Primary Hyperoxaluria (PH) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Product Name: Oxabact_Oxalobacter formigenes, strain HC-1
Product Code: OC5 (enteric coated capsules)
Pharmaceutical Form: Gastro-resistant capsule, hard
INN or Proposed INN: OXALOBACTER FORMIGENES, S
Sponsors
OxThera Intellectual Property AB
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Signed informed consent (as applicable for the age of the patient). An appendix to the informed consent form will be signed by patients continuing on treatment after week 14. 2.Male or female patients = 2 years of age. Patients have to be able to swallow size 4 capsules twice daily, or use a gastric tube that allows for administration of size 4 capsules. 3.A diagnosis of PH (as determined by standard diagnostic methods). 4.Patient should be on a stable dialysis regimen for at least two weeks before baseline. 5.Plasma oxalate =40 micromole/L prior to the dialysis session. 6.Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must remain on the stable dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating vitamin B6 during study participation. Are the trial subjects under 18? yes Number of subjects for this age range: 1 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 11 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 7.Inability to swallow size 4 capsules twice daily, or using a gastric tube not suited for administration of size 4 capsules via the tube. 8.Ongoing treatment with immunosuppressive medication. 9.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. 10.Use of antibiotics to which O. formigenes is sensitive (see section 7.2.2), including current antibiotic use, or antibiotics use within 14 days of initiating study medication. 11.Current treatment with a separate ascorbic acid preparation. Standard of care vitamin supplement for patients on dialysis are allowed. 12.Pregnancy. 13.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. 14.Presence of a medical condition that the Principal Investigator considers likely to make the patient susceptible to adverse effect of study treatment or unable to follow study procedures. 15.Participation in any study of an investigational product, biologic, device, or other agent within 30 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 OC5 to reduce total plasma oxalate levels during OC5 treatment in patients with Primary Hyperoxaluria (PH) who are on dialysis.;Secondary Objective: To evaluate the efficacy of OC5 to reduce free plasma oxalate levels during OC5 treatment in patients with Primary Hyperoxaluria (PH) who are on dialysis. To evaluate the safety of OC5 in patients with PH who are on dialysis. To evaluate changes in number of O. formigenes in faeces following administration of OC5. To evaluate effect of stopping OC5 treatment for 4 weeks after the first 6 weeks of treatment. To evaluate results from Speckle Tracking Echocardiography or traditional echocardiography.;Primary end point(s): Change in plasma oxalate (total plasma oxalate) level during OC5 treatment, compared with baseline.;Timepoint(s) of evaluation of this end point: The change in total plasma oxalate levels will be based on the average from two baseline measurements (week 2 and week 4) and one measurement every 4th week throughout the first 24 months of the continued treatment period and every 3-4 months during the last 12 months of the continued treatment period. The change in plasma oxalate during 4 weeks follow-up period after the end of the 6 weeks treatment period with OC5, will be based on one measurement at week 10 and one measurement at week 14. The last observation will be carried forward in case of missing values at week 14 and/or week 10. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Change in plasma oxalate (free plasma oxalate) level during OC5 treatment, compared with baseline. 2. Change in plasma oxalate (total plasma oxalate) values from week 10 to week 14, following the 4 week off-treatment period. 3. Change in number of O. formigenes in faeces during OC5 treatment. 4. To evaluate results from Speckle Tracking Echocardiography or traditional echocardiography. SAFETY - Adverse events, haematology, clinical chemistry, urinalysis ;Timepoint(s) of evaluation of this end point: ad 1: see timepoint for primary endpoint ad 2: From Wk 11 to Wk 14 (4 weeks off OC5 treatment). ad 3: O. formigenes: weeks 4, 10, 14, every 2 months throughout year 1 and 2 of the continued treatment period and every 3-4 months thereafter. ad 4: Speckle Tracking Echocardiography (STE) and traditional echocardiography: if possible, at screening visit (+/- 1 month) or before start of continued treatment period (at visit week 14). STE and traditional echocardiography will then be repeated at week 24 in the continued treatment period and thereafter every 6 months until end of treatment. Safety endpoints: weeks 0, 4, 10, 14 during the first 14 weeks of the study, monthly throughout years 1 and 2 in the continued treatment period and every 3-4 months throughout year 3. | — |
Countries
Germany
Contacts
Public ContactChief Operating Officer
OxThera Intellectual Property AB
Outcome results
None listed