Clinical trial • Phase IV • Oncology
Avapritinib for Systemic mastocytosis
Phase IV trial of Avapritinib for Systemic mastocytosis. open-label. 34 participants.
Overview
- Trial Therapeutic Area
- Oncology
- Trial Disease
- Systemic mastocytosis
- Trial Stage
- Phase IV
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 07-06-2024
- First CTIS Authorization Date
- 08-10-2024
Trial design
open-label Phase IV trial across 5 sites in Italy, Belgium, Norway and others.
- Open Label
- Yes
- Target Sample Size
- 34
Eligibility
Recruits 34 paediatric patients.
- Pregnancy Exclusion
- Patient is pregnant and/or breastfeeding.
- Vulnerable Population
- For pediatric patients, the informed consent may be obtained from the patient’s parent or legal guardian. If consent cannot be expressed in writing, then the consent must be formally documented and witnessed, ideally via an independent trusted witness. Parent(legal guardian consent will be determinde by local, regional and/or national guidelines.
Inclusion criteria
- {"criterion_text":"- 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.\n- Completed the parent study treatment requirements and demonstrated compliance with the parent study requirements, as assessed by the Principal Investigator.\n- Continue to clinically benefit from treatment with avapritinib, as determined by the Principal Investigator from the parent study.\n- 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.\n- Agree to continue to use highly effective contraception as defined in this protocol.\n- 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
- {"criterion_text":"- Patient is participating in another interventional study.\n- Patient is unwilling or unable to comply with study procedures and study restrictions.\n- Patient is pregnant and/or breastfeeding."}
Endpoints
Primary endpoints
- {"endpoint_text":"- no primary end points","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 34
- Recruitment Window Months
- 38
- Consent Approach
- 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. Country-specific subject information and informed consent forms are provided (examples in Italian, Dutch, French, Norwegian, English as per ICF documents listed).
Geography
- Total Number Of Sites
- 5
- Total Number Of Participants
- 26
Italy
- Earliest CTIS Part Ii Submission Date
- 29-08-2024
- Latest Decision Or Authorization Date
- 01-04-2026
- Processing Time Days
- 580
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona
- Department Name
- Department of Medicine, Division of Allergy and Immunology, University of Salerno
- Contact Person Name
- Massimo Triggiani
- Contact Person Email
- mtriggiani@unisa.it
Belgium
- Earliest CTIS Part Ii Submission Date
- 02-09-2024
- Latest Decision Or Authorization Date
- 31-03-2026
- Processing Time Days
- 575
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Antwerp University Hospital
- Department Name
- Immunology - Allergology - Rheumatology
- Contact Person Name
- Vito Sabato
- Contact Person Email
- vito.sabato@uza.be
Norway
- Earliest CTIS Part Ii Submission Date
- 19-09-2024
- Latest Decision Or Authorization Date
- 31-03-2026
- Processing Time Days
- 558
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Oslo University Hospital HF
- Department Name
- Department of Hematology
- Contact Person Name
- Ingunn Dybedal
- Contact Person Email
- idybedal@ous-hf.no
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-09-2024
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 573
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Department of Internal Medicine
- Contact Person Name
- Hanneke Oude Elberink
- Contact Person Email
- j.n.g.oude.elberink@umcg.nl
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Department of Internal Medicine
- Contact Person Name
- Paulus Van Daele
- Contact Person Email
- p.l.a.vandaele@erasmusmc.nl
Sponsor
Primary sponsor
- Full Name
- Blueprint Medicines Corp.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Endpoint Clinical Inc.
- Responsibilities
- sponsorDuties codes: 3
- Name
- PPD Development LP
- Responsibilities
- sponsorDuties codes: 13; 15: Pharmacovigilance case processing, medical review, reporting and submission (PV)
- Name
- Syneos Health Netherlands B.V.
- Responsibilities
- sponsorDuties codes: 1, 12, 2, 5, 6, 7
Third parties
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 13; 15: Pharmacovigilance case processing, medical review, reporting and submission (PV)","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Syneos Health Netherlands B.V.","duties_or_roles":"sponsorDuties codes: 1, 12, 2, 5, 6, 7","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Avapritinib
- Active Substance
- Avapritinib
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Authorised (MIA number 20377)
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