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This study will evaluate the efficacy and safety of OC5 in patients with PH.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Oxabact OC5 capsules | Experimental | Oxabact OC5 - Oxalobacter formigenes HC-1 |
|
| Placebo capsules | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oxabact OC5 - Oxalobacter formigenes HC-1 | Biological | Active study drug |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment | Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Kidney Function | Evaluation based on eGFR calculation using the 2009 creatinine-based "Schwartz bedside" equation (for children below 18 years of age) (Schwartz et al., 2009) and 2009 creatinine-based CKD-EPI equation for adults (Levey et al., 2009). Subjects who turn 18 during the study period were continuously evaluated using the Schwartz equation, ie the equation used at baseline was kept throughout the study. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gesa Schalk, MD | KindernierenZentrum, Bonn, Germany | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Vanderbilt University Hospital | Nashville | Tennessee | 37232 | United States | ||
| Centre Hospitalier Universitaire de Liège |
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Recruitment began 09 January 2018. Primary study completion 15 April 2021
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| ID | Title | Description |
|---|---|---|
| FG000 | Oxabact OC5 Capsules | Oxabact OC5 - Oxalobacter formigenes HC-1 Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug |
| FG001 | Placebo Capsules | Placebo Placebo: Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 8, 2021 | Sep 14, 2021 |
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| Placebo |
| Other |
Placebo |
|
| 52 weeks |
| Frequency of Kidney Stone Events | Number of kidney stone events for each patient | Through week 48 |
| Liège |
| Belgium |
| Hôpital Robert Debré | Paris | 75019 | France |
| Kindernierenzentrum Bonn | Bonn | 53127 | Germany |
| Hospital Vall d' Hebron | Barcelona | Spain |
| Hédi Chaker University Hospital | Sfax | 3000 | Tunisia |
| Sahloul University Hospital | Sousse | 4054 | Tunisia |
| Charles Nicolle University Hospital | Tunis | 1008 | Tunisia |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Nottingham Children's Hospital | Nottingham | NG7 2UH | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Oxabact OC5 Capsules | Oxabact OC5 - Oxalobacter formigenes HC-1 Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug |
| BG001 | Placebo Capsules | Placebo Placebo: Placebo |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Primary Hyperoxaluria Type | Primary Hyperoxaluria is the result of rare genetic mutations. The three main types of primary hyperoxaluria are the result of different gene deficiencies. Primary Hyperoxaluria Type I is the result of mutations in the AGXT gene (alanine-glyoxylate aminotransferase) Primary Hyperoxaluria Type II is the result of mutations in the GRHPR gene (glyoxylate reductase-hydroxypyruvate reductase) | Count of Participants | Participants |
| |||||||||||||||||
| Stage of Chronic Kidney Disease | Chronic Kidney Disease is categorized in five stages: I-V based on kidney function. Stage I is mild damage with an eGFR of 90 or greater Stage II is mild damage with an eGFR between 60 and 89 Stage III is moderate damage with an eGFR between 30 and 59 | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment | Change from baseline in total plasma oxalate concentration after 52 weeks of treatment in micromole/liter | Full analysis set using a mixed repeated measures model for change for missing data | Posted | Mean | Standard Deviation | micromole/liter | 52 weeks |
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| Secondary | Change From Baseline in Kidney Function | Evaluation based on eGFR calculation using the 2009 creatinine-based "Schwartz bedside" equation (for children below 18 years of age) (Schwartz et al., 2009) and 2009 creatinine-based CKD-EPI equation for adults (Levey et al., 2009). Subjects who turn 18 during the study period were continuously evaluated using the Schwartz equation, ie the equation used at baseline was kept throughout the study. | Patients with baseline and week 52 assessments | Posted | Least Squares Mean | Standard Error | millilitres/minute/1.73m2 | 52 weeks |
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| ||||||||||||||||||||||||||||||||||||
| Secondary | Frequency of Kidney Stone Events | Number of kidney stone events for each patient | Number of events | Posted | Number | Number of kidney stone events | Through week 48 |
|
|
Adverse events were collected over the 52 week study period
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Oxabact OC5 Capsules | Oxabact OC5 - Oxalobacter formigenes HC-1 Oxabact OC5 - Oxalobacter formigenes HC-1: Active study drug | 0 | 13 | 3 | 13 | 13 | 13 |
| EG001 | Placebo Capsules | Placebo Placebo: Placebo | 0 | 12 | 4 | 12 | 11 | 12 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Calculus urinary | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Urinary tract obstruction | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Bacterial pyelonephritis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Urinary tact infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| |
| Calculus urinary | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
The investigator may not publish the results of their cohort of subjects until the full study has been submitted for publication. They may not submit for publication or present the results of this study without allowing OxThera 30 days in which to review and comment on the pre-publication manuscript. The investigator may not submit the results of the study for publication without the prior consent of OxThera, unless the review period has passed and there has been no reaction from the sponsor.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Operating Officer | OxThera | 0046 86600223 | elisabeth.lindner@oxthera.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 8, 2021 | Sep 14, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006960 | Hyperoxaluria, Primary |
| ID | Term |
|---|---|
| D006959 | Hyperoxaluria |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| >=65 years |
|
| Male |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
|
| United States |
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| United Kingdom |
|
| Tunisia |
|
| Germany |
|
| Spain |
|
| PH Type II |
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| Stage II |
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| Stage III |
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