New drug combo aims to boost red blood cells in kids with rare blood disorder
NCT ID NCT06449001
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participants will receive weight-based danicopan doses for 12 weeks, with follow-up on blood counts, transfusion needs, and quality of life. The goal is better disease control, not a cure.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
danicopan
What this could lead to
If it works, this could offer a new add-on treatment option to better control anemia in children with PNH who still have symptoms despite current therapy.
What could go wrong
This is a very small early-phase trial with only 6 participants, so results may not apply broadly. The drug is added to existing treatments, not a standalone cure, and risks include infection and side effects from the combination.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Research Site
RECRUITINGSaskatoon, Saskatchewan, S7N 0W8, Canada
-
Research Site
RECRUITINGParis, 77019, France
-
Research Site
RECRUITINGLeeds, LS9 7TF, United Kingdom
-
Research Site
RECRUITINGLondon, SE5 9RS, United Kingdom