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

Primary Hyperoxaluria Clinical Trial

Official title:

A Phase III Double-blind, Randomised Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03116685
• Other study ID #: OC5-DB-02
• Status: Completed
• Phase: Phase 3
• Start date: January 9, 2018
• Est. completion date: April 15, 2021

Study information

• Verified date: October 2021
• Source: OxThera
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the efficacy and safety of OC5 in patients with PH.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 15, 2021
• Est. primary completion date: April 15, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hyperoxaluria, Primary
• Primary Hyperoxaluria

Intervention

Biological:
• Oxabact OC5 - Oxalobacter formigenes HC-1
Active study drug

Other:
• Placebo
Placebo

Locations

Country	Name	City	State
Belgium	Centre Hospitalier Universitaire de Liège	Liège
France	Hôpital Robert Debré	Paris
Germany	Kindernierenzentrum Bonn	Bonn
Spain	Hospital Vall d' Hebron	Barcelona
Tunisia	Hédi Chaker University Hospital	Sfax
Tunisia	Sahloul University Hospital	Sousse
Tunisia	Charles Nicolle University Hospital	Tunis
United Kingdom	Royal Free Hospital	London
United Kingdom	Nottingham Children's Hospital	Nottingham
United States	Vanderbilt University Hospital	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
OxThera

Countries where clinical trial is conducted

United States,  Belgium,  France,  Germany,  Spain,  Tunisia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	52 weeks
Secondary	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.	52 weeks
Secondary	Frequency of Kidney Stone Events	Number of kidney stone events for each patient	Through week 48