Erythropoietic Protoporphyria clinical trials at UCSF
5 in progress, 2 open to eligible people
Erythropoietic protoporphyria is a rare inherited disorder that causes skin pain from light. UCSF is running a long study that records health and symptom changes. UCSF is also testing bitopertin in people ages 12 and older for safety and effect.
Bitopertin in Participants With EPP or XLP (APOLLO)
open to eligible people ages 12 years and up
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are: - Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP. - How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment. Researchers will compare bitopertin to a placebo look-alike substance that contains no drug. Participants will complete daily questionnaires and attend study visits for assessments.
San Francisco, California and other locations
Longitudinal Study of the Porphyrias
open to all eligible people
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.
San Francisco, California and other locations
Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Sorry, not currently recruiting here
To evaluate the long-term safety and tolerability of oral dersimelagon.
San Francisco, California and other locations
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Sorry, accepting new patients by invitation only
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
San Francisco, California and other locations
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
Sorry, in progress, not accepting new patients
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
San Francisco, California and other locations
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