Clinical trial NCT02620254: Mast Cell Connect: A Registry for Patients With Mastocytosis
Official title: Mast Cell Connect: A Registry for Patients With Mastocytosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mastocytosis
Keywords: mastocytosis
cutaneous mastocytosis
urticaria pigmentosa
telangiectasia macularis eruptiva perstans (TMEP)
diffuse cutaneous mastocytosis
solitary cutaneous mastocytosis
systemic mastocytosis
indolent systemic mastocytosis
smoldering systemic mastocytosis
aggressive systemic mastocytosis
systemic mastocytosis with associated clonal hematologic non-mast cell lineage disease
mast cell leukemia
Lead sponsor: Blueprint Medicines Corporation
Sponsor class: INDUSTRY
Collaborators: name: PatientCrossroads
First posted: 2015-12-02
Enrollment: 743
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a diagnosis of mastocytosis, including systemic mastocytosis and cutaneous mastocytosis and any subtypes of these diseases.

Brief summary: The Mast Cell Connect Registry is a voluntary, observational database that will capture demographic, socioeconomic, and disease information directly from patients with mastocytosis via a secure web-based tool. No experimental intervention is involved.

Detailed description: Mastocytosis is an extremely rare and heterogeneous spectrum of diseases characterized by the buildup of genetically altered mast cells. Patients experience a wide range of symptoms and in some cases, mast cell buildup can lead to organ dysfunction and failure. Current treatments address disease symptoms and not the underlying cause. To facilitate the development of new therapies for mastocytosis, it is important for the community to support clinical trials and to document the impact of the disease, including disease natural history and the impact on patients, in a systematic way. Mast Cell Connect is a web-based registry that allows mastocytosis patients and caregivers to enter information about the experience of the patient living with mastocytosis directly into an online data collection tool.

The Mast Cell Connect Registry allows mastocytosis patients and caregivers to enter information about the experience of the patient living with mastocytosis directly into a web-based data collection tool. Two forms of data will be collected: responses to surveys administered on the web-based portal, and de-identified data curated from medical reports uploaded by patients or their caregivers.

Eligibility criteria: Inclusion Criteria:

* Any patient with a diagnosis of mastocytosis, including systemic mastocytosis and cutaneous mastocytosis and any subtypes of these diseases, who is willing and able to provide written online informed consent

Exclusion Criteria:

* None

Primary outcomes:
measure: Further the understanding of the epidemiology of mastocytosis and its subtypes
timeFrame: Through completion of the study
Secondary outcomes:
Other outcomes:
measure: Improve the collective understanding of the natural history of mastocytosis and its impact on patients
timeFrame: Through completion of the study
measure: Assist in the development of mastocytosis therapy by increasing participation in clinical trials and other research studies for patients with mastocytosis
timeFrame: Through completion of the study
Locations:
city: Cambridge
country: United States
facility: Registry participation is worldwide and not limited to this facility
state: Massachusetts
Study officials:
affiliation: Blueprint Medicines Corporation
name: Anthony L Boral, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: