A Pivotal Phase 3 Study of the Efficacy and Safety of DMX-200 in Patients With focal segmental glomerulosclerosis (FSGS) Who Are Receiving an angiotensin II receptor blocker (ARB)
- Conditions
- Focal segmental glomerulosclerosis (FSGS)Renal and Urogenital - Kidney disease
- Registration Number
- ACTRN12622000066785
- Lead Sponsor
- Dimerix Bioscience Pty Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 286
DOUBLE BLIND PERIOD
1. Patients must be 12 to 80 years old (adolescents will only be recruited in Argentina, Mexico, UK, US).
2. A diagnosis of primary FSGS, genetic FSGS, or FSGS of undetermined cause.
4. If taking corticosteroids, the dosage must be stable for greater than or equal to 4 weeks prior to Screening and during Stabilization.
5. If taking aldosterone inhibitors, mineralocorticoid receptor antagonists, direct renin inhibitors, or sodium-glucose co-transporter-2 (SGLT2) inhibitors, or endothelin receptor antagonists (ERAs, including dual antagonists), the dose and regimen must be stable for greater than or equal to 12 weeks prior to Screening and during Stabilization.
7. Estimated GFR
8. Seated blood pressure less than or equal to 160/100 mm Hg (mean of 3 values) (patients greater than or equal to 18 years of age) or between the 5th and 95th percentile for age, sex, and height 29 (patients <18 years of age) at Screening.
9. Body weight greater than or equal to 35 kg (all patients) AND a BMI less than or equal to 40 kg/m2 (patients greater than or equal to 18 years of age) or between the 5th and 98th percentile for age and sex (patients <18 years of age) at Screening.
10. A female patient is eligible to participate if she is not pregnant or planning to become pregnant during the study, not breastfeeding, and at least one of the following conditions applies:
b. Is of childbearing potential and beginning at menarche, agrees to use a highly effective method of contraception consistently during the treatment period.
method of contraception consistently during the treatment period
11. A male patient with a female partner of childbearing potential is eligible to participate if he agrees to use acceptable contraception
OLE PERIOD
1. Patients who have completed participation in the double-blind period, including the Week 104 visit, and who may derive benefit from (continued) treatment with DMX-200, and/or continued follow-up
2. The patient received blinded Investigational Product throughout the duration of the double-blind period up to the Week 104 visit
3. The patient continues to meet the contraceptive requirements
DOUBLE-BLIND PERIOD
1. Has FSGS secondary to another condition.
2. History of type 1 diabetes mellitus, or uncontrolled type 2 diabetes mellitus (defined as glycated hemoglobin [HbA1c] >8%)
3. History of lymphoma, leukemia, or any active malignancy within the past 2 years
4. Active clinically significant hepatobiliary disease.
5. Documented history of heart failure (New York Heart Association Class III/IV) or a major adverse cardiac event within 12 weeks prior to Screening.
6. Has a physical, medical, or psychological condition, that in the opinion of the Investigator, may interfere with the evaluation the study.
7. The patient has a history of alcohol or illicit drug use disorder within 1 year prior to Screening.
8. Had a prior organ transplant or stem cell transplant, with the exception of corneal transplant.
9. Positive screening assessment for viral hepatitis B surface antigen, or anti-hepatitis C virus antibody AND positive HCV RNA, or human immunodeficiency virus 1 and 2.
10. Serum potassium levels >5.5 mmol/L at Screening.
11. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2 × upper limit of normal (ULN) at Screening
12. Treatment with immunosuppressant biological drugs, calcineurin inhibitors, cyclophosphamide, azathioprine, or mycophenolate mofetil within 12 weeks prior to Screening.
13. History of serious side effects or allergic response to an angiotensin II antagonist or has a known sensitivity to any components in the Investigational Product.
14. Unable to swallow oral medication.
15. Prior participation in any Dimerix-sponsored DMX-200 clinical study.
16. Participation in a clinical study with an Investigational Product within 28 days or 5 half-lives (whichever is longer) prior to Screening or plans to participate in another study during the course of this study.
17. Are study site personnel directly affiliated with this study and their immediate families.
OLE PERIOD
1. The patient has met the criteria for permanent IP discontinuation or study discontinuation.
2. Any safety concerns identified during the double-blind period which, in the Investigator's opinion, may interfere with the patient's continued participation during the OLE period.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method