ePHex Clinical Trial

Oxalobacter formigenes (Oxabact®) versus placebo in patients with primary hyperoxaluria

A phase III double-blind, randomised study to evaluate the long-term efficacy and safety of Oxabact® in patients with primary hyperoxaluria.

Diagnosis: Primary Hyperoxaluria Type 1, 2 and 3

Phase: 3

Drug: Oxalobacter formigenes (Oxabact®)

EudraCT Number: 2017-000684-33

Patient Summary: To evaluate the long-term efficacy and safety of Oxabact® in patients with primary hyperoxaluria

Health Professional Summary: Protocol Entry Criteria:

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. Ongoing treatment with immunosuppressive medication.
  3. Patients requiring dialysis or at immediate risk for kidney failure or in need of dialysis in the near future.
  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, including current antibiotic use, or antibiotics use within 14 days of initiating study medication.
  6. Current treatment with a separate ascorbic acid preparation.
  7. Pregnancy.
  8. 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.
  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 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.

Special Study Parameters:

This double-blind, randomised international multi-center study will evaluate the efficacy and safety of OC5 in patients with PH. The study will include patients with maintained renal function but with an eGFR below the lower limit of the normal ranges (<90 ml/min/1.73 m2) and a total plasma oxalate concentration ≥ 10 μmol/L at baseline.

After the baseline period, eligible patients will be randomised to start on twice daily dosing of Oxabact® or placebo in a 1:1 ratio.

Estimated Enrollment:   22 patients will be randomized

Locations: 

United States, Massachusetts
Boston Children’s Hospital
Boston, Massachusetts, United States, 02115
Contact: Lillian Mead    857-218-3747 Lillian.mead@childrens.harvard.edu
Principal Investigator: Michelle Baum, MD

United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Contact: Jenn Manggaard, CCRP    507-255-7768 manggaard.jennifer@mayo.edu
Principal Investigator: John Lieske, MD

United States, Tennessee
Vanderbilt University Hospital
Nashville, Tennessee, United States, 37232
Contact: Edward R Gould, MD    615-936-1179 edward.r.gould@vumc.org
Principal Investigator: Edward R Gould, MD 

Belgium
Centre Hospitalier Universitaire de Liège
Liège, Belgium
Contact: Laure Collard, MD Laure.collard@chu.ulg.ac.be
Principal Investigator: Laure Collard, MD

France
Hôpital Femme Mère Enfant, CHU de Lyon
Lyon, France

Hôpital Robert Debré
Paris, France, 75019
Contact: Georges Deschênes, MD georges.deschenes@aphp.fr
Principal Investigator: Georges Deschênes, MD

Germany
Universitätsklinikum Bonn, Dept of Pediatric Nephrology
Bonn, Germany, 53113

Netherlands
Academy Medical Center, University of Amsterdam
Amsterdam, Netherlands

Spain
Hospital Vall d’ Hebron
Barcelona, Spain
Contact: Gema Ariceta, MD +34 934893082 gariceta@vhebron.net
Principal Investigator: Gema Ariceta, MD

United Kingdom
Royal Free Hospital
London, United Kingdom, NW3 2QG
Contact: Rayma Barros-Howe +44 2074726687 ext 33262

https://www.clinicaltrials.gov/ct2/show/NCT03116685?term=OC5-DB02&rank=1

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