Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria (NCT NCT03116685)
NCT ID: NCT03116685
Last Updated: 2021-12-10
Results Overview
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
25 participants
Primary outcome timeframe
52 weeks
Results posted on
2021-12-10
Participant Flow
Recruitment began 09 January 2018. Primary study completion 15 April 2021
Participant milestones
| Measure |
Oxabact OC5 Capsules
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
Placebo
Placebo: Placebo
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
12
|
|
Overall Study
COMPLETED
|
13
|
11
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Oxabact OC5 Capsules
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
Placebo
Placebo: Placebo
|
|---|---|---|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Baseline characteristics by cohort
| Measure |
Oxabact OC5 Capsules
n=13 Participants
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=12 Participants
Placebo
Placebo: Placebo
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
4 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
|
12.9 years
STANDARD_DEVIATION 6.4 • n=5 Participants
|
18.3 years
STANDARD_DEVIATION 16.5 • n=7 Participants
|
15.5 years
STANDARD_DEVIATION 12.4 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
11 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
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
24 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
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 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
Germany
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
6 participants
n=5 Participants
|
2 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Primary Hyperoxaluria Type
PH Type I
|
13 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Primary Hyperoxaluria Type
PH Type II
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Stage of Chronic Kidney Disease
Stage I
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Stage of Chronic Kidney Disease
Stage II
|
11 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Stage of Chronic Kidney Disease
Stage III
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: Full analysis set using a mixed repeated measures model for change for missing data
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter
Outcome measures
| Measure |
Oxabact OC5 Capsules
n=13 Participants
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=12 Participants
Placebo
Placebo: Placebo
|
|---|---|---|
|
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Baseline value
|
14.8 micromole/liter
Standard Deviation 5.7
|
14.4 micromole/liter
Standard Deviation 5.4
|
|
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Change from baseline to 52 weeks
|
-0.71 micromole/liter
Standard Deviation 1.34
|
2.35 micromole/liter
Standard Deviation 1.46
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: Patients with baseline and week 52 assessments
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.
Outcome measures
| Measure |
Oxabact OC5 Capsules
n=12 Participants
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=8 Participants
Placebo
Placebo: Placebo
|
|---|---|---|
|
Change From Baseline in Kidney Function
|
-1.35 millilitres/minute/1.73m2
Standard Error 2.68
|
-0.06 millilitres/minute/1.73m2
Standard Error 3.12
|
SECONDARY outcome
Timeframe: Through week 48Population: Number of events
Number of kidney stone events for each patient
Outcome measures
| Measure |
Oxabact OC5 Capsules
n=13 Participants
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=12 Participants
Placebo
Placebo: Placebo
|
|---|---|---|
|
Frequency of Kidney Stone Events
|
7 Number of kidney stone events
|
8 Number of kidney stone events
|
Adverse Events
Oxabact OC5 Capsules
Serious events: 3 serious events
Other events: 13 other events
Deaths: 0 deaths
Placebo Capsules
Serious events: 4 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Oxabact OC5 Capsules
n=13 participants at risk
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=12 participants at risk
Placebo
Placebo: Placebo
|
|---|---|---|
|
Renal and urinary disorders
Calculus urinary
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Renal colic
|
7.7%
1/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Renal impairment
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Ureterolithiasis
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
0.00%
0/12 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Urinary tract obstruction
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Bacterial pyelonephritis
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Escherichia urinary tract infection
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
0.00%
0/12 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Pyelonephritis acute
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
0.00%
0/12 • Adverse events were collected over the 52 week study period
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
Other adverse events
| Measure |
Oxabact OC5 Capsules
n=13 participants at risk
Oxabact OC5 - Oxalobacter formigenes HC-1
Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug
|
Placebo Capsules
n=12 participants at risk
Placebo
Placebo: Placebo
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
15.4%
2/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Tonsillitis
|
15.4%
2/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Urinary tact infection
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 3 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/13 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Pharyngitis
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
15.4%
2/13 • Number of events 3 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
General disorders
Pyrexia
|
15.4%
2/13 • Number of events 3 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 4 • Adverse events were collected over the 52 week study period
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
15.4%
2/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
0.00%
0/12 • Adverse events were collected over the 52 week study period
|
|
Nervous system disorders
Headache
|
15.4%
2/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
0.00%
0/12 • Adverse events were collected over the 52 week study period
|
|
Infections and infestations
Upper respiratory tract infection
|
7.7%
1/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Calculus urinary
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Nephrolithiasis
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
16.7%
2/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Renal and urinary disorders
Renal colic
|
15.4%
2/13 • Number of events 7 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 2 • Adverse events were collected over the 52 week study period
|
|
Gastrointestinal disorders
Vomiting
|
7.7%
1/13 • Number of events 1 • Adverse events were collected over the 52 week study period
|
33.3%
4/12 • Number of events 6 • Adverse events were collected over the 52 week study period
|
|
Gastrointestinal disorders
Abdominal pain
|
15.4%
2/13 • Number of events 2 • Adverse events were collected over the 52 week study period
|
8.3%
1/12 • Number of events 1 • Adverse events were collected over the 52 week study period
|
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