New drug could keep rare blood disease in check
NCT ID NCT06831058
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This pilot study tests efgartigimod, a drug that lowers harmful antibodies, in 15 adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. Participants receive weekly infusions for 8 weeks, then are monitored for another 8 weeks. The goal is to see if the drug can safely raise ADAMTS13 activity and prevent relapses better than the current standard, rituximab.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- efgartigimod
- What this could lead to
- If it works, this could point toward a safer maintenance therapy to prevent relapses of iTTP without long-term immune suppression.
- What could go wrong
- This is a very small pilot study (15 people) comparing results to past patients, not a controlled trial. The effect may be temporary, and the drug may not prevent relapses better than existing treatments.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2025
- Expected to finish
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May 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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. Subject must provide a signed informed consent form 2. Subject is 18 years or older at the time of screening 3. Subject has a prior history of iTTP as defined by the presence of ADAMTS13 activity \< 10% with ADAMTS13 antibodies or inhibitor, thrombocytopenia (platelet count \< 100) and microangiopathic hemolytic anemia (defined by the presence of schistocytes on blood smear) 4. Subject is in clinical remission from iTTP (normal platelet count) for at least 90 days 5. Subject has ADAMTS13 activity \< 70% and \> 30% on 2 separate occasions separate by at least 7 days 6. Subject is at least 6 months from last dose of rituximab or other intravenous immunosuppression 7. If taking other oral immunosuppressants, no change in dose for at least 60 days 8. Female subjects of childbearing potential must present with a negative pregnancy test and agree to employ highly effective birth control measures for duration of study. Sexually active male subjects must agree to use an effective method of contraception for the duration of the study Exclusion Criteria: 1. Subject has been diagnosed with cTTP 2. Subject has been exposed to another investigational product within 30 days prior to enrollment or is scheduled to participate in another clinical study involving investigational product or investigational device during the course of the study 3. Subject is unable to understand the nature, scope, and possible consequences of the study. 4. Subject is pregnant or lactating 5. Subject has a known life-threatening hypersensitivity reaction to efgartigimod
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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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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
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