CLNP023L12201 - LNP023 in autoimmune benign hematological disorders
Research type
Research Study
Full title
An open-label, multi-center, phase 2 basket study to assess efficacy, safety and pharmacokinetics of iptacopan (LNP023) in participants with autoimmune benign hematological disorders
IRAS ID
300992
Contact name
Nicola Cooper
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2021-002039-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 10 months, 11 days
Research summary
This is an open-label, single-arm (within each cohort), multi-center, study to assess the efficacy, safety and pharmacokinetics of iptacopan in participants with autoimmune benign haematological disorders.
The study consists of a screening period, a 12-week treatment period (Part A), a washout (for responders, i.e. patients who respond to initial treatment)/follow up (non-responders, i.e. patients who do not respond to the treatment in the first 24 weeks) after Part A.
Patients who have responded to the treatment in Part A will be invited to participate in an additional treatment period of up to 24 months (Part B).
The total study duration from screening until end-of-study visit (EOS) is approximately 6 months for participants not meeting the primary endpoint (non-responders) and up to 31 months for participants meeting the primary endpoint (responders).
Lay Summary of Results
The English lay summary will be available one year post global LPLV (17/05/25). 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%7Csurreyborders.rec%40hra.nhs.uk%7C74957252065f4a206b5a08dd2b1970b9%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638714110439011800%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=Nfk1en23LoYBz9LG%2BVejOYUCWubscAG8egXt9f%2BgouE%3D&reserved=0.
Has the registry been updated to include summary results?: No
If yes - please enter the URL to summary results:
If no – why not?: Results due to be posted on the registry 1 year after LPLV on 17 May 2025.
Did you follow your dissemination plan submitted in the IRAS application form (Q A51)?: Pending
If yes, describe or provide URLs to disseminated materials:
If pending, date when dissemination is expected: 17/05/2025
If no, explain why you didn't follow it:
Have participants been informed of the results of the study?: Pending
If yes, describe and/or provide URLs to materials shared and how they were shared:
If pending, date when feedback is expected: 17/05/2025
If no, explain why they haven't:
Have you enabled sharing of study data with others?: Yes
If yes, describe or provide URLs to how it has been shared: This trial data availability is according to the criteria and process described on the https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicalstudydatarequest.com%2F&data=05%7C02%7Csurreyborders.rec%40hra.nhs.uk%7C74957252065f4a206b5a08dd2b1970b9%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638714110439023975%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=3H7zxBqLIBC1J4DCkzs6i8jjZ8suTjZrWzkklnKZiD0%3D&reserved=0
If no, explain why sharing hasn't been enabled:
Have you enabled sharing of tissue samples and associated data with others?: No
If yes, describe or provide a URL:
If no, explain why: Not Allowed as per Novartis policy to maintain patient privacy.REC name
London - Surrey Borders Research Ethics Committee
REC reference
21/LO/0755
Date of REC Opinion
29 Oct 2021
REC opinion
Favourable Opinion