A Phase 3 interventional study of nomacopan (rVA576) in Thrombotic Microangiopathies, sponsored by AKARI Therapeutics. Terminated at 9 sites in 3 countries. Open to participants aged 6 Months to 18 Years. Per ClinicalTrials.gov, last updated 2025-06-05.
Sponsored by AKARI Therapeutics · Phase 3, Interventional, and Treatment
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
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
52 studies on the registry are indexed under Thrombotic Microangiopathies; 21 are open to participants now.
This study's enrollment of 10 is below the median of 40 across 24 interventional studies indexed under Thrombotic Microangiopathies.
Browse Thrombotic Microangiopathies studies →AKARI Therapeutics is the lead sponsor of 8 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
If an enrolled patient has a positive ADAMTS13 test (\<10%) returned from their screening assessment, the patient should be withdrawn from the study
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: 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
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.
Time frame: 24 weeks
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)
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)
Lactate dehydrogenase (LDH) ≤ULN
Time frame: 24 weeks
Normalisation of Lab Parameters
Number of participants with a normalised haptoglobin value (where haptoglobin is within the normal ranges)
Time frame: 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.
Time frame: 24 weeks
The study population consists of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and developed haematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT
| Milestone | Nomacopan |
|---|---|
| Started | 10 |
| Completed | 6 |
| Not completed | 4 |
| Withdrew: Death | 4 |
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.
| Participants | Nomacopan |
|---|---|
| RBC transfusion independent for ≥ 28 days | 2 |
| Urine protein creatinine ratio (UPCR) ≤ 2 mg/mg for ≥ 28 days | 4 |
Plasma sC5b-9 ≤ upper limit of normal (ULN)
| Participants | Nomacopan |
|---|---|
| Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less) | 10 |
Lactate dehydrogenase (LDH) ≤ULN
| Participants | Nomacopan |
|---|---|
| Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less) | 2 |
Number of participants with a normalised haptoglobin value (where haptoglobin is within the normal ranges)
| Participants | Nomacopan |
|---|---|
| Normalisation of Lab Parameters | 4 |
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.
| Participants | Nomacopan |
|---|---|
| Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More. | 0 |
Collected over Duration of the study (Maximum of 2 year follow-up). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Nomacopan - 1.7 mg/kg | 0/1 (0%) | 0/1 (0%) | 0/1 (0%) |
| Nomacopan - 1.3 mg/kg | 0/3 (0%) | 0/3 (0%) | 1/3 (33.3%) |
| Nomacopan - 1.0 mg/kg | 0/6 (0%) | 0/6 (0%) | 1/6 (16.7%) |
| Nomacopan - 0.30 mg/kg | 4/10 (40%) | 6/10 (60%) | 7/10 (70%) |
| Nomacopan - 0.45 mg/kg | 0/3 (0%) | 2/3 (66.7%) | 2/3 (66.7%) |
| Nomacopan - 0.60 mg/kg | — | — | — |
| Event | Nomacopan - 1.7 mg/kg | Nomacopan - 1.3 mg/kg | Nomacopan - 1.0 mg/kg | Nomacopan - 0.30 mg/kg | Nomacopan - 0.45 mg/kg | Nomacopan - 0.60 mg/kg |
|---|---|---|---|---|---|---|
| Device related infectionInfections and infestations | 0/1 | 0/3 | 0/6 | 0/10 | 1/3 | — |
| HypoglycaemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 0/10 | 1/3 | — |
| Posterior reversible encephalopathy syndromeNervous system disorders | 0/1 | 0/3 | 0/6 | 0/10 | 1/3 | — |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 0/1 | 0/3 | 0/6 | 2/10 | 1/3 | — |
| Multiple organ dysfunction syndromeGeneral disorders | 0/1 | 0/3 | 0/6 | 2/10 | 0/3 | — |
| Cardiac arrestCardiac disorders | 0/1 | 0/3 | 0/6 | 1/10 | 0/3 | — |
| CytopeniaBlood and lymphatic system disorders | 0/1 | 0/3 | 0/6 | 1/10 | 0/3 | — |
| Generalised oedemaGeneral disorders | 0/1 | 0/3 | 0/6 | 1/10 | 0/3 | — |
| Generalised tonic-clonic seizureNervous system disorders | 0/1 | 0/3 | 0/6 | 1/10 | 0/3 | — |
| HypertensionVascular disorders | 0/1 | 0/3 | 0/6 | 1/10 | 0/3 | — |
| Event | Nomacopan - 1.7 mg/kg | Nomacopan - 1.3 mg/kg | Nomacopan - 1.0 mg/kg | Nomacopan - 0.30 mg/kg | Nomacopan - 0.45 mg/kg | Nomacopan - 0.60 mg/kg |
|---|---|---|---|---|---|---|
| HyperkalaemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 5/10 | 1/3 | — |
| PyrexiaGeneral disorders | 0/1 | 0/3 | 0/6 | 5/10 | 1/3 | — |
| AnaemiaBlood and lymphatic system disorders | 0/1 | 0/3 | 0/6 | 4/10 | 1/3 | — |
| LeukopeniaBlood and lymphatic system disorders | 0/1 | 0/3 | 0/6 | 4/10 | 1/3 | — |
| HypermagnesaemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 4/10 | 1/3 | — |
| HypokalaemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 3/10 | 1/3 | — |
| HypocalcaemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 2/10 | 1/3 | — |
| HyponatraemiaMetabolism and nutrition disorders | 0/1 | 1/3 | 0/6 | 2/10 | 1/3 | — |
| HypophosphataemiaMetabolism and nutrition disorders | 0/1 | 0/3 | 0/6 | 2/10 | 1/3 | — |
| Blood bicarbonate decreasedInvestigations | 0/1 | 0/3 | 0/6 | 2/10 | 1/3 | — |
| Age, Customized(Participants) | Nomacopan |
|---|---|
| ≥ 0.5 to < 2 years | 1 |
| ≥ 2 to < 9 years | 3 |
| ≥ 9 to < 18 years | 6 |
| Sex: Female, Male(Participants) | Nomacopan |
|---|---|
| Female | 6 |
| Male | 4 |
| Race (NIH/OMB)(Participants) | Nomacopan |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Nomacopan |
|---|---|
| United States | 1 |
| United Kingdom | 9 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
This study is terminated, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nomacopan
Thrombotic Microangiopathies →
AKARI Therapeutics