Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 27 November 2023
• Main ID: NCT03228940
• Date of registration: 18/07/2017
• Prospective Registration: Yes
• Primary sponsor: Resverlogix Corp
• Public title: Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease
• Scientific title: An Open-Label Study to Assess the Safety and Effect on Key Biomarkers of Oral RVX000222 in Subjects With Fabry Disease
• Date of first enrolment: November 22, 2022
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/ct2/show/NCT03228940
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Canada

Contacts

• Name: Michael West, MD
• Affiliation: Queen Elizabeth II Health Sciences Centre, Victoria General Site

Key inclusion & exclusion criteria

Inclusion Criteria:

Subjects who meet the following criteria may be enrolled:

1. Provide written informed consent before participation in the study.

2. Aged between 18 and 75 years, inclusive.

3. Diagnosis of Fabry disease, either

1. receiving enzyme replacement therapy for at least 6 months at time of screening
(Cohort 1).

2. not receiving enzyme replacement therapy at time of screening and not having
received enzyme replacement therapy in the past (Cohort 2).

4. Female subjects must meet one of the following:

1. 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-

2. 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.

5. 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 Criteria:

Subjects who meet any of the following criteria will not be enrolled:

1. 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.

2. Patients with prior transplantations of organs or bone marrow.

3. Patients with unstable cardiac condition including heart attack, stroke, uncontrolled
atrial fibrillation or a major cardiac procedure within 3 months.

4. Current or recent (within 12 months prior to Screening) treatment with cyclosporine.

5. 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.

6. 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.

7. Have a screening 12-lead ECG considered clinically significant by the investigator
requiring a corrective intervention in the short-term.

8. Have any known allergy or intolerance to any compound in the test products or any
other closely related compound.

9. ALT or AST >1.5 x ULN at Screen.

10. Total bilirubin >ULN at Screen.

11. Use of diclofenac, clavulanic acid or regular use of acetaminophen >1g per day.

12. Have participated in a clinical study and received any investigational medication
within the last 30 days preceding Visit 1 (Screening).

13. Patients whose safety may be compromised by study participation due to, for example,
an infection within the last 30 days.

14. Are not, in the opinion of the investigator, able or willing to comply with the
protocol.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: RVX000222

Primary Outcome(s)

Changes in clinical laboratory parameters [Time Frame: 12 weeks]

Adverse events [Time Frame: 16 weeks]

Secondary Outcome(s)

Changes in markers of alpha-galactosidase (a-GAL A) deficiency [Time Frame: 12 weeks]

Changes in key markers of Chronic Kidney Disease-Bone Mineral Disease (CKD-BMD) [Time Frame: 12 weeks]

Initial uptake and steady-state level of RVX000222 [Time Frame: 12 weeks]

Changes in key markers of inflammation [Time Frame: 12 weeks]

Change in Alkaline Phosphatase [Time Frame: 12 weeks]

Secondary ID(s)

RVX222-CS-020

Source(s) of Monetary Support

Please refer to primary and secondary sponsors