for people ages 12 years and up (full criteria)
at San Francisco, California and other locations
study started
completion around
Principal Investigator
by Bruce Wang, MD
Headshot of Bruce Wang
Bruce Wang



This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Official Title

An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP)


Erythropoietic Protoporphyria, EPP, DISC-1459, porphyria, RO4917838


You can join if…

Open to people ages 12 years and up

  • Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
  • Aged ≥12 years upon study consent
  • Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants

You CAN'T join if...

  • Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
  • Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study
  • Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months
  • Planned treatment with afamelanotide or dersimelagon during the study
  • Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
  • If female, pregnant, or breastfeeding
  • Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
  • Score of PHQ-8 ≥10 at screening or any response of "yes" on the C-SSRS
  • Grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the stud


  • UCSF
    San Francisco California 94117 United States
  • Fred Hutchinson Cancer Center
    Seattle Washington 98109 United States

Lead Scientist at UCSF

  • Bruce Wang, MD
    My lab studies how the different cell types in the liver, in particular the hepatocyte, are generated during development, patterned and maintained during adulthood, and regenerate after injury. Our long-term goals are to improve the understanding of liver disease pathophysiology and develop novel methods of treatment for liver diseases, including cell replacement therapy.


not yet accepting patients
Start Date
Completion Date
Disc Medicine, Inc
Phase 2/3 Erythropoietic Protoporphyria Research Study
Study Type
Expecting 200 study participants
Last Updated