Danicopan Add-On Therapy for Paroxysmal Nocturnal Hemoglobinuria

Recruiting in Palo Alto (17 mi)

+86 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Alexion

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.

Research Team

Eligibility Criteria

This trial is for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have finished a previous Alexion study using Danicopan as an extra treatment. They must be able to consent, follow the study rules, and be vaccinated against Neisseria meningitidis. Those with conditions that could affect the study or pose risks can't join, nor can women who are pregnant, breastfeeding, or planning pregnancy.

Inclusion Criteria

Patient capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form and in this protocol

You took part in a previous study sponsored by Alexion that involved using danicopan along with a C5 inhibitor treatment.

I have been vaccinated against Neisseria meningitidis as required.

Exclusion Criteria

Patient permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study

Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant

I am not pregnant, breastfeeding, nor planning to become pregnant during the study.

Treatment Details

Interventions

Danicopan (Complement Inhibitor)

Trial OverviewThe trial is testing the long-term safety and effectiveness of Danicopan when added to another drug called a C5 inhibitor in patients with PNH. It's a single-arm extension study meaning everyone gets the same treatment without comparison to a placebo or other medication.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: DanicopanExperimental Treatment1 Intervention

Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.