Increlex Global Patient Registry
Research type
Research Study
Full title
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents with Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD)
IRAS ID
305764
Contact name
Helen Storr
Contact email
Sponsor organisation
Ipsen Pharma
Clinicaltrials.gov Identifier
305764, ENCePP register number; R00790-Ltr- 4-Patel, Manchester University NHS Foundation Trust; Ipsen Increlex EU Registry, Barts Health NHS Trust; 27952800002, Leeds Teaching Hospitals NHS Trust; 09CM06, Great Ormond Street Hospital for Children NHS Foundation Trust
Duration of Study in the UK
21 years, 8 months, 2 days
Research summary
This is a multicentre, global, prospective, non-interventional, observational, open-ended, post-authorisation safety surveillance registry designed to collect safety and effectiveness data on the use of Increlex® in children and adolescents with Severe Primary Insulin-like Growth Factor-1 Deficiency. Data on treatment exposure, compliance and QoL of participants will also be collected.
The decision to prescribe Increlex® will be made prior to and independently of the decision to enrol the patient in the registry. The data collected for a participant will be those that exist in their medical records as part of standard medical care except for additional QoL assessments and post treatment safety follow-up in the context of fulfilling the Specific Obligation. Data will be collected utilising an electronic Case Report Form via the internet.
The primary objective of the registry is to collect safety data in children and adolescents receiving Increlex® for the treatment of SPIGFD, in particular to permit the description and incidence of serious adverse events, targeted AEs and all AEs. Secondary objectives include the description of long-term safety data in all participants, and in the subset exposed to Increlex® for at least 3 years, and of the effectiveness of Increlex® treatment up to adult height, evaluation of Increlex® exposure (dose and treatment duration) and compliance, description of Increlex® effectiveness according to the dose, assessments of QoL during and after treatment and description of the timeframe of occurrence of neoplasia and hypoglycaemia.
This expanded global registry will increase enrolment and provide the opportunity to monitor the safety of Increlex® and the risks identified within the EU RMP in a larger group of children and adolescents with SPIGFD. It will also enable the Sponsor to ensure that the product is used according to the locally approved product information and to measure the effectiveness of the additional risk minimisation measures.REC name
London - Westminster Research Ethics Committee
REC reference
21/PR/1351
Date of REC Opinion
17 Nov 2021
REC opinion
Further Information Favourable Opinion