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Mast Cell Connect: A Registry for Patients With Mastocytosis

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ClinicalTrials.gov Identifier: NCT02620254
Recruitment Status : Recruiting
First Posted : December 2, 2015
Last Update Posted : September 20, 2017
Sponsor:
Collaborator:
PatientCrossroads
Information provided by (Responsible Party):
Blueprint Medicines Corporation

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.

Condition or disease
Mastocytosis

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.


Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Mast Cell Connect: A Registry for Patients With Mastocytosis
Study Start Date : November 2015
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020





Primary Outcome Measures :
  1. Further the understanding of the epidemiology of mastocytosis and its subtypes [ Time Frame: Through completion of the study ]

Other Outcome Measures:
  1. Improve the collective understanding of the natural history of mastocytosis and its impact on patients [ Time Frame: Through completion of the study ]
  2. Assist in the development of mastocytosis therapy by increasing participation in clinical trials and other research studies for patients with mastocytosis [ Time Frame: Through completion of the study ]


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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:


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02620254


Contacts
Contact: Blueprint Medicines 617-714-6678 mastcellregistry@blueprintmedicines.com

Locations
United States, Massachusetts
Registry participation is worldwide and not limited to this facility Recruiting
Cambridge, Massachusetts, United States
Sponsors and Collaborators
Blueprint Medicines Corporation
PatientCrossroads
Investigators
Principal Investigator: Anthony L Boral, MD, PhD Blueprint Medicines Corporation

Additional Information:
Responsible Party: Blueprint Medicines Corporation
ClinicalTrials.gov Identifier: NCT02620254     History of Changes
Other Study ID Numbers: Mastocytosis Registry
First Posted: December 2, 2015    Key Record Dates
Last Update Posted: September 20, 2017
Last Verified: September 2017

Keywords provided by Blueprint Medicines Corporation:
mastocytosis
cutaneous mastocytosis
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
urticaria pigmentosa
telangiectasia macularis eruptiva perstans (TMEP)
mast cell leukemia

Additional relevant MeSH terms:
Mastocytosis
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Skin Diseases