Primary Hyperoxaluria (PH) MedDRA version: 20.1 Level: LLT Classification code 10020702 Term: Hyperoxalemia System Organ Class: 100000004861
Conditions
Sponsors
OxThera Intellectual Property AB
Eligibility
Sex/Gender
All
Inclusion 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 =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 7. Inability to swallow size 4 capsules. 8. Subjects that have undergone transplantation (solid organ or bone marrow). 9. Subjects requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period. 10. 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. 11. Use of antibiotics to which O. formigenes is sensitive. (This includes current antibiotic use, or antibiotics use within 14 days of initiating study medication.) 12. Current treatment with a separate ascorbic acid preparation. 13. Pregnant women (or women who are planning to become pregnant) or lactating women. 14. Women of childbearing potential who are not using adequate contraceptive precautions. 15. 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). 16. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of Oxabact® following 52 weeks treatment in patients with maintained kidney function, but below the lower limit of the normal range (eGFR < 90 ml/min/1.73 m2) and a total plasma oxalate concentration = 10 µmol/L at baseline ;Secondary Objective: To obtain additional safety data from 52 weeks continuous treatment with Oxabact® ;Primary end point(s): Change from baseline in total plasma oxalate concentration after 52 weeks of treatment.;Timepoint(s) of evaluation of this end point: after 52 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Key secondary endpoint: 1. Change from baseline in kidney function (eGFR) after 52 weeks of treatment. 2. Frequency of kidney stones events after 52 weeks of treatment. Stone events are defined as (i) Patient- or investigator reported symptoms, or (ii) Stone passages or removals, or (iii) Increase in number of stones assessed by ultrasound. Other endpoints 3. Percent change from baseline in total plasma oxalate concentration after 52 weeks of treatment. 4. Subjects achieving ‘near-normalization’ of total plasma oxalate concentration (<10 µmol/L) at least twice during weeks 24 to 52 of treatment. 5. Change from baseline in myocardial function in the heart as measured by Speckle Tracking Echocardiography (STE) and traditional echocardiography. 6. Change from baseline in free plasma oxalate concentration after 52 weeks of treatment. 7. Change from baseline in urinary oxalate excretion after 52 weeks of treatment. 8. Change from baseline in grade of nephrocalcinosis as assessed by Ultrasound. 9. Change in number of O. formigenes in stool. 10. Association between change in number of O. formigenes in stool and change in total plasma oxalate concentration. 11. Change from baseline in score of Quality of Life questionnaire. 12. Change from baseline in markers for renal function, renal tubular capacity and inflammation: Urine: magnesium, phosphorus, citrate, calcium, glycolate, creatinine, urea, calcium oxalate crystals, pH, osmolality and urinary volume. Blood: magnesium, phosphorus, citrate, calcium, glycolate, BUN, ALP, bicarbonate, CRP, WBC, creatinine and cystatine C ;Timepoint(s) of evaluation of this end point: 1. after 52 weeks of treatment 2. after 52 weeks of treatment 3. after 52 weeks of treatment 4. after 24 to 52 weeks of treatment 5. after 48 weeks of treatment 6. after 52 weeks of treatment 7. after 52 weeks of treatment 8. after 48 weeks of treatment 9. after 52 weeks of treatment 10. after 52 weeks of treatment 11. Quality of | — |
Countries
Belgium, France, Germany, Netherlands, Spain, Tunisia, United Kingdom, United States
Contacts
Public ContactDirector Regulatory Affairs
OxThera Intellectual Property AB
Outcome results
None listed