Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis

NCT02000219 | Completed Phase 2 Results

• 1 other identifier: OC5-OL-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering plasma oxalate levels in patients with primary hyperoxaluria who are on dialysis.

Conditions

Primary Hyperoxaluria

Keywords

hyperoxaluria; oxalate; PH; dialysis

Outcome Measures

Primary Outcomes (1)

• Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline.

  Total plasma oxalate measured in umol/L

  Baseline (average of week 2 & 4 pretreatment); treatment weeks 6, 8 & 10 (on treatment 2, 4, & 6 weeks); off treatment weeks 12 & 14 (2 & 4 weeks off treatment); patients were then eligible for treatment up to 3 years & oxalate was measured every 6 months

Secondary Outcomes (3)

• Change in Pre Dialysis Plasma Oxalate (Free Plasma Oxalate) Level During Study Compared With Baseline.

  Baseline (average of week 2 & 4 pretreatment); treatment weeks 6, 8 & 10 (on treatment 2, 4, & 6 weeks); off treatment weeks 12 & 14 (2 & 4 weeks off treatment); patients were then eligible for treatment up to 3 years & oxalate was measured every 6 months

• Change in Left Ventricular Ejection Fraction From Baseline.

  At baseline and approximately every 6 months throughout the 3 year continued treatment.

• Speckle Tracking Echocardiography

  At baseline and approximately every 6 months throughout the 3 year continued treatment.

Study Arms (1)

Oxabact OC5 capsule

EXPERIMENTAL

This is an open-label study so all patients will receive the active drug product, Oxalobacter formigenes, OC5. This will be administered as an enteric-coated capsules twice daily for 6 weeks of treatment. In Germany, the protocol has been amended such that patients can receive OC5 for a further 3 year of continued treatment after the initial part of the study.

Biological: Oxalobacter formigenes

Interventions

Oxalobacter formigenes BIOLOGICAL

The dose will be (not less than) NLT ≥1E+09 colony forming units (CFU) twice daily. The dose (an enteric-coated capsule) will be administered orally with breakfast and dinner.

Also known as: Oxabact, OC5

Oxabact OC5 capsule

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent (as applicable for the age of the subject). An appendix to the informed consent will be signed by patients continuing on treatment after week 14.
• Male or female subjects ≥ 2 years of age. Subjects have to be able to swallow size 4 capsules twice daily for 6 weeks, or use a gastric tube that allows for administration of size 4 capsules.
• A diagnosis of PH (as determined by standard diagnostic methods).
• Patient should be on a stable dialysis regimen for at least two weeks before baseline.
• Pre-dialysis plasma oxalate ≥40 micromole/L.
• 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.

You may not qualify if:

• Inability to swallow size 4 capsules twice daily for 6 weeks or using a gastric tube not suited for administration of size 4 capsules via the tube.
• Ongoing treatment with immunosuppressive medication.
• 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.
• Use of antibiotics to which O. formigenes is sensitive, including current antibiotic use, or antibiotics use within 14 days of initiating study medication.
• Current treatment with a separate ascorbic acid preparation. Standard of care vitamin supplement for patients on dialysis is allowed.
• Pregnancy.
• 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.
• 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.
• 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.

Sponsors & Collaborators

• OxThera: lead
• FP7-SME-2013 Research for the benefit of SMEs program: collaborator

Study Sites (1)

Universitätsklinikum Bonn, Department of Paediatric Nephrology

Bonn, DE-53113, Germany

Location

MeSH Terms

Conditions

Hyperoxaluria, Primary; Hyperoxaluria

Condition Hierarchy (Ancestors)

Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Chief Operating Officer
• Organization: OxThera

elisabeth.lindner@oxthera.com

0046 86600223

Study Officials

• Gesa Schalk, M.D.

  University of Bonn

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2013
• First Posted: December 4, 2013
• Study Start: May 19, 2014
• Primary Completion: January 29, 2020
• Study Completion: January 29, 2020
• Last Updated: January 10, 2022
• Results First Posted: December 9, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)