Hyperoxaluria, Primary
Conditions
Brief summary
Open label extension study of Oxabact OC5 in patients with primary hyperoxaluria
Detailed description
OC5-OL-02 (ePHex-OLE) is a 2 year, open-label, extension study to evaluate the long-term efficacy and safety of Oxabact OC5 for patients with primary hyperoxaluria who completed treatment in the parent double-blind, placebo-controlled study OC5-DB-02 (ePHex).
Interventions
BIOLOGICALOxabact OC5 - Oxalobacter formigenes Strain HC-1
Live, commensal bacteria
Sponsors
OxThera
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label study in which all patients will receive Oxabact OC5
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Signed informed consent (as applicable for the age of the subject). 2. Participation in and completion of study OC5-DB-02. 3. Subjects who had received vitamin B6 during OC5-DB-02 study should maintain a stable dose. Subjects not receiving vitamin B6 during OC5-DB-02 must be willing to refrain from initiating pyridoxine during study participation.
Exclusion criteria
1. Inability to swallow size 4 capsules. 2. Use of antibiotics to which O. formigenes is sensitive. 3. Current treatment with a separate ascorbic acid preparation. 4. Pregnant or lactating women (or women who are planning to become pregnant). 5. Women of childbearing potential who are not using adequate contraceptive precautions. 6. 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). 7. Participation in any interventional study of another investigational product, biologic, device, or other agent or not willing to forego other forms of investigational treatment during this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | 104 weeks | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Plasma Oxalate Concentration | Up to 104 weeks | 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. |
Countries
Belgium, Germany, Spain, Tunisia, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Oxabact OC5 Capsules; OC5 in ePHex Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria | 11 |
| Oxabact OC5 Capsules; Placebo in ePHex Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Oxabact OC5 - Oxalobacter formigenes Strain HC-1: Live, commensal bacteria | 11 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Physician Decision | 0 | 2 |
| Overall Study | Study termination by sponsor | 10 | 8 |
| Overall Study | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | Oxabact OC5 Capsules; OC5 in ePHex | Total | Oxabact OC5 Capsules; Placebo in ePHex |
|---|---|---|---|
| Age, Categorical <=18 years | 7 Participants | 15 Participants | 8 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 7 Participants | 3 Participants |
| Age, Continuous | 14.0 years STANDARD_DEVIATION 6.9 | 15.5 years STANDARD_DEVIATION 11 | 17.1 years STANDARD_DEVIATION 14.2 |
| Primary hyperoxaluria diagnosis PH Type I | 11 Participants | 21 Participants | 10 Participants |
| Primary hyperoxaluria diagnosis PH Type II | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 10 Participants | 21 Participants | 11 Participants |
| Region of Enrollment Belgium | 0 participants | 2 participants | 2 participants |
| Region of Enrollment Germany | 2 participants | 6 participants | 4 participants |
| Region of Enrollment Spain | 5 participants | 7 participants | 2 participants |
| Region of Enrollment Tunisia | 3 participants | 4 participants | 1 participants |
| Region of Enrollment United Kingdom | 1 participants | 2 participants | 1 participants |
| Region of Enrollment United States | 0 participants | 1 participants | 1 participants |
| Sex: Female, Male Female | 8 Participants | 12 Participants | 4 Participants |
| Sex: Female, Male Male | 3 Participants | 10 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 11 | 0 / 11 |
| other Total, other adverse events | 7 / 11 | 8 / 11 |
| serious Total, serious adverse events | 3 / 11 | 1 / 11 |
Outcome results
Primary
Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment
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.
Time frame: 104 weeks
Population: Patients with at least one efficacy measurement in the open-label period
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 |
| Oxabact OC5 Capsules; OC5 in ePHex | 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 | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 8 | 7.825 mL/min/1.73 m2 | Standard Deviation 4.264 |
| Oxabact OC5 Capsules; Placebo in ePHex | 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 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 10 | 4.530 mL/min/1.73 m2 | Standard Deviation 0.665 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Baseline value | 58.617 mL/min/1.73 m2 | Standard Deviation 20.858 |
| Oxabact OC5 Capsules; Placebo in ePHex | 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 | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 2 | -1.218 mL/min/1.73 m2 | Standard Deviation 5.255 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 12 | -0.994 mL/min/1.73 m2 | Standard Deviation 6.778 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 4 | -2.932 mL/min/1.73 m2 | Standard Deviation 9.889 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 21 | -3.340 mL/min/1.73 m2 | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 6 | 3.083 mL/min/1.73 m2 | Standard Deviation 8.614 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Kidney Function (eGFR) After 12 and 24 Months of Open-label Oxabact Treatment | Month 15 | 2.190 mL/min/1.73 m2 | Standard Deviation 0.764 |
Secondary
Change From Baseline in Plasma Oxalate Concentration
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.
Time frame: Up to 104 weeks
Population: Patients with at least one post-baseline plasma oxalate measurement
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Baseline value | 15.9 μmol/L | Standard Deviation 6.6 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 2 Change from baseline | 2.1 μmol/L | Standard Deviation 7.2 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 4 Change from baseline | 4.0 μmol/L | Standard Deviation 8.5 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 6 Change from baseline | 6.1 μmol/L | Standard Deviation 10 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 8 Change from baseline | 13.8 μmol/L | Standard Deviation 14.6 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 10 Change from baseline | 5.4 μmol/L | Standard Deviation 6 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 12 Change from baseline | 14.0 μmol/L | Standard Deviation 15.7 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 15 Change from baseline | 12.3 μmol/L | Standard Deviation 9.6 |
| Oxabact OC5 Capsules; OC5 in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 21 Change from baseline | -2.3 μmol/L | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 8 Change from baseline | -5.5 μmol/L | Standard Deviation 0.7 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 18 Change from baseline | 20.5 μmol/L | Standard Deviation 24.7 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 10 Change from baseline | -8.5 μmol/L | Standard Deviation 2.1 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Baseline value | 18.1 μmol/L | Standard Deviation 7.3 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 24 Change from baseline | -7.0 μmol/L | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 2 Change from baseline | 1.1 μmol/L | Standard Deviation 7 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 12 Change from baseline | 0.4 μmol/L | Standard Deviation 5.7 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 4 Change from baseline | 1.3 μmol/L | Standard Deviation 6.1 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 21 Change from baseline | 4.0 μmol/L | — |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 6 Change from baseline | 2.3 μmol/L | Standard Deviation 4.8 |
| Oxabact OC5 Capsules; Placebo in ePHex | Change From Baseline in Plasma Oxalate Concentration | Month 15 Change from baseline | 4.0 μmol/L | Standard Deviation 8.5 |