A rollover study of avapritinib (also called BLU-285) in adults Who Participated in a Previous Avapritinib Study

Start 5 Nov 2024 · Status Ongoing, recruiting · 4 EU/EEA countries · 5 sites · Protocol BLU-285-2404

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)

Status Ongoing, recruiting

Participants planned 60

Countries 4

Sites 5

Systemic Mastocytosis

The primary objective of this rollover study is to evaluate the long term-safety of avapritini as assessed by incidence of SAEs, AESIs and AEs leading to discontinuation

Key facts

Sponsor: Blueprint Medicines Corp.
Participant type: Patients
Age range: 18-64 years
Gender: Male and Female
Therapeutic area: Diseases [C] - Neoplasms [C04]
Trial duration: 5 Nov 2024 → ongoing
Decision date (initial): 2024-10-08
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy

The primary objective of this rollover study is to evaluate the long term-safety of avapritini as assessed by incidence of SAEs, AESIs and AEs leading to discontinuation

Secondary objectives 1

The secondary objective of this rollover study is to provide access to avapritinib for patients who had clinical benefit from avapritinib treatment in a Blueprint Medicines sponsored study.

Conditions and MedDRA coding

Systemic Mastocytosis

Version	Level	Code	Term	System organ class
21.1	PT	10042949	Systemic mastocytosis	100000004864

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

Participated in a Blueprint Medicines sponsored avapritinib clinical study and is either receiving or on interrupted treatment with avapritinib. If the patient has been on drug hold for over 56 days, the Medical Monitor should be consulted prior to enrollment and study drug administration. The Sponsor reserves the right to not roll over patients who have been on drug hold for an extended time period. - Inclusion of pediatric patients with CNS tumors applies in Canada only.
Completed the parent study treatment requirements and demonstrated compliance with the parent study requirements, as assessed by the Principal Investigator.
Continue to clinically benefit from treatment with avapritinib, as determined by the Principal Investigator from the parent study.
Able to give written informed consent. Written informed consent must be obtained prior to enrolling in the rollover study and receiving the study treatment. If consent cannot be expressed in writing, then the consent must be formally documented and witnessed, ideally via an independent trusted witness. For pediatric patients, the informed consent may be obtained from the patient’s parent or legal guardian. Parent(legal guardian consent will be determinde by local, regional and/or national guidelines.
Agree to continue to use highly effective contraception as defined in this protocol.
Female patients of childbearing potential must have negative highly sensitive serum pregnancy test within 20 days before the first dose of avapritinib. Pregnancy will be documented by a serum beta human chorionic gonadotropin (β-hCG) pregnancy test consistent with pregnancy. Women with β-hCG values that are within the range for pregnancy but are not pregnant (false-positives) may be enrolled with written approval of the Sponsor after pregnancy has been excluded. Women of non-childbearing potential (postmenopausal, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) do not require a serum β-hCG pregnancy test."

Exclusion criteria 3

Patient is participating in another interventional study.
Patient is unwilling or unable to comply with study procedures and study restrictions.
Patient is pregnant and/or breastfeeding.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

no primary end points

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Avapritinib

PRD8835309 · Product

Active substance: Avapritinib
Pharmaceutical form: FILM-COATED TABLET
Route of administration: ORAL USE
Max daily dose: 0 mg milligram(s)
Max total dose: 0 mg milligram(s)
Max treatment duration: 39 Month(s)
Authorisation status: Not Authorised
MA holder: BLUEPRINT MEDICINES
Paediatric formulation: No
Orphan designation: No

Avapritinib

PRD8835310 · Product

Active substance: Avapritinib
Pharmaceutical form: FILM-COATED TABLET
Route of administration: ORAL USE
Max daily dose: 0 mg milligram(s)
Max total dose: 0 mg milligram(s)
Max treatment duration: 39 Week(s)
Authorisation status: Not Authorised
MA holder: BLUEPRINT MEDICINES
Paediatric formulation: No
Orphan designation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Blueprint Medicines Corp.

Sponsor organisation: Blueprint Medicines Corp.
Address: 45 Sidney Street
City: Cambridge
Postcode: 02139-4133
Country: United States

Scientific contact point

Organisation: Blueprint Medicines Corp.
Contact name: Medical Monitor

Public contact point

Organisation: Blueprint Medicines Corp.
Contact name: Medical Monitor

Third parties 3

Organisation	City, country	Duties
Endpoint Clinical Inc. ORG-100040567	Wakefield, United States	Interactive response technologies (IRT)
PPD Development LP ORG-100011560	Wilmington, United States	Code 13, Other
Syneos Health Netherlands B.V. ORG-100013861	Amsterdam, Netherlands	On site monitoring, Code 12, Code 2, Code 5, Data management, E-data capture

Locations

4 EU/EEA countries · 5 investigational sites

By country

Country	MS status	Planned subjects	Sites
Belgium	Authorised, recruiting	7	1
Italy	Ongoing, recruiting	2	1
Netherlands	Authorised, recruiting	7	2
Norway	Authorised, recruiting	10	1
Rest of world United Kingdom	—	34	—

Investigational sites

Belgium

1 site · Authorised, recruiting

Antwerp University Hospital

Immunology - Allergology - Rheumatology, Drie Eikenstraat 655, 2650, Edegem

Italy

1 site · Ongoing, recruiting

Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona

Department of Medicine, Division of Allergy and Immunology, University of Salerno, Largo Citta' D'ippocrate 1, 84131, Salerno

Netherlands

2 sites · Authorised, recruiting

Universitair Medisch Centrum Groningen

Department of Internal Medicine, Hanzeplein 1, 9713 GZ, Groningen

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Department of Internal Medicine, Dr. Molewaterplein 40, 3015 GD, Rotterdam

Norway