Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA)NCT04247906
Single Patient Expanded Access Treatment Plan For The Investigational Product Narsoplimab
A study testing Narsoplimab in people with Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA), run by Omeros Corporation.
Trial summary
The purpose of the EAP is to provide access to hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) patients who may benefit from treatment with narsoplimab based on the medical judgement of their respective physicians. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA)
- What is being tested?
- Narsoplimab
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
- Patient (adult or pediatric) must have undergone allogeneic or autologous hematopoietic stem cell transplant (HSCT)
- Patient must be capable of understanding an informed consent form (ICF) or the parent or legal guardian of the patient is able to understand and sign a written ICF prior to the initiation of any treatment procedures.
- Patient must have HSCT- TMA defined as having thrombocytopenia and evidence of microangiopathic hemolytic anemia.
About this study
Official title: Single Patient Expanded Access Treatment Plan (EAP; Also Known As A Compassionate Use Plan) To Provide Patients Access To The Investigational Product Narsoplimab
The purpose of the EAP is to provide access to hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) patients who may benefit from treatment with narsoplimab based on the medical judgement of their respective physicians.
- Being tested
- Narsoplimab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NarsoplimabDrug
Also called OMS721
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Omeros Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Impatients N.V. trading as myTomorrows
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04247906
- Sponsor's protocol ID
- 721-TMA-EA
- Status last verified
- Mar 2026
- First submitted
- 28 Jan 2020
- Last updated
- 5 Mar 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Patient Expanded Access Treatment Plan (EAP; Also Known As A Compassionate Use Plan) To Provide Patients Access To The Investigational Product Narsoplimab
- ClinicalTrials.gov ID
- NCT04247906
- Acronym / sponsor ID
- 721-TMA-EA
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Omeros CorporationIndustry (pharma, biotech, devices)
- Collaborators
- Impatients N.V. trading as myTomorrows
- First posted
- 30 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the EAP is to provide access to hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) patients who may benefit from treatment with narsoplimab based on the medical judgement of their respective physicians.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NarsoplimabAlso: OMS721 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2026.
Related trials for Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA)
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Assessing the Incidence of Transplant Associated Thrombotic Microangiopathy (TA-TMA) in Adult Patients Undergoing Allogeneic Stem Cell Transplant (SCT)
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Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy
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Safety and Efficacy of Eculizumab in High-risk TA-TMA
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Identification of Plasma Biomarkers for Early Diagnosis of Transplant-associated Thrombotic Microangiopathy
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RecruitingObservational study
Thrombotic Microangiopathy (TMA) Associated With Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Adult Patients
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Narsoplimab in people with Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA), run by Omeros Corporation.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2026.