A clinical study to confirm the dose, effectiveness and safety of nomacopan in patients with Thrombotic Microangiopathy (clotting of the blood vessels) which can be a complication of bone marrow transplants.

Phase 1

• Conditions: MedDRA version: 20.0Level: PTClassification code 10043645Term: Thrombotic microangiopathySystem Organ Class: 10005329 - Blood and lymphatic system disorders; MedDRA version: 21.1Level: PTClassification code 10063581Term: Stem cell transplantSystem Organ Class: 10042613 - Surgical and medical procedures; MedDRA version: 22.0Level: PTClassification code 10067859Term: Allogenic stem cell transplantationSystem Organ Class: 10042613 - Surgical and medical procedures; MedDRA version: 22.0Level: PTClassification code 10081347Term: Autologous haematopoietic stem cell transplantSystem Organ Class: 10042613 - Surgical and medical procedures; Therapeutic area: Body processes [G] - Immune system processes [G12]

• Registration Number: EUCTR2020-000086-17-GB
• Lead Sponsor: Akari Therapeutics Plc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-03-24
• Last Update Posted: 15 September 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 72

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 100 days of their first allogeneic or autologous HSCT.
4. Clinical diagnosis of TMA with all of the following diagnostic criteria:
? elevated serum C5b-9 (80% or more of ULN for age)
? Urine protein creatinine ratio > 2 mg/mg (demonstrated on two separate morning samples, at least one day apart)
? elevated LDH (> ULN)
? thrombocytopaenia (< 50,000 per mm3)
? low haemoglobin concentration (< LLN) or Histological diagnosis of TMA with evidence of complement deposition:
? Urine protein creatinine ratio > 2 mg/mg (demonstrated on two separate morning samples, at least one day apart), and
? elevated serum C5b-9 (80% or more of ULN for age)
5. Provision of written informed consent.
6. Provision of informed assent

Are the trial subjects under 18? yes
Number of subjects for this age range: 72
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patients weighing less than 5 kg.
2. Patients with a positive direct Coomb's test.
3. Patients who do not receive nomacopan within 14 days of the initial diagnosis of TMA.
4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection (including unresolved or untreated Neisseria meningitidis infection and E. coli Shiga toxin) at the time of diagnosis of the TMA.
5. Grade 4 Acute GVHD (as per the Glucksberg grading system, see section 18.9).
6. Received eculizumab or any other complement blocker therapy at any time.
7. Known hypersensitivity to the active ingredient or excipients
8. Patients who are pregnant and/or breastfeeding. All females of
childbearing potential require a negative pregnancy test at screening.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified