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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 78 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jun 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 1\) Age ≥ 18 years, regardless of gender. 2) Subjects who have previously received and completed NTQ5082 study treatment and, as determined by the investigator, have benefited from treatment and are likely to benefit from continued NTQ5082 treatment, or subjects in the control group receiving eculizumab. 3\) Subjects who have received ACYW135 group meningococcal and pneumococcal vaccines as required by the previous protocol, and whose vaccine protection period, as determined by the investigator, falls within the treatment duration of this study. 4\) Subjects who agree to use at least one effective contraceptive method during sexual intercourse with their partner from the time they sign the informed consent form until 4 weeks after the last dose, refrain from cryopreservation of sperm or eggs, and refrain from sperm or egg donation. 5\) Subjects who agree to sign the informed consent form and commit to abide by all study regulations. Exclusion Criteria: * 1\) Previous history of bone marrow/hematopoietic stem cell or solid organ transplant (e.g., heart, lung, kidney, liver). 2\) Previous history of splenectomy. 3) Previous history of recurrent invasive infection with encapsulated bacteria (e.g., meningococci, Streptococcus pneumoniae), previous history of systemic anti-tuberculosis treatment, or current tuberculosis infection. 4\) History of malignancy within 5 years prior to screening, excluding cured localized basal cell carcinoma of the skin, squamous cell carcinoma of the skin, papillary thyroid carcinoma, or carcinoma in situ of the cervix. 5\) Known or suspected hereditary complement deficiency or primary or severe secondary immunodeficiency. 6) Patients with a history of clinically significant kidney, heart, liver, or lung disease who are deemed unsuitable for participation by the investigator, including but not limited to severe kidney disease (e.g., eGFR \<30 mL/min/1.73 m²), advanced heart disease (e.g., NYHA class IV), or severe lung disease (e.g., severe pulmonary hypertension (WHO class IV). 7\) Patients suspected of being allergic to the study drug or any component of the study drug. 8\) Pregnant or lactating female subjects. 9) Other conditions deemed unsuitable for participation by the investigator.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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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.
- Hidden blood disorder may explain mysterious strokes in young adults
- 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