Clinical trial NCT03116685: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Hyperoxaluria
Lead sponsor: OxThera
Sponsor class: INDUSTRY
First posted: 2017-04-17
Enrollment: 25
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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. 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.

Primary outcomes:
measure: Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment
timeFrame: 52 weeks
Secondary outcomes:
measure: Change From Baseline in Kidney Function
timeFrame: 52 weeks
measure: Frequency of Kidney Stone Events
timeFrame: Through week 48
Other outcomes:
Locations:
city: Nashville
country: United States
facility: Vanderbilt University Hospital
state: Tennessee
city: Liège
country: Belgium
facility: Centre Hospitalier Universitaire de Liège
city: Paris
country: France
facility: Hôpital Robert Debré
city: Bonn
country: Germany
facility: Kindernierenzentrum Bonn
city: Barcelona
country: Spain
facility: Hospital Vall d' Hebron
city: Sfax
country: Tunisia
facility: Hédi Chaker University Hospital
city: Sousse
country: Tunisia
facility: Sahloul University Hospital
city: Tunis
country: Tunisia
facility: Charles Nicolle University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham Children's Hospital
Study officials:
affiliation: KindernierenZentrum, Bonn, Germany
name: Gesa Schalk, MD
role: PRINCIPAL_INVESTIGATOR
References: