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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford Children's Hospital
Palo Alto, California, United States
Duke University Medical Center, Children's Health Center
Durham, North Carolina, United States
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
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Children's Hospitall of Philadelphia
Philadelphia, Pennsylvania, United States
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
Wroclaw, Poland
The Royal Marsden NHS Foundation Trust
London, United Kingdom
St. Georges University Hospital
London, United Kingdom
Great Ormond Street Hospital (GOSH)
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom