injectable, or implanted contraceptives, IUD, complete abstinence, use of a condom by the sexual partner, or sterile sexual partner.
- 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.
- Participation in any study of an investigational product, biologic, device, or other agent within 30 days prior to screening or not willing to forego other forms of investigational treatment during this study.
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
France4 sites
Hôpital des Enfants, Centre de référence maladies rénales rares du Sud-Ouest (SORARE), CHU de Bordeaux
Bordeaux, 33076
Hôpital Femme Mère Enfant, Lyon - Paediatric Dept
Lyon, 69677 Bron
Hôpital Robert-Debré, Néphrologie Pédiatrique
Paris, Cedex 19, 75945
Hôpital Necker-Enfants Malades,Centre de référence des Maladies Rénales Héréditaires de l'Enfant et de l'Adulte (MARHEA)
Paris, 75015
United Kingdom3 sites
Birmingham Children's Hospital NHS Foundation Trust - Dept of Nephrology
Birmingham, B4 6NH
Royal Free Hospital -UCL Centre for Nephrology
London, NW3 2QG
Great Ormond Street Hospital for Children NHS Trust
London, WCIN 3JH
Germany1 site
Universitätsklinikum Bonn, Dept of Paediatric Nephrology
Bonn, DE-53113
About this study
Official title: A Phase 1/2, Randomised, Placebo-controlled, Double-blind, Multi-centre Study to Evaluate the Efficacy and Safety of OC5 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
- Oxabact OC5 capsules, Placebo capsules
- 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.
Oxabact OC5 capsulesBiological
Also called Oxalobacter formigenes
The dose will be not less than (NLT) 1E+09 colony forming units (CFU) twice daily for 8 to 10 weeks. The dose (an enteric-coated size 4 capsule) will be administered orally with breakfast and dinner.
Placebo capsulesDrug
An enteric-coated placebo capsule manufactured to mimic the OC5 capsule. The capsule will be administered orally with breakfast and dinner twice daily for 8 to 10 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Oxabact OC5 capsules | Experimental | The active study drug consists of Oxalobacter formigenes OC5 in enteric-coated size-4 capsules. The dose (not less than (NLT) 1E+09 colony forming units (CFU)) will be administrated orally with breakfast and dinner as one capsule two times per day for 8 to 10 weeks. Receives: Biological: Oxabact OC5 capsules |
| Placebo capsules | Placebo comparator | The placebo study drug consists of microcrystalline cellulose in enteric-coated size-4 capsules. It has been manufactured to mimic the OC5 capsule. The dose will be administrated orally with breakfast and dinner as one capsule two times per day for 8 to 10 weeks. Receives: Drug: Placebo capsules |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in urinary oxalate levels from Baseline to week 8 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Secondary outcomes (10)
Change in urinary oxalate levels from Baseline to week 8 of treatment in subsets of subjects
Change in urinary oxalate levels from Baseline to week 8 of treatment in subsets of subjects defined by:
- baseline urinary oxalate level, above and below 1.5 mmol/24h/1.73m2
- concomitant vitamin B6 therapy and no vitamin B6 therapy
- eGFR of ≥90 mL/min/1.73m2 (normal renal function) and < 90 mL/min/1.73m2 (mild to moderate reduction in renal function)
- age below 18 and age 18 or above
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Number of subjects who reach urinary oxalate levels below 0.5, 0.7 and 1.0 mmol/24h/1.73m2 respectively from Baseline to week 8 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in plasma oxalate levels from Baseline to week 8 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in urinary oxalate levels from Baseline to week 4 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 10 of the study)
Correlation between change in plasma oxalate levels and change in urinary oxalate levels, from Baseline to week 8 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in number of O. formigenes in faeces from Baseline to week 8 of treatment.
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Adverse events
Measured: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Haematology
Blood samples taken for hematology at weeks 0, 5, 10 and 14. Complete blood count with differential and platelet count evaluated.
Measured: 14 weeks (Throughout the study)
Clinical Chemistry
Blood samples taken for clinical chemistry at weeks 0, 5, 10 and 14. Blood Urea Nitrogen, creatinine, electrolytes (Na+, K+, Mg++, Ca++, HCO3+, Cl), glucose, pH, albumin, alkaline phosphatase, ALT, AST, total bilirubin and total protein evaluated.
Measured: 14 weeks (Throughout the study)
Urinalysis
Urine samples will be taken at weeks 0, 5, 10 and 14 of the study. Protein, glucose and pH evaluated.
Measured: 14 weeks (Throughout the study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2013 (estimated)
- Study startDec 2013
- Primary completionJan 2015
- Study completionJan 2015
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- OxThera
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FP7-SME-2013 Research for the benefit of SMEs program
- Responsible party
- Sponsor
- Investigators
- Bernd Hoppe, MD PhD, Principal investigator, Universitätsklinikum Bonn, Dept of Paediatric Nephrology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02012985
- Sponsor's protocol ID
- OC5-DB-01
- Status last verified
- Oct 2015
- First submitted
- 11 Dec 2013
- Last updated
- 16 Oct 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Randomised, Placebo-controlled, Double-blind, Multi-centre Study to Evaluate the Efficacy and Safety of OC5 to Reduce Urinary Oxalate in Subjects With Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT02012985
- Acronym / sponsor ID
- OC5-DB-01
- Conditions
- Primary Hyperoxaluria
- Keywords
- hyperoxaluria, oxalate, PH
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- OxTheraIndustry (pharma, biotech, devices)
- Collaborators
- FP7-SME-2013 Research for the benefit of SMEs program
- Study start
- Dec 2013
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 17 Dec 2013 (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 OC5 capsules | Experimental | Biological: Oxabact OC5 capsulesThe active study drug consists of Oxalobacter formigenes OC5 in enteric-coated size-4 capsules. The dose (not less than (NLT) 1E+09 colony forming units (CFU)) will be administrated orally with breakfast and dinner as one capsule two times per day for 8 to 10 weeks. |
| Placebo capsules | Placebo comparator | Drug: Placebo capsulesThe placebo study drug consists of microcrystalline cellulose in enteric-coated size-4 capsules. It has been manufactured to mimic the OC5 capsule. The dose will be administrated orally with breakfast and dinner as one capsule two times per day for 8 to 10 weeks. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Signed informed consent (as applicable for the age of the subject).
- Male or female subjects ≥ 2 years of age (Germany \& France) / Male or female subjects ≥ 5 years of age (United Kingdom)
- A diagnosis of PH type I, II or III (as determined by standard diagnostic methods).
- A mean urinary oxalate excretion of > 1.0 mmol/24h/1.73m2, based on at least three eligible urine collections performed during baseline (weeks 1-4).
- Renal function defined as an estimated GFR ≥ 40 ml/min normalised to 1.73m2 body surface area, or a creatinine clearance of ≥ 40 ml/min normalised to 1.73m2 body surface area.
Key exclusion criteria (11 listed)
- Inability to collect complete 24-hour urine samples. Each urine collection will be evaluated for completeness based on urine qualitative criteria.
- Inability to swallow size 4 capsules twice daily for 8 to 10 weeks.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- The existence of secondary hyperoxaluria, e.g. hyperoxaluria due to bariatric surgery or chronic gastrointestinal diseases such as cystic fibrosis, chronic inflammatory bowel disease and short-bowel syndrome.
- Use of antibiotics to which O. formigenes is sensitive, including chronic use, a history of more than two courses of antibiotic use during the past 6 months, current antibiotic use, or antibiotics use within 14 days of initiating study medication.
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 |
|---|---|---|
| Oxabact OC5 capsulesAlso: Oxalobacter formigenesThe dose will be not less than (NLT) 1E+09 colony forming units (CFU) twice daily for 8 to 10 weeks. The dose (an enteric-coated size 4 capsule) will be administered orally with breakfast and dinner. | Biological | Oxabact OC5 capsules |
| Placebo capsulesAn enteric-coated placebo capsule manufactured to mimic the OC5 capsule. The capsule will be administered orally with breakfast and dinner twice daily for 8 to 10 weeks. | Drug | Placebo capsules |
Primary outcome
Change in urinary oxalate levels from Baseline to week 8 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
10 secondary outcomes
Change in urinary oxalate levels from Baseline to week 8 of treatment in subsets of subjects
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Number of subjects who reach urinary oxalate levels below 0.5, 0.7 and 1.0 mmol/24h/1.73m2 respectively from Baseline to week 8 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in plasma oxalate levels from Baseline to week 8 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in urinary oxalate levels from Baseline to week 4 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 10 of the study)
Correlation between change in plasma oxalate levels and change in urinary oxalate levels, from Baseline to week 8 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Change in number of O. formigenes in faeces from Baseline to week 8 of treatment.
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Adverse events
Time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)
Haematology
Time frame: 14 weeks (Throughout the study)
Clinical Chemistry
Time frame: 14 weeks (Throughout the study)
Urinalysis
Time frame: 14 weeks (Throughout the study)
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 16 Oct 2015.
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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.
Bernd Hoppe, MD PhD principal investigator
Universitätsklinikum Bonn, Dept of Paediatric Nephrology
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Oxabact OC5 capsules in people with Primary Hyperoxaluria, run by OxThera at 8 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 6 inclusion and 11 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 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in France, United Kingdom, Germany. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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?
Change in urinary oxalate levels from Baseline to week 8 of treatment. (time frame: 8 weeks of active treatment (i.e. between Weeks 7 and 14 of the study)). 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 16 Oct 2015.