Could a new pill tame a rare blood disease? early safety trial begins
NCT ID NCT07760467
First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time
Summary
This early-stage trial is testing an experimental oral drug called TUL321 in healthy adults to see if it is safe and tolerable. The study will also measure how the drug moves through the body and whether food affects its absorption. TUL321 is being developed as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The trial involves giving single and multiple doses of the drug or a placebo to about 94 participants aged 18 to 45.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- TUL321
- What this could lead to
- If successful, this could support further development of TUL321 as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH).
- What could go wrong
- This is an early-phase trial in healthy people, so it won't show whether the drug works for PNH. Safety and side effects are the main focus, and the drug may not prove effective in later patient trials.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
Third Xiangya Hospital of Central South University
Changsha, Hunan, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug pegcetacoplan tested in teens with rare blood disorder
- New PNH drug VSA012 enters early human testing
- New pill CMS-D017 enters first human safety trials
- Thousands with blood diseases to be tracked in massive chinese registry
- New drug SLN12140 aims to control PNH in early trial
- New siRNA drug ADX-038 enters human trials for PNH anemia