Clinical trial NCT04784455: Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy
Official title: Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Thrombotic Microangiopathies
Lead sponsor: AKARI Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-03-05
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy
Primary purpose: TREATMENT

Brief summary: 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

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

1. Patients weighing less than 5 kg.
2. Patients with a positive direct Coombs' test.
3. Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
5. Grade 4 Acute graft-versus-host disease (GVHD)
6. Received eculizumab or any other complement blocker therapy at any time.
7. 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10%) returned from their screening assessment, the patient should be withdrawn from the study

Primary outcomes:
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.
timeFrame: 24 weeks
Secondary outcomes:
measure: Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)
timeFrame: 24 weeks
measure: Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)
timeFrame: 24 weeks
measure: Normalisation of Lab Parameters
timeFrame: 24 weeks
measure: Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford Children's Hospital
state: California
city: Durham
country: United States
facility: Duke University Medical Center, Children's Health Center
state: North Carolina
city: Philadelphia
country: United States
facility: Children's Hospitall of Philadelphia
state: Pennsylvania
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
city: London
country: United Kingdom
facility: The Royal Marsden NHS Foundation Trust
city: London
country: United Kingdom
facility: St. Georges University Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH)
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
Study officials:
References: