DeFi
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients with Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF).
IRAS ID
254990
Contact name
Charlotte Benson
Contact email
Sponsor organisation
SpringWorks Subsidiary 2, PBC
Eudract number
2018-001991-39
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 10 months, 0 days
Research summary
Desmoids tumours, also referred to as aggressive fibromatosis (or DT/AF), are rare, locally invasive, slow growing soft tissue tumours. DT/AF develop more often in young adults with a peak age of about 30 years. They are more common in women than men.
This research study is a double-blind, placebo-controlled study and is being conducted to determine the efficacy and safety of nirogacestat (the study drug) in participants with progressing DT/AF. Placebo-controlled means that some of the participants will receive the study drug, while some receive placebo. Double-blind means that neither the study participant or their doctor will know whether they are receiving nirogacestat or placebo.
Nirogacestat is an investigational new drug. This means it has not yet been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) or any other health or regulatory authority. However, it can be used in this type of research study (known as a clinical trial).
The clinical trial will consist of 2 parts:
Double-blind phase:
During this phase of the study, participants will be screened for up to 28 days prior to the first dose of treatment (nirogacestat or placebo) and eligibility will be based on the inclusion and exclusion criteria.
Open-label phase:
Following disease progression, or completion of the double-blind phase, participants’ treatment assignment will be unblinded, and if eligible, participants will have the option to enrol in the optional open-label extension phase.
SpringWorks Therapeutics is the “Sponsor” of this clinical trial and is responsible for the development and oversight of the study. Up to 105 participants will take part in this clinical trial from about 60 medical centres in the UK, USA, Canada, and EU.
The primary objective of this study is to determine the efficacy (this means how well the treatment works) of nirogacestat in adult participants with progressing DT/AF.
Lay Summary of Results:
The SpringWorks NIR-DT-301 study involved 52 sites across 7 countries (Belgium, Canada, Germany, Italy, Netherlands, United Kingdom, and United States of America). The study evaluated the safety, efficacy, and tolerability of nirogacestat in adults with Progressing Desmoid Tumours / Aggressive Fibromatosis (DT/AF).
Participants were planned to receive nirogacestat or placebo via randomiszation. 142 participants were randomised with the first occurring 15-May-2019.
When the study results were analysed, there was an improvement observed in progression free survival in the nirogacestat group compared to placebo. Meaningful benefit was also seen in the nirogacestat group compared to placebo in pain and quality of life assessments.
Has the registry been updated to include summary results?: Yes
If yes - please enter the URL to summary results: https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fwww.clinicaltrialsregister.eu%252Fctr-search%252Fsearch%253Fquery%253DA%252BRandomized%25252C%252BDouble-Blind%25252C%252BPlacebo-Controlled%25252C%252B%252BPhase%252B3%252BTrial%252Bof%252BNirogacestat%252BVersus%252BPlacebo%252Bin%252BAdult%252B%252BPatients%252Bwith%252BProgressing%252BDesmoid%252BTumors%25252FAggressive%252B%252BFibromatosis%252B%252528DT%25252FAF%252529.%2FNBTI%2FXMPAAQ%2FAQ%2F083ba890-a634-4c1e-9eb9-1f395f3ee8e7%2F1%2FcmfBiJUt80&data=05%7C02%7Csurreyborders.rec%40hra.nhs.uk%7Cbf463357c078409e123e08de0d9e8e40%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638963171798620345%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=q3wBWQP4DVDLrwSqh07URLYTqnen0mpJThD3AN24L3A%3D&reserved=0
If no – why not?:
Did you follow your dissemination plan submitted in the IRAS application form (Q A51)?: Yes
If yes, describe or provide URLs to disseminated materials: N/A
If pending, date when dissemination is expected:
If no, explain why you didn't follow it:
Have participants been informed of the results of the study?: No
If yes, describe and/or provide URLs to materials shared and how they were shared:
If pending, date when feedback is expected:
If no, explain why they haven't: Investigators have been provided with CSR synopsis and can inform participants, as needed.
Have you enabled sharing of study data with others?: No
If yes, describe or provide URLs to how it has been shared:
If no, explain why sharing hasn't been enabled: Sharing of study data was not planned
Have you enabled sharing of tissue samples and associated data with others?: No
If yes, describe or provide a URL:
If no, explain why: Sharing of tissue samples and associated data was not planned.
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London - Surrey Borders Research Ethics Committee
REC reference
19/LO/0993
Date of REC Opinion
26 Jul 2019
REC opinion
Further Information Favourable Opinion