Porphyria clinical trials at UCSF

3 in progress, 2 open to eligible people

Porphyria is a condition where the body has trouble making heme, a part of blood. UCSF is studying the long-term effects of porphyria and its impact on pregnancy. UCSF is also exploring if a drug called bitopertin can safely treat certain types of porphyria in participants aged 12 and up.

Showing trials for

  • 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

  • Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

    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

Our lead scientists for Porphyria research studies include .

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