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A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

Primary Purpose

Primary Hyperoxaluria

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Oxabact OC5 - Oxalobacter formigenes HC-1
Placebo
Sponsored by
OxThera
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Hyperoxaluria

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)
  2. A diagnosis of PH (as determined by standard diagnostic methods).
  3. eGFR < 90 ml/min/1.73 m2. The Schwartz formula will be used to estimate GFR for children (age below 18), and CKD-EPI formula will be used for adults (age 18 or above).
  4. Plasma oxalate concentration ≥10 μmol/L in total plasma oxalate.
  5. Male or female patients ≥ 2 years of age.
  6. Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must not change the dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating pyridoxine during study participation.

Exclusion Criteria:

  1. Inability to swallow size 4 capsules.
  2. Subjects that have undergone transplantation (solid organ or bone marrow).
  3. Patients requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period.
  4. 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.
  5. Use of antibiotics to which O. formigenes is sensitive. (This includes current antibiotic use, or antibiotics use within 14 days of initiating study medication).
  6. Current treatment with a separate ascorbic acid preparation.
  7. Pregnant women (or women who are planning to become pregnant) or lactating women.
  8. Women of childbearing potential who are not using adequate contraceptive precautions. Please see section 7.3 regarding requirements for contraception.
  9. 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).
  10. Participation in any interventional study of another investigational product, biologic, device, or other agent within 60 days prior to the first dose of OC5 or not willing to forego other forms of investigational treatment during this study.

Sites / Locations

  • Vanderbilt University Hospital
  • Centre Hospitalier Universitaire de Liège
  • Hôpital Robert Debré
  • Kindernierenzentrum Bonn
  • Hospital Vall d' Hebron
  • Hédi Chaker University Hospital
  • Sahloul University Hospital
  • Charles Nicolle University Hospital
  • Royal Free Hospital
  • Nottingham Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Oxabact OC5 capsules

Placebo capsules

Arm Description

Oxabact OC5 - Oxalobacter formigenes HC-1

Placebo

Outcomes

Primary Outcome Measures

Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter

Secondary Outcome Measures

Change From Baseline in Kidney Function
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.
Frequency of Kidney Stone Events
Number of kidney stone events for each patient

Full Information

First Posted
April 7, 2017
Last Updated
December 9, 2021
Sponsor
OxThera
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1. Study Identification

Unique Protocol Identification Number
NCT03116685
Brief Title
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Official Title
A Phase III Double-blind, Randomised Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Study Type
Interventional

2. Study Status

Record Verification Date
October 2021
Overall Recruitment Status
Completed
Study Start Date
January 9, 2018 (Actual)
Primary Completion Date
April 15, 2021 (Actual)
Study Completion Date
April 15, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
OxThera

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study will evaluate the efficacy and safety of OC5 in patients with PH.
Detailed Description
To evaluate the efficacy of Oxabact following 52 weeks treatment in subjects with maintained kidney function, but below the lower limit of the normal range (estimated glomerular filtration rate [eGFR] < 90 ml/min/1.73 m2) and a total plasma oxalate (Pox) concentration ≥ 10 μmol/L. Parameters to be evaluated include the ability to stabilise/reduce Pox concentration, to stabilise/improve kidney function and to reduce oxalate deposits in primary hyperoxaluria (PH) subjects.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Oxabact OC5 capsules
Arm Type
Experimental
Arm Description
Oxabact OC5 - Oxalobacter formigenes HC-1
Arm Title
Placebo capsules
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Biological
Intervention Name(s)
Oxabact OC5 - Oxalobacter formigenes HC-1
Intervention Description
Active study drug
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
Description
Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Change From Baseline in Kidney Function
Description
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.
Time Frame
52 weeks
Title
Frequency of Kidney Stone Events
Description
Number of kidney stone events for each patient
Time Frame
Through week 48

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) A diagnosis of PH (as determined by standard diagnostic methods). eGFR < 90 ml/min/1.73 m2. The Schwartz formula will be used to estimate GFR for children (age below 18), and CKD-EPI formula will be used for adults (age 18 or above). Plasma oxalate concentration ≥10 μmol/L in total plasma oxalate. Male or female patients ≥ 2 years of age. Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must not change the dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating pyridoxine during study participation. Exclusion Criteria: Inability to swallow size 4 capsules. Subjects that have undergone transplantation (solid organ or bone marrow). Patients requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period. 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. (This includes current antibiotic use, or antibiotics use within 14 days of initiating study medication). Current treatment with a separate ascorbic acid preparation. Pregnant women (or women who are planning to become pregnant) or lactating women. Women of childbearing potential who are not using adequate contraceptive precautions. Please see section 7.3 regarding requirements for contraception. 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). Participation in any interventional study of another investigational product, biologic, device, or other agent within 60 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, MD
Organizational Affiliation
KindernierenZentrum, Bonn, Germany
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vanderbilt University Hospital
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Centre Hospitalier Universitaire de Liège
City
Liège
Country
Belgium
Facility Name
Hôpital Robert Debré
City
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Kindernierenzentrum Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Hospital Vall d' Hebron
City
Barcelona
Country
Spain
Facility Name
Hédi Chaker University Hospital
City
Sfax
ZIP/Postal Code
3000
Country
Tunisia
Facility Name
Sahloul University Hospital
City
Sousse
ZIP/Postal Code
4054
Country
Tunisia
Facility Name
Charles Nicolle University Hospital
City
Tunis
ZIP/Postal Code
1008
Country
Tunisia
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Nottingham Children's Hospital
City
Nottingham
ZIP/Postal Code
NG7 2UH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

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