Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis (NCT NCT02000219)
NCT ID: NCT02000219
Last Updated: 2022-01-10
Results Overview
Total plasma oxalate measured in umol/L
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
Baseline (average of week 2 & 4 pretreatment); treatment weeks 6, 8 & 10 (on treatment 2, 4, & 6 weeks); off treatment weeks 12 & 14 (2 & 4 weeks off treatment); patients were then eligible for treatment up to 3 years & oxalate was measured every 6 months
Results posted on
2022-01-10
Participant Flow
Participant milestones
| Measure |
Oxabact OC5 Capsule
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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14-Week Study Period
STARTED
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12
|
|
14-Week Study Period
COMPLETED
|
9
|
|
14-Week Study Period
NOT COMPLETED
|
3
|
|
Continuation Period
STARTED
|
8
|
|
Continuation Period
COMPLETED
|
3
|
|
Continuation Period
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Oxabact OC5 Capsule
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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14-Week Study Period
Physician Decision
|
1
|
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14-Week Study Period
Adverse Event
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1
|
|
14-Week Study Period
Underwent transplantation
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1
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Continuation Period
Withdrawal by Subject
|
1
|
|
Continuation Period
Had liver transplant
|
2
|
|
Continuation Period
Non-compliance with study drug
|
2
|
Baseline Characteristics
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis
Baseline characteristics by cohort
| Measure |
Oxabact OC5 Capsule
n=12 Participants
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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Age, Continuous
|
29.3 years
STANDARD_DEVIATION 17.0 • n=5 Participants
|
|
Sex: Female, Male
Female
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4 Participants
n=5 Participants
|
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Sex: Female, Male
Male
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8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
White
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12 Participants
n=5 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
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Region of Enrollment
France
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1 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
11 participants
n=5 Participants
|
|
Primary hyperoxaluria Type I
|
12 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (average of week 2 & 4 pretreatment); treatment weeks 6, 8 & 10 (on treatment 2, 4, & 6 weeks); off treatment weeks 12 & 14 (2 & 4 weeks off treatment); patients were then eligible for treatment up to 3 years & oxalate was measured every 6 monthsPopulation: Data presented by visit for patients completing each visit
Total plasma oxalate measured in umol/L
Outcome measures
| Measure |
Oxabact OC5 Capsule
n=12 Participants
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
Baseline value (average of week 2 and 4 pretreatment)
|
147.57 μmol/L
Standard Deviation 39.63
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
2 week treatment change from baseline
|
11.34 μmol/L
Standard Deviation 2.68
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
4 week treatment change from baseline
|
7.46 μmol/L
Standard Deviation 26.93
|
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
6 week treatment change from baseline
|
-5.68 μmol/L
Standard Deviation 21.02
|
|
Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
2 week off treatment change from baseline
|
-4.56 μmol/L
Standard Deviation 18.23
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
4 week off treatment change from baseline
|
-8.41 μmol/L
Standard Deviation 19.26
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 24 weeks change from baseline
|
-35.99 μmol/L
Standard Deviation 42.60
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 52 weeks change from baseline
|
-33.99 μmol/L
Standard Deviation 34.78
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 76 weeks change from baseline
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-28.04 μmol/L
Standard Deviation 38.45
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 104 weeks change from baseline
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-67.36 μmol/L
Standard Deviation 47.77
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 128 weeks change from baseline
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-46.25 μmol/L
Standard Deviation 26.90
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Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
continued treatment 156 weeks change from baseline
|
-44.65 μmol/L
Standard Deviation 54.75
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SECONDARY outcome
Timeframe: Baseline (average of week 2 & 4 pretreatment); treatment weeks 6, 8 & 10 (on treatment 2, 4, & 6 weeks); off treatment weeks 12 & 14 (2 & 4 weeks off treatment); patients were then eligible for treatment up to 3 years & oxalate was measured every 6 monthsPopulation: Patients completing each study visit are included
Comparison of free plasma oxalate compared to the four week baseline period.
Outcome measures
| Measure |
Oxabact OC5 Capsule
n=12 Participants
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
|
|---|---|
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Baseline
|
106.91 μmol/L
Standard Deviation 35.50
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|
Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at the end of six weeks of treatment
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1.59 μmol/L
Standard Deviation 18.77
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline 4 weeks after treatment for 6 weeks
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-6.50 μmol/L
Standard Deviation 17.31
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 24 weeks
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-10.17 μmol/L
Standard Deviation 15.27
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 52 weeks
|
2.52 μmol/L
Standard Deviation 13.79
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 76 weeks
|
-17.47 μmol/L
Standard Deviation 13.71
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 104 weeks
|
-47.29 μmol/L
Standard Deviation 30.46
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 128 weeks
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-38.54 μmol/L
Standard Deviation 10.59
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Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Change from baseline at 156 weeks
|
-5.36 μmol/L
Standard Deviation 20.99
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SECONDARY outcome
Timeframe: At baseline and approximately every 6 months throughout the 3 year continued treatment.Population: Data presented for patients with baseline and visit values recorded
Mean left ventricular ejection fraction measured as a percentage. Reference range 55-70%.
Outcome measures
| Measure |
Oxabact OC5 Capsule
n=12 Participants
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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Change in Left Ventricular Ejection Fraction From Baseline.
Baseline
|
50.20 Percent
Standard Deviation 10.26
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 24
|
59.43 Percent
Standard Deviation 4.08
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 52
|
59.83 Percent
Standard Deviation 5.85
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 80
|
59.60 Percent
Standard Deviation 5.18
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 104
|
59.40 Percent
Standard Deviation 5.94
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 128
|
58.00 Percent
Standard Deviation 2.16
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Change in Left Ventricular Ejection Fraction From Baseline.
Week 156
|
58.67 Percent
Standard Deviation 7.57
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SECONDARY outcome
Timeframe: At baseline and approximately every 6 months throughout the 3 year continued treatment.Population: Data presented for patients completing analyses
Global longitudinal strain (GLS), which measures the maximal shortening of myocardial longitudinal length during systole compared to the resting length in diastole. The normal range for GLS is considered to be \< -18%.
Outcome measures
| Measure |
Oxabact OC5 Capsule
n=9 Participants
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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Speckle Tracking Echocardiography
Baseline value
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-17.37 percentage of myocardial length change
Standard Deviation 5.30
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|
Speckle Tracking Echocardiography
Week 24
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-19.15 percentage of myocardial length change
Standard Deviation 2.52
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Speckle Tracking Echocardiography
Week 52
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-18.29 percentage of myocardial length change
Standard Deviation 3.27
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|
Speckle Tracking Echocardiography
Week 80
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-19.53 percentage of myocardial length change
Standard Deviation 1.65
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|
Speckle Tracking Echocardiography
Week 104
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-19.99 percentage of myocardial length change
Standard Deviation 2.96
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|
Speckle Tracking Echocardiography
Week 128
|
-19.60 percentage of myocardial length change
Standard Deviation 1.73
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|
Speckle Tracking Echocardiography
Week 156
|
-18.52 percentage of myocardial length change
Standard Deviation 1.65
|
Adverse Events
Oxabact OC5 Capsule
Serious events: 7 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Oxabact OC5 Capsule
n=12 participants at risk
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
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|---|---|
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Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Injury, poisoning and procedural complications
Shunt occlusion
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Injury, poisoning and procedural complications
Shunt stenosis
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Injury, poisoning and procedural complications
Shunt thrombosis
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Infections and infestations
Device related infection
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Infections and infestations
Device related sepsis
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Nervous system disorders
Nervous system disorder
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Nervous system disorders
Transient ischaemic attack
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
Other adverse events
| Measure |
Oxabact OC5 Capsule
n=12 participants at risk
This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment.
In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.
Oxalobacter formigenes: The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
41.7%
5/12 • Number of events 7 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
2/12 • Number of events 5 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Infections and infestations
Upper respiratory tract infection
|
58.3%
7/12 • Number of events 12 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Vascular disorders
Hypotension
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
25.0%
3/12 • Number of events 7 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
|
Surgical and medical procedures
Dialysis device insertion
|
16.7%
2/12 • Number of events 3 • Adverse events were collected from enrollment through last follow up visit, up to 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator may not publish the results of their cohort of subjects until the full study has been submitted for publication. They may not submit for publication or present the results of this study without allowing OxThera 30 days in which to review and comment on the pre-publication manuscript. The investigator may not submit the results of the study for publication without the prior consent of OxThera, unless the review period has passed and there has been no reaction from the sponsor.
- Publication restrictions are in place
Restriction type: OTHER