New pill shows promise for rare blood disorder in Long-Term trial
NCT ID NCT07177872
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took another drug (eculizumab) to see if NTQ5082 safely controls the disease long-term. The main goal is to check for side effects and whether it helps maintain healthy hemoglobin levels without needing blood transfusions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- NTQ5082 capsules
- What this could lead to
- If successful, this could provide a new long-term treatment option to control PNH, reducing symptoms like fatigue and the need for blood transfusions.
- What could go wrong
- This is an open-label study with no placebo, so results may be biased. Long-term safety and effectiveness are still being evaluated, and the drug may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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
Locations
-
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences ,
Tianjin, Tianjin Municipality, 300020, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A pill that could replace infusions for a rare blood disease?
- Every-8-Week shot could simplify PNH treatment
- Real-World test: can a targeted drug keep PNH in check for years?
- New transplant method aims to reduce complications in bone marrow failure patients
- No treatment, just observation: large study looks at PNH blood cells
- New hope for rare blood disorder: Long-Term study of XH-S003 underway