Name:
A Phase 2b/3 Adaptive, Randomized, Active-controlled Study Evaluating the Efficacy, Safety, and Tolerability of Povetacicept Versus Calcineurin Inhibitor in the Treatment of Primary Membranous Nephropathy.
Description:
This study will test an investigational medication called bitopertin. Bitopertin is being tested to see if it can help people with Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). EPP and XLP are rare genetic disorders characterized by the buildup of a substance called PPIX. PPIX buildup in the skin leads to painful reactions in people when exposed to sunlight and it can also be linked to liver disease. There is no cure for EPP and XLP. The available treatments
may involve protecting from light and sun, taking beta carotene for light sensitivity, managing liver
health, using sun protection medications, and other symptom management like wound care and pain relief.
This study will try to find out:
• If bitopertin works, i.e., if bitopertin can improve symptoms of EPP or XLP.
This will be done by looking at how long patients can be exposed to sunlight without feeling pain after 6 months of treatment, and by measuring blood PPIX levels over the 6-month treatment period.
• How safe bitopertin is, i.e., what side effects bitopertin causes and how bad they are.
This will be done by checking how well patients tolerate the treatment, based on side effects and laboratory test results, over the 6-month treatment period.
Stage:
Recruitment
Principal Investigator:
Dr Fiona Browne
Sponsor:
Disc Medicine
Network Partners
