SEG101B2201 (SOLACE)
Research type
Research Study
Full title
A phase 2, multicentre, open-label study to assess appropriate dosing and to evaluate safety of Crizanlizumab, with or without hydroxyurea/hydroxycarbamide, in sequential, descending age groups of paediatric sickle cell disease patients with vaso-occlusive crisis
IRAS ID
253960
Contact name
Baba Inusa
Contact email
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2017-001747-12
Duration of Study in the UK
3 years, 6 months, 7 days
Research summary
Summary of Research
This study is in children (18 years to 6 months years old) with sickle cell disease (SCD). This inherited disease affects ~300,000 people born each year and causes abnormally shaped red blood cells which results in inflammation, chronic pain,
fatigue and low red blood cell levels with acute episodes of severe pain. Patients experience a reduced quality of life with severe organ damage and early mortality.Current limited treatment options include hydration, pain relief and antibiotics to manage the acute episodes of pain(crises)also called vaso occlusive crises (VOC). Hydroxyurea is the only FDA-approved therapy for crises, working by increasing the ratio of normal:mutated red blood cells but has uncertain side effects. Frequent blood transfusions are used and bone marrow transplants are effective but also come with long term side effects.
The treatment being investigated in this study, crizanlizumab is a non-marketed drug; However, Crizanlizumab was tested in healthy volunteers and is currently being tested in adult Sickle Cell Disease patients. Based on the evidence to date, no difference in the mechanism of action of crizanlizumab is expected between adult and paediatric patients. Crizanlizumab is a humanized monoclonal antibody which blocks the action of a group of cells responsible for blood vessel inflammation and reduces their obstruction by abnormal red blood cells, thus reducing pain and organ damage.
To address the unmet need in paediatric patients, the current proposed study is designed to confirm and establish appropriate dosing and to evaluate safety of Crizanlizumab in paediatric SCD patients with a history of VOC, with or without HU/HC, based on the favourable safety and efficacy profile of crizanlizumab in adults.
Patients will be on study for approximately 24 months and the aim is to recruit 4 patients in 2-3 UK sites.
Summary of Results
The English lay summary will be available one year post global LPLV (05May25). It will be available to PIs to share with patients and posted in the public domain on Novartis public website https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.novctrd.com%2F&data=05%7C02%7Clondoncentral.rec%40hra.nhs.uk%7C5c53d31fc46d498bf90208dd889f60a8%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638816940279921244%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=XYKeTs%2BioNIlVAcS6N2H8lLD31Ps0nO%2FMVuX1yIzo8I%3D&reserved=0
REC name
London - Central Research Ethics Committee
REC reference
19/LO/0391
Date of REC Opinion
24 May 2019
REC opinion
Further Information Favourable Opinion