Thrombotic MicroangiopathiesPhase 3NCT04784455

Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

A Phase 3 study testing nomacopan (rVA576) in people with Thrombotic Microangiopathies, run by AKARI Therapeutics at 9 sites in 3 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy Read more about this study

Who is it for?: Ages 6 months to 18 years
Conditions the study is for: Thrombotic Microangiopathies
What is being tested?: nomacopan (rVA576)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 9 sitesin 3 countries
Study dates: 1 Feb 2021to 15 May 2024
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 6 months to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
Undergone allogeneic or autologous HSCT.
TMA diagnosis within a year of their allogeneic or autologous HSCT.
Clinical or histological diagnosis of TMA
Provision of written informed consent.
Provision of informed assent

Who cannot join

Patients weighing less than 5 kg.
Patients with a positive direct Coombs' test.
Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
Grade 4 Acute graft-versus-host disease (GVHD)
Received eculizumab or any other complement blocker therapy at any time.
Known hypersensitivity to the active ingredient or excipients
If an enrolled patient has a positive ADAMTS13 test (<10%) returned from their screening assessment, the patient should be withdrawn from the study

Trial locations and contacts

9 sites in 3 countries.

Country

City, postcode or hospital

9 sites in 3 countries

United States4 sites

Duke University Medical Center, Children's Health Center
Durham, North Carolina, 27710
Children's Hospital Los Angeles
Los Angeles, California, 90027
Stanford Children's Hospital
Palo Alto, California, 94304
Children's Hospitall of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

United Kingdom4 sites

The Royal Marsden NHS Foundation Trust
London, SM25PT
St. Georges University Hospital
London, SW170QT
Great Ormond Street Hospital (GOSH)
London, WC1N3JH
Royal Manchester Children's Hospital
Manchester, M139WL

Other trials in United Kingdom

Poland1 site

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
Wroclaw, 50556

Other trials in Poland

About this study

Official title: Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

Read the detailed description

This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days.

Part A: dose algorithm, safety and efficacy

Part B: safety and efficacy

Conditions: Thrombotic Microangiopathies
Being tested: nomacopan (rVA576)
Medical subject headings: Thrombotic Microangiopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

nomacopan (rVA576)Drug
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT

Study groups in NCT04784455
Study group	Role	What they receive
nomacopan (rVA576)	Experimental	The study population will consist of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and develop transplant-associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT Receives: Drug: nomacopan (rVA576)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24
Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Measured: 24 weeks

Secondary outcomes (4)

Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)
Plasma sC5b-9 ≤ upper limit of normal (ULN)
Measured: 24 weeks
Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)
Lactate dehydrogenase (LDH) ≤ULN
Measured: 24 weeks
Normalisation of Lab Parameters
Number of participants with a normalised haptoglobin value (where haptoglobin is within the normal ranges)
Measured: 24 weeks
Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.
Transfusion independence is defined as no platelet transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Feb 202115 May 2024

First posted5 Mar 2021
Study start1 Feb 2021
Primary completion15 May 2024
Study completion15 May 2024

Why it stopped: The early termination of this study is a business decision following a portfolio reprioritization plan. The decision is not related to any Efficacy, Safety or Clinical concerns regarding Nomacopan/rVA576

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Thrombotic MicroangiopathiesStudies 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: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy
Masking: None (open label)
Enrolment: 10 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04784455
Sponsor's protocol ID: AK901
Status last verified: Apr 2025
First submitted: 19 Nov 2020
Last updated: 5 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia

All Thrombotic Microangiopathies trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
ClinicalTrials.gov ID: NCT04784455
Acronym / sponsor ID: AK901
Conditions: Thrombotic Microangiopathies
Phase: Phase 3
Study type: Interventional
Recruitment status: TerminatedReason stopped: The early termination of this study is a business decision following a portfolio reprioritization plan. The decision is not related to any Efficacy, Safety or Clinical concerns regarding Nomacopan/rVA576
Enrolment: 10 participants (actual)
Lead sponsor: AKARI TherapeuticsIndustry (pharma, biotech, devices)
Study start: 1 Feb 2021 (actual)
Primary completion: 15 May 2024 (actual)
Study completion: 15 May 2024 (actual)
First posted: 5 Mar 2021 (actual)
Results: Posted 15 May 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

Detailed description

This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days.

Part A: dose algorithm, safety and efficacy

Part B: safety and efficacy

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy

Arm	Type	Receives
nomacopan (rVA576)	Experimental	Drug: nomacopan (rVA576)The study population will consist of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and develop transplant-associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT

Who may qualify

Ages: 6 months to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (6 listed)

Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
Undergone allogeneic or autologous HSCT.
TMA diagnosis within a year of their allogeneic or autologous HSCT.
Clinical or histological diagnosis of TMA
Provision of written informed consent.

Key exclusion criteria (8 listed)

Patients weighing less than 5 kg.
Patients with a positive direct Coombs' test.
Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
Grade 4 Acute graft-versus-host disease (GVHD)

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
nomacopan (rVA576)The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT	Drug	nomacopan (rVA576)

Primary outcome

Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
Time frame: 24 weeks
Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24 Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.

4 secondary outcomes

Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)
Time frame: 24 weeks
Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)
Time frame: 24 weeks
Normalisation of Lab Parameters
Time frame: 24 weeks
Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.
Time frame: 24 weeks

Results and safety

Results posted to the registry on 15 May 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Nomacopan	10	6

Primary outcome: Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.

Time frame: 24 weeks. Count of participants. Unit: Participants

Measure	Nomacopan n = 10
RBC transfusion independent for ≥ 28 days	2
Urine protein creatinine ratio (UPCR) ≤ 2 mg/mg for ≥ 28 days	4

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Duration of the study (Maximum of 2 year follow-up). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Nomacopan - 1.7 mg/kg	0 of 1 (0%)	0 of 1 (0%)	0 of 1
Nomacopan - 1.3 mg/kg	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3
Nomacopan - 1.0 mg/kg	0 of 6 (0%)	1 of 6 (16.7%)	0 of 6
Nomacopan - 0.30 mg/kg	6 of 10 (60%)	7 of 10 (70%)	4 of 10
Nomacopan - 0.45 mg/kg	2 of 3 (66.7%)	2 of 3 (66.7%)	0 of 3
Nomacopan - 0.60 mg/kg	0 of 0	0 of 0	0

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Thrombotic Microangiopathies

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia

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.

9 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing nomacopan (rVA576) in people with Thrombotic Microangiopathies, run by AKARI Therapeutics at 9 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 5 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States, United Kingdom, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

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.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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.

What does the primary outcome measure?

Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More. (time frame: 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04784455.

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 Jun 2025.