Trial Outcomes & Findings for An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria (NCT NCT03938272)
NCT ID: NCT03938272
Last Updated: 2021-12-08
Results Overview
Change in eGFR measured as mL/min/1.73m2. Baseline values are from the period before treatment with Oxabact. For the patients treated with Oxabact in the double-blind study, baseline is prior to treatment in that study. For patients who were treated with placebo in the double-blind study, baseline values are from the extension study, which was 48 to 52 weeks in the double-blind study.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
22 participants
Primary outcome timeframe
104 weeks
Results posted on
2021-12-08
Participant Flow
Participant milestones
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria was received in both the Core study OC-DB-02 (ePHex) and this open label extension
|
Oxabact OC5 Capsules; Placebo in ePHex
Placebo in the Core study OC-DB-02; and Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria in this open label extension
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
11
|
|
Overall Study
COMPLETED
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
11
|
10
|
Reasons for withdrawal
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria was received in both the Core study OC-DB-02 (ePHex) and this open label extension
|
Oxabact OC5 Capsules; Placebo in ePHex
Placebo in the Core study OC-DB-02; and Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria in this open label extension
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Physician Decision
|
0
|
2
|
|
Overall Study
Study termination by sponsor
|
10
|
8
|
Baseline Characteristics
An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Baseline characteristics by cohort
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
n=11 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Oxabact OC5 Capsules; Placebo in ePHex
n=11 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
14.0 years
STANDARD_DEVIATION 6.9 • n=5 Participants
|
17.1 years
STANDARD_DEVIATION 14.2 • n=7 Participants
|
15.5 years
STANDARD_DEVIATION 11.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
5 participants
n=5 Participants
|
2 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Tunisia
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Primary hyperoxaluria diagnosis
PH Type I
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Primary hyperoxaluria diagnosis
PH Type II
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 104 weeksPopulation: Patients with at least one efficacy measurement in the open-label period
Change in eGFR measured as mL/min/1.73m2. Baseline values are from the period before treatment with Oxabact. For the patients treated with Oxabact in the double-blind study, baseline is prior to treatment in that study. For patients who were treated with placebo in the double-blind study, baseline values are from the extension study, which was 48 to 52 weeks in the double-blind study.
Outcome measures
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
n=9 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Oxabact OC5 Capsules; Placebo in ePHex
n=10 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
|---|---|---|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Baseline value
|
74.193 mL/min/1.73 m2
Standard Deviation 9.952
|
58.617 mL/min/1.73 m2
Standard Deviation 20.858
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 8
|
-9.293 mL/min/1.73 m2
Standard Deviation 13.633
|
7.825 mL/min/1.73 m2
Standard Deviation 4.264
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 12
|
-5.840 mL/min/1.73 m2
Standard Deviation 11.672
|
-0.994 mL/min/1.73 m2
Standard Deviation 6.778
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 21
|
-12.193 mL/min/1.73 m2
|
-3.340 mL/min/1.73 m2
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 24
|
—
|
-7.110 mL/min/1.73 m2
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 2
|
-1.568 mL/min/1.73 m2
Standard Deviation 3.129
|
-1.218 mL/min/1.73 m2
Standard Deviation 5.255
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 4
|
-3.246 mL/min/1.73 m2
Standard Deviation 8.158
|
-2.932 mL/min/1.73 m2
Standard Deviation 9.889
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 6
|
-9.547 mL/min/1.73 m2
Standard Deviation 14.017
|
3.083 mL/min/1.73 m2
Standard Deviation 8.614
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 10
|
1.798 mL/min/1.73 m2
Standard Deviation 3.312
|
4.530 mL/min/1.73 m2
Standard Deviation 0.665
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 15
|
-5.862 mL/min/1.73 m2
Standard Deviation 8.954
|
2.190 mL/min/1.73 m2
Standard Deviation 0.764
|
|
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
Month 18
|
—
|
2.785 mL/min/1.73 m2
Standard Deviation 0.516
|
SECONDARY outcome
Timeframe: Up to 104 weeksPopulation: Patients with at least one post-baseline plasma oxalate measurement
Change in eGFR measured as mL/min/1.73m2. Baseline values are from the period before treatment with Oxabact. For the patients treated with Oxabact in the double-blind study, baseline is prior to treatment in that study. For patients who were treated with placebo in the double-blind study, baseline values are from the extension study, which was 48 to 52 weeks in the double-blind study.
Outcome measures
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
n=9 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Oxabact OC5 Capsules; Placebo in ePHex
n=10 Participants
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
|---|---|---|
|
Change From Baseline in Plasma Oxalate Concentration
Month 2 Change from baseline
|
2.1 μmol/L
Standard Deviation 7.2
|
1.1 μmol/L
Standard Deviation 7.0
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 4 Change from baseline
|
4.0 μmol/L
Standard Deviation 8.5
|
1.3 μmol/L
Standard Deviation 6.1
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 6 Change from baseline
|
6.1 μmol/L
Standard Deviation 10.0
|
2.3 μmol/L
Standard Deviation 4.8
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 8 Change from baseline
|
13.8 μmol/L
Standard Deviation 14.6
|
-5.5 μmol/L
Standard Deviation 0.7
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 10 Change from baseline
|
5.4 μmol/L
Standard Deviation 6.0
|
-8.5 μmol/L
Standard Deviation 2.1
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 12 Change from baseline
|
14.0 μmol/L
Standard Deviation 15.7
|
0.4 μmol/L
Standard Deviation 5.7
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 15 Change from baseline
|
12.3 μmol/L
Standard Deviation 9.6
|
4.0 μmol/L
Standard Deviation 8.5
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 18 Change from baseline
|
—
|
20.5 μmol/L
Standard Deviation 24.7
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 21 Change from baseline
|
-2.3 μmol/L
|
4.0 μmol/L
|
|
Change From Baseline in Plasma Oxalate Concentration
Month 24 Change from baseline
|
—
|
-7.0 μmol/L
|
|
Change From Baseline in Plasma Oxalate Concentration
Baseline value
|
15.9 μmol/L
Standard Deviation 6.6
|
18.1 μmol/L
Standard Deviation 7.3
|
Adverse Events
Oxabact OC5 Capsules; OC5 in ePHex
Serious events: 3 serious events
Other events: 7 other events
Deaths: 0 deaths
Oxabact OC5 Capsules; Placeob in ePHex
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
n=11 participants at risk
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Oxabact OC5 Capsules; Placeob in ePHex
n=11 participants at risk
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
|---|---|---|
|
Renal and urinary disorders
Nephrolithiasis
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
|
Renal and urinary disorders
Renal colic
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
|
General disorders
Pyrexia
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Infections and infestations
Pyelonephritis
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Infections and infestations
Pyelonephritis acute
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Injury, poisoning and procedural complications
Burns second degree
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
Other adverse events
| Measure |
Oxabact OC5 Capsules; OC5 in ePHex
n=11 participants at risk
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
Oxabact OC5 Capsules; Placeob in ePHex
n=11 participants at risk
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria
|
|---|---|---|
|
Infections and infestations
COVID-19
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
27.3%
3/11 • Number of events 3 • Open label extension up to 24 months of treatment
|
|
Infections and infestations
Urinary tract infection
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
|
Infections and infestations
Pharyngotonsillitis
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
|
Gastrointestinal disorders
Abdominal pain upper
|
18.2%
2/11 • Number of events 4 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Gastrointestinal disorders
Diarrhoea
|
9.1%
1/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
|
Renal and urinary disorders
Nephrolithiasis
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
|
Renal and urinary disorders
Calculus urinary
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
|
Renal and urinary disorders
Renal colic
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 4 • Open label extension up to 24 months of treatment
|
|
General disorders
Pyrexia
|
36.4%
4/11 • Number of events 4 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
18.2%
2/11 • Number of events 4 • Open label extension up to 24 months of treatment
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
27.3%
3/11 • Number of events 3 • Open label extension up to 24 months of treatment
|
|
Investigations
Eosinophil count increased
|
18.2%
2/11 • Number of events 2 • Open label extension up to 24 months of treatment
|
0.00%
0/11 • Open label extension up to 24 months of treatment
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
9.1%
1/11 • Number of events 1 • Open label extension up to 24 months of treatment
|
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