Refer a Patient
Have a Patient with Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) Who May Be Interested in the RestoreD Study?
The RestoreD Study is a phase 2, randomized, double-blind, placebo-controlled, dose-finding study. Approximately 126 participants will be enrolled in this study.
The investigational medication, ALXN2050, is a tablet taken twice a day orally that is being compared with a placebo in addition to standard of care for the treatment of LN and IgAN symptoms. Eventually, all participants enrolled in the RestoreD Study will have the opportunity to receive the investigational medication.
The duration of participation is approximately 164 weeks and consists of a screening period (up to 6 weeks), a blinded initial evaluation period (26 weeks), a blinded extended treatment period (24 weeks), and an open-label extension period (up to 2 years). Final study assessments will be performed 30 days after a participant’s final study treatment dose.
The primary objective of the RestoreD Study is to evaluate the safety and efficacy of oral ALXN2050 given in addition to standard of care and compared to placebo, demonstrate proof of concept for efficacy of factor D inhibition, and establish the best dose level in participants with LN or IgAN.
Key Inclusion Criteria
- Be between 18 and 75 years of age
- Have a confirmed diagnosis of LN (a form of systemic lupus erythematosus [SLE]) or primary IgAN based on kidney biopsy. Participants with LN must have an active renal flare.
- For participants in the LN cohort, the diagnosis of LN must have been confirmed by biopsy obtained ≤ 6 months prior to screening or during the screening period.
- The diagnosis of primary IgAN must have been established based on kidney biopsy obtained any time prior to or during the screening period.
- IgAN participants: must be on a stable and optimal dose of renin–angiotensin system (RAS) inhibitor treatment – this includes the highest tolerable dose of an angiotensin-converting enzyme (ACE) inhibitor or angiotensin-converting enzyme (ARB) dose for at least 3 months prior to consenting to the study. Additionally, no change in dose of RAS inhibitor treatment should be expected during the first 50 weeks of study treatment.
- LN or IgAN participants receiving treatment with sodium-glucose cotransporter-2 (SGLT-2) inhibitors (e.g., Jardiance, Invokana, etc.) must be on a stable dose for at least 3 months prior to consenting to the study. Additionally, no change in dose of RAS inhibitor treatment should be expected during the first 50 weeks of study treatment.
- Have proteinuria (high levels of protein in urine)
- Have controlled and stable blood pressure over the past 3 months prior to randomization
Key Exclusion Criteria
- Concomitant significant renal disease other than LN or IgAN on the most recent kidney biopsy prior to or during the screening period
- History of or planned solid organ or bone marrow transplant during the study
- History of seizure, splenectomy, complement deficiency, drug or alcohol abuse, N. meningitidis infection, or malignancy within 5 years prior to screening, with the exception of non-melanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence
- Prior history or clinically active SLE-related cerebritis, seizures, pericarditis, stroke, stroke syndrome, or secondary etiologies of IgAN (e.g., SLE, cirrhosis, celiac disease) requiring treatment
- Clinically active Henoch-Schonlein purpura (IgA vasculitis) requiring treatment
- Estimated GFR ≤ 30 mL/min/1.73 m2 during screening calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
There are additional eligibility requirements, which the study doctor can explain to interested individuals.