Primary HyperoxaluriaPhase 3NCT03116685
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
A Phase 3 study testing Oxabact OC5 - Oxalobacter formigenes HC-1 in people with Primary Hyperoxaluria, run by OxThera at 10 sites in 7 countries.
Trial summary
This study will evaluate the efficacy and safety of OC5 in patients with PH. 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 - Oxalobacter formigenes HC-1
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 10 sitesin 7 countries
- Study dates
- 9 Jan 2018to 15 Apr 2021
- Phase
- Phase 3
- 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 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)
- A diagnosis of PH (as determined by standard diagnostic methods).
- eGFR < 90 ml/min/1.73 m2. The Schwartz formula will be used to estimate GFR for children (age below 18), and CKD-EPI formula will be used for adults (age 18 or above).
- Plasma oxalate concentration ≥10 μmol/L in total plasma oxalate.
- Male or female patients ≥ 2 years of age.
- Patients 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. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating pyridoxine during study participation.
Who cannot join
- Inability to swallow size 4 capsules.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- Patients requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period.
- 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. (This includes current antibiotic use, or antibiotics use within 14 days of initiating study medication).
- Current treatment with a separate ascorbic acid preparation.
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- Pregnant women (or women who are planning to become pregnant) or lactating women.
- Women of childbearing potential who are not using adequate contraceptive precautions. Please see section 7.3 regarding requirements for contraception.
- Presence of a medical condition that the Investigator considers likely to make the subject susceptible to adverse effect of study treatment or unable to follow study procedures or any condition that is likely to interfere with the study drug mechanism of action (such as abnormal GI function).
- Participation in any interventional study of another investigational product, biologic, device, or other agent within 60 days prior to the first dose of OC5 or not willing to forego other forms of investigational treatment during this study.
Trial locations and contacts
10 sites in 7 countries.
10 sites in 7 countries
Tunisia3 sites
Hédi Chaker University Hospital
Sfax, 3000
Sahloul University Hospital
Sousse, 4054
Charles Nicolle University Hospital
Tunis, 1008
United Kingdom2 sites
Royal Free Hospital
London, NW3 2QG
Nottingham Children's Hospital
Nottingham, NG7 2UH
United States1 site
Vanderbilt University Hospital
Nashville, Tennessee, 37232
About this study
Official title: A Phase III Double-blind, Randomised Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
This study will evaluate the efficacy and safety of OC5 in patients with PH.
Read the detailed description
To evaluate the efficacy of Oxabact following 52 weeks treatment in subjects with maintained kidney function, but below the lower limit of the normal range (estimated glomerular filtration rate [eGFR] < 90 ml/min/1.73 m2) and a total plasma oxalate (Pox) concentration ≥ 10 μmol/L. Parameters to be evaluated include the ability to stabilise/reduce Pox concentration, to stabilise/improve kidney function and to reduce oxalate deposits in primary hyperoxaluria (PH) subjects.
- Conditions
- Primary Hyperoxaluria
- Being tested
- Oxabact OC5 - Oxalobacter formigenes HC-1, Placebo
- Medical subject headings
- Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oxabact OC5 - Oxalobacter formigenes HC-1Biological
Active study drug
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Oxabact OC5 capsules | Experimental | Oxabact OC5 - Oxalobacter formigenes HC-1 Receives: Biological: Oxabact OC5 - Oxalobacter formigenes HC-1 |
| Placebo capsules | Placebo comparator | Placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter
Measured: 52 weeks
Secondary outcomes (2)
Change From Baseline in Kidney Function
Evaluation based on eGFR calculation using the 2009 creatinine-based "Schwartz bedside" equation (for children below 18 years of age) (Schwartz et al., 2009) and 2009 creatinine-based CKD-EPI equation for adults (Levey et al., 2009). Subjects who turn 18 during the study period were continuously evaluated using the Schwartz equation, ie the equation used at baseline was kept throughout the study.
Measured: 52 weeks
Frequency of Kidney Stone Events
Number of kidney stone events for each patient
Measured: Through week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2017
- Study start9 Jan 2018
- Primary completion15 Apr 2021
- Study completion15 Apr 2021
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.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- OxThera
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gesa Schalk, MD, Principal investigator, KindernierenZentrum, Bonn, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03116685
- Sponsor's protocol ID
- OC5-DB-02
- Status last verified
- Oct 2021
- First submitted
- 7 Apr 2017
- Last updated
- 10 Dec 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Double-blind, Randomised Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT03116685
- Acronym / sponsor ID
- OC5-DB-02
- Conditions
- Primary Hyperoxaluria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- OxTheraIndustry (pharma, biotech, devices)
- Study start
- 9 Jan 2018 (actual)
- Primary completion
- 15 Apr 2021 (actual)
- Study completion
- 15 Apr 2021 (actual)
- First posted
- 17 Apr 2017 (actual)
- Results
- Posted 9 Dec 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the efficacy and safety of OC5 in patients with PH.
Detailed description
To evaluate the efficacy of Oxabact following 52 weeks treatment in subjects with maintained kidney function, but below the lower limit of the normal range (estimated glomerular filtration rate [eGFR] < 90 ml/min/1.73 m2) and a total plasma oxalate (Pox) concentration ≥ 10 μmol/L. Parameters to be evaluated include the ability to stabilise/reduce Pox concentration, to stabilise/improve kidney function and to reduce oxalate deposits in primary hyperoxaluria (PH) subjects.
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 - Oxalobacter formigenes HC-1Oxabact OC5 - Oxalobacter formigenes HC-1 |
| Placebo capsules | Placebo comparator | Other: PlaceboPlacebo |
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)
- A diagnosis of PH (as determined by standard diagnostic methods).
- eGFR < 90 ml/min/1.73 m2. The Schwartz formula will be used to estimate GFR for children (age below 18), and CKD-EPI formula will be used for adults (age 18 or above).
- Plasma oxalate concentration ≥10 μmol/L in total plasma oxalate.
- Male or female patients ≥ 2 years of age.
Key exclusion criteria (10 listed)
- Inability to swallow size 4 capsules.
- Subjects that have undergone transplantation (solid organ or bone marrow).
- Patients requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period.
- 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. (This includes 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 - Oxalobacter formigenes HC-1Active study drug | Biological | Oxabact OC5 capsules |
| PlaceboPlacebo | Other | Placebo capsules |
Primary outcome
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Time frame: 52 weeks
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter
2 secondary outcomes
Change From Baseline in Kidney Function
Time frame: 52 weeks
Frequency of Kidney Stone Events
Time frame: Through week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Gesa Schalk, MD principal investigator
KindernierenZentrum, Bonn, Germany
10 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Oxabact OC5 - Oxalobacter formigenes HC-1 in people with Primary Hyperoxaluria, run by OxThera at 10 sites in 7 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 10 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 10 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Tunisia, United Kingdom, United States, Belgium, France and 2 more countries. 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 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?
Yes. Results are posted in the registry (first posted 9 Dec 2021). 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?
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment (time frame: 52 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 10 Dec 2021.