AK901 Study of Nomacopan in HSCT-TMA
Research type
Research Study
Full title
Multicentre Study of Nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
IRAS ID
279760
Contact name
Robert Chiesa
Contact email
Sponsor organisation
Akari Therapeutics Plc
Eudract number
2020-000086-17
Duration of Study in the UK
4 years, 10 months, 28 days
Research summary
Summary of Research
Hematopoietic stem cell transplant (HSCT) associated thrombotic microangiopathy (HSCT-TMA) is a rare disease and in high-risk patients, such as the patients to be recruited in this study, the outcome is very poor with a reported death rates as high as 90%. There is no approved therapy for HSCT-TMA.
The complement system is a collection of proteins involved in the immune system process. There is evidence that the activation of this system is frequently involved with HSCT-TMA. Nomacopan, the drug being studied here, is expected to be effective in the treatment of HSCT-TMA as it works by reducing the activation of this system.
To date, nomacopan has been given to 64 people (32 healthy volunteers and 32 patients) and has generally been found to be well tolerated. There have been no serious drug-related reactions and the most frequent side effects have been mild to moderate injection site reactions.
The purpose of this study, is to determine whether nomacopan, is safe and effective in the treatment of this disease.
This study has two parts.
Part A will determine the effective dose of nomacopan and will have a total of 7 patients who will undergo treatment with nomacopan for 24 weeks followed by a safety assessment after 30 days. Using the data from part A, Part B will look at the effectiveness of the selected nomacopan dose in up to 65 patients for 24-weeks followed by a safety assessment after 30 days. Additionally, follow-up visits will be performed at one and two years.Study procedures will include blood and urine tests, physical examinations, nose & throat swabs & tests to check the heart's rhythm and electrical activity, this is called an electrocardiogram (ECG).
Summary of Results
The study was in two parts - Part A and Part B. However, the study was closed after Part A of the study due to the Sponsors decision to close the HSCT-TMA programme and not related to any safety issues. In Part A, 10 patients were treated with the study drug - nomacopan.
Overall, there were signs that the use of nomacopan may have had some beneficial effect in the management of hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).
Nomacopan was generally well tolerated. Of the 178 treatment-emergent adverse events, one was considered related to nomacopan (Grade 4 neutropenia) and two events were considered possibly related (Grade 4 respiratory failure and Grade 3 liver function test increased). There were 16 SAEs reported, 15 were considered unrelated and 1 SUSAR considered related to nomacopan– the aforementioned Grade 4 respiratory failure.
REC name
London - Central Research Ethics Committee
REC reference
20/LO/0878
Date of REC Opinion
6 Aug 2020
REC opinion
Further Information Favourable Opinion