HELIOS: Long-term study of DISC-1459 (Bitopertin) in patients with EPP or XLP
Research type
Research Study
Full title
HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
IRAS ID
1013062
Contact name
Melanie Chin
Contact email
Sponsor organisation
Disc Medicine Inc.
Eudract number
2025-523275-27
Clinicaltrials.gov Identifier
Research summary
This study will test an investigational medication called bitopertin to see if it can help people with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). Main goal of this study is to find how safe bitopertin is, when used long term, i.e., what side effects bitopertin causes and how bad they are, when bitopertin is used for a long period of time. This will be done by checking how well patients tolerate the treatment, based on side effects and laboratory test results, throughout the treatment period. If addition, the study will also find if bitopertin works when used for a long period of time. This will be done by looking at how long patients can be exposed to sunlight without feeling pain, and by measuring blood protoporphyrin IX (PPIX) levels and blood bitopertin levels.
All eligible patients will receive bitopertin in this study, which will be given as tablets to be swallowed on an empty stomach once every morning. Bitopertin tablets come in contains either 10 mg or 30 mg dosesof bitopertin. Eligible adults patients will be given 60 mg once daily. The starting dose for adolescents will be 30 mg once daily. The study doctor may increase the dose for adolescents to 60 mg once daily after 2 weeks. Participants currently receiving 20 mg daily (in DISC-1459-301 study) will continue to receive the same dose.
The study will include up to 230 participants aged 12 years or older who are currently participating or have participated in the prior clinical study with bitopertin and also have completed the treatment phase and End-of-Study visit in those studies. The study has 2 parts: Screening and Treatment periods (which will last until bitopertin is available in market or until the participant withdraws, whichever is sooner): Participants will be required to visit the study clinic 1 time during the first month, followed by visits at Week 8, 16, 24 and 52, and then every 6 months. Participants will be asked to complete diaries and questionnaires.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
25/NW/0320
Date of REC Opinion
18 Dec 2025
REC opinion
Further Information Favourable Opinion