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
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10%) returned from their screening assessment, the patient should be withdrawn from the study
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 |
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