Aggressive Systemic Mastocytosis (ASM) Clinical Trial
Official title:
Patient-Reported Outcome Questionnaire Development for Symptoms of Systemic Mastocytosis
| Verified date | January 2017 |
| Source | Adelphi Values LLC |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Copernicus Group IRB |
| Study type | Observational |
The purpose of this study is to explore the key symptoms of individuals with systemic
mastocytosis. Interviews of enrolled individuals will be conducted to learn about the
disease symptoms and condition. The interview will last approximately 60 minutes and will be
conducted by a trained interviewer, be audio-recorded (with patient consent), and all
information provided by the patient will be treated confidentially and made anonymous so
that it is non-identifiable. The interview may be conducted face-to-face, over the phone, or
virtually via Skype, a free video conferencing program, depending upon the patient's
geographic location. Patients will be compensated for their participating time.
This is not a medication-related study, and no medication will be distributed or tested
during this study. Participation in this study will not affect any treatment or assistance
that a patient currently receives or may receive in the future.
| Status | Completed |
| Enrollment | 5 |
| Est. completion date | December 2015 |
| Est. primary completion date | December 2015 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - Documented diagnosis of one of the following conditions based upon World Health Organization (WHO) diagnostic criteria: aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated clonal hematologic non-mast cell lineage disease (SM-AHNMD), mast cell leukemia (MCL), smoldering systemic mastocytosis (SSM), indolent systemic mastocytosis (ISM) - Diagnosis confirmed by bone marrow biopsy - Fluency in English - Willingness and ability to participate in a one-hour interview Exclusion Criteria: - A condition or situation that would interfere with participation in an interview (e.g., cognitive impairment or disorder, alcohol or drug abuse) - Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness - Any other prior malignancy except for the following: Adequately treated basal cell or squamous cell skin cancer; In situ cervical cancer; Adequately treated Stage I or II cancer from which the subject is currently in complete remission, or other cancer from which the subject has been disease-free for 2 years |
Observational Model: Case-Only, Time Perspective: Cross-Sectional
| Country | Name | City | State |
|---|---|---|---|
| United States | Adelphi Values LLC | Boston | Massachusetts |
| Lead Sponsor | Collaborator |
|---|---|
| Adelphi Values LLC | Blueprint Medicines Corporation |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Collection of qualitative interview data on the symptoms of advanced systemic mastocytosis | Once | No |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04996875 -
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
|
Phase 2 |