Status:
WITHDRAWN
Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease
Lead Sponsor:
Resverlogix Corp
Conditions:
Fabry Disease
Eligibility:
All Genders
18-75 years
Phase:
PHASE1
PHASE2
Brief Summary
Fabry Disease (FD) is a rare X-linked lysosomal storage disorder (LSD) caused by mutations in the GLA gene which translates into decreased activity or lack of function of the enzyme alpha-galactosidas...
Detailed Description
Fabry Disease (FD) is a rare X-linked lysosomal storage disorder (LSD) caused by mutations in the GLA gene coding for the enzyme alpha-galactosidase A (α-GAL A). As a consequence globotriaosylceramide...
Eligibility Criteria
Inclusion
- Subjects who meet the following criteria may be enrolled:
- Provide written informed consent before participation in the study.
- Aged between 18 and 75 years, inclusive.
- Diagnosis of Fabry disease, either
- receiving enzyme replacement therapy for at least 6 months at time of screening (Cohort 1).
- not receiving enzyme replacement therapy at time of screening and not having received enzyme replacement therapy in the past (Cohort 2).
- Female subjects must meet one of the following:
- If of childbearing potential, must have a negative urine pregnancy test and must also be willing to practice total abstinence or to use an approved (non-hormonal) form of birth-control throughout the study treatment phase and up to 28 days after the last study drug dose.
- OR-
- Meet at least one of the following criteria:
- Be postmenopausal, defined as having been amenorrheic for at least 2 years.
- Have had a hysterectomy or a bilateral oophorectomy.
- Male subjects who have not had a vasectomy must practice abstinence or use an approved method of birth control, including barrier contraception, throughout the study treatment phase and up to 3 months after the last study drug dose.
Exclusion
- Subjects who meet any of the following criteria will not be enrolled:
- Patients with stage 5 Chronic Kidney Disease (CKD) receiving renal replacement therapy with either hemodialysis or peritoneal dialysis, renal transplant or with eGFR \<15 ml/min/1.73m2.
- Patients with prior transplantations of organs or bone marrow.
- Patients with unstable cardiac condition including heart attack, stroke, uncontrolled atrial fibrillation or a major cardiac procedure within 3 months.
- Current or recent (within 12 months prior to Screening) treatment with cyclosporine.
- History of malignancy of any organ system, treated or untreated, within the past 2 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
- Evidence of cirrhosis from liver imaging or biopsy, a history of hepatic encephalopathy, esophageal or gastric varices, active hepatitis, or prior porta-caval shunt procedure, or a Child-Pugh score of at least 5 points.
- Have a screening 12-lead ECG considered clinically significant by the investigator requiring a corrective intervention in the short-term.
- Have any known allergy or intolerance to any compound in the test products or any other closely related compound.
- ALT or AST \>1.5 x ULN at Screen.
- Total bilirubin \>ULN at Screen.
- Use of diclofenac, clavulanic acid or regular use of acetaminophen \>1g per day.
- Have participated in a clinical study and received any investigational medication within the last 30 days preceding Visit 1 (Screening).
- Patients whose safety may be compromised by study participation due to, for example, an infection within the last 30 days.
- Are not, in the opinion of the investigator, able or willing to comply with the protocol.
Key Trial Info
Start Date :
November 22 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 22 2022
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT03228940
Start Date
November 22 2022
End Date
November 22 2022
Last Update
November 18 2023
Active Locations (1)
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1
Queen Elizabeth II Health Sciences Centre, Victoria General Site
Halifax, Nova Scotia, Canada, B3H2Y9