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Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis

Primary Purpose

Primary Hyperoxaluria

Status
Completed
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
Oxalobacter formigenes
Sponsored by
OxThera
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Hyperoxaluria focused on measuring hyperoxaluria, oxalate, PH, dialysis

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Signed informed consent (as applicable for the age of the subject). An appendix to the informed consent will be signed by patients continuing on treatment after week 14.
  2. Male or female subjects ≥ 2 years of age. Subjects have to be able to swallow size 4 capsules twice daily for 6 weeks, or use a gastric tube that allows for administration of size 4 capsules.
  3. A diagnosis of PH (as determined by standard diagnostic methods).
  4. Patient should be on a stable dialysis regimen for at least two weeks before baseline.
  5. Pre-dialysis plasma oxalate ≥40 micromole/L.
  6. Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must remain on the stable dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating vitamin B6 during study participation.

    Exclusion Criteria:

  7. Inability to swallow size 4 capsules twice daily for 6 weeks or using a gastric tube not suited for administration of size 4 capsules via the tube.
  8. Ongoing treatment with immunosuppressive medication.
  9. 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.
  10. Use of antibiotics to which O. formigenes is sensitive, including current antibiotic use, or antibiotics use within 14 days of initiating study medication.
  11. Current treatment with a separate ascorbic acid preparation. Standard of care vitamin supplement for patients on dialysis is allowed.
  12. Pregnancy.
  13. Women of childbearing potential who are not using adequate contraceptive precautions. Sexually active females, unless surgically sterile or at least 2 years post-menopausal, must be using a highly effective contraception (including oral, transdermal, injectable, or implanted contraceptives, IUD, abstinence, use of a condom by the sexual partner or sterile sexual partner) for 30 days prior to the first dose of OC5 and must agree to continue using such precautions during the clinical study.
  14. 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.
  15. Participation in any study of an investigational product, biologic, device, or other agent within 30 days prior to the first dose of OC5 or not willing to forego other forms of investigational treatment during this study.

Sites / Locations

  • Universitätsklinikum Bonn, Department of Paediatric Nephrology

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Oxabact OC5 capsule

Arm Description

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.

Outcomes

Primary Outcome Measures

Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
Total plasma oxalate measured in umol/L

Secondary Outcome Measures

Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Comparison of free plasma oxalate compared to the four week baseline period.
Change in Left Ventricular Ejection Fraction From Baseline.
Mean left ventricular ejection fraction measured as a percentage. Reference range 55-70%.
Speckle Tracking Echocardiography
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%.

Full Information

First Posted
November 20, 2013
Last Updated
December 9, 2021
Sponsor
OxThera
Collaborators
FP7-SME-2013 Research for the benefit of SMEs program
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1. Study Identification

Unique Protocol Identification Number
NCT02000219
Brief Title
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis
Official Title
A Phase 2 Open-label Multi-centre Study to Evaluate the Efficacy and Safety of Oxabact® to Reduce Plasma Oxalate in Subjects With Primary Hyperoxaluria Who Are on Dialysis
Study Type
Interventional

2. Study Status

Record Verification Date
October 2021
Overall Recruitment Status
Completed
Study Start Date
May 19, 2014 (Actual)
Primary Completion Date
January 29, 2020 (Actual)
Study Completion Date
January 29, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
OxThera
Collaborators
FP7-SME-2013 Research for the benefit of SMEs program

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering plasma oxalate levels in patients with primary hyperoxaluria who are on dialysis.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria
Keywords
hyperoxaluria, oxalate, PH, dialysis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Oxabact OC5 capsule
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Oxalobacter formigenes
Other Intervention Name(s)
Oxabact, OC5
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.
Description
Total plasma oxalate measured in umol/L
Time Frame
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
Secondary Outcome Measure Information:
Title
Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.
Description
Comparison of free plasma oxalate compared to the four week baseline period.
Time Frame
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
Title
Change in Left Ventricular Ejection Fraction From Baseline.
Description
Mean left ventricular ejection fraction measured as a percentage. Reference range 55-70%.
Time Frame
At baseline and approximately every 6 months throughout the 3 year continued treatment.
Title
Speckle Tracking Echocardiography
Description
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%.
Time Frame
At baseline and approximately every 6 months throughout the 3 year continued treatment.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Signed informed consent (as applicable for the age of the subject). An appendix to the informed consent will be signed by patients continuing on treatment after week 14. Male or female subjects ≥ 2 years of age. Subjects have to be able to swallow size 4 capsules twice daily for 6 weeks, or use a gastric tube that allows for administration of size 4 capsules. A diagnosis of PH (as determined by standard diagnostic methods). Patient should be on a stable dialysis regimen for at least two weeks before baseline. Pre-dialysis plasma oxalate ≥40 micromole/L. Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must remain on the stable dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating vitamin B6 during study participation. Exclusion Criteria: Inability to swallow size 4 capsules twice daily for 6 weeks or using a gastric tube not suited for administration of size 4 capsules via the tube. Ongoing treatment with immunosuppressive medication. 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, including current antibiotic use, or antibiotics use within 14 days of initiating study medication. Current treatment with a separate ascorbic acid preparation. Standard of care vitamin supplement for patients on dialysis is allowed. Pregnancy. Women of childbearing potential who are not using adequate contraceptive precautions. Sexually active females, unless surgically sterile or at least 2 years post-menopausal, must be using a highly effective contraception (including oral, transdermal, injectable, or implanted contraceptives, IUD, abstinence, use of a condom by the sexual partner or sterile sexual partner) for 30 days prior to the first dose of OC5 and must agree to continue using such precautions during the clinical study. 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. Participation in any study of an investigational product, biologic, device, or other agent within 30 days prior to the first dose of OC5 or not willing to forego other forms of investigational treatment during this study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gesa Schalk, M.D.
Organizational Affiliation
University of Bonn
Official's Role
Principal Investigator
Facility Information:
Facility Name
Universitätsklinikum Bonn, Department of Paediatric Nephrology
City
Bonn
ZIP/Postal Code
DE-53113
Country
Germany

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis

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