Aggressive Systemic Mastocytosis (ASM)NCT02380222
Patient-Reported Outcome Questionnaire for Systemic Mastocytosis
An observational study of Aggressive Systemic Mastocytosis (ASM) and SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), run by Adelphi Values LLC at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), Mast Cell Leukemia (MCL)
- What is being tested?
- Interview
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2014to Dec 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Residents of the United States with advanced systemic mastocytosis who volunteer to participate in the study
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Patient-Reported Outcome Questionnaire Development for Symptoms of Systemic Mastocytosis
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.
- Conditions
- Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), Mast Cell Leukemia (MCL), Smoldering Systemic Mastocytosis (SSM), Indolent Systemic Mastocytosis (ISM) ISM Subgroup Fully Recruited
- Being tested
- Interview
- Keywords
- Systemic mastocytosis, Patient-reported outcome (PRO)
- Medical subject headings
- Mastocytosis, Systemic, Leukemia, Mast-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InterviewOther
Single interview lasting approximately one hour
| Study group | Role | What they receive |
|---|---|---|
| ASM | Not described Receives: Other: Interview | |
| SM-AHNMD | Not described Receives: Other: Interview | |
| MCL | Not described Receives: Other: Interview | |
| SSM | Not described Receives: Other: Interview | |
| ISM | Not described Receives: Other: Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Collection of qualitative interview data on the symptoms of advanced systemic mastocytosis
Measured: Once
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2015 (estimated)
- Study startJul 2014
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aggressive Systemic Mastocytosis (ASM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Adelphi Values LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Blueprint Medicines Corporation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02380222
- Sponsor's protocol ID
- BL7064A
- Status last verified
- Jan 2017
- First submitted
- 26 Feb 2015
- Last updated
- 10 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient-Reported Outcome Questionnaire Development for Symptoms of Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT02380222
- Acronym / sponsor ID
- BL7064A
- Conditions
- Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), Mast Cell Leukemia (MCL), Smoldering Systemic Mastocytosis (SSM), Indolent Systemic Mastocytosis (ISM) ISM Subgroup Fully Recruited
- Keywords
- Systemic mastocytosis, Patient-reported outcome (PRO)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Adelphi Values LLCIndustry (pharma, biotech, devices)
- Collaborators
- Blueprint Medicines Corporation
- Study start
- Jul 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 5 Mar 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| ASM | – | Other: Interview |
| SM-AHNMD | – | Other: Interview |
| MCL | – | Other: Interview |
| SSM | – | Other: Interview |
| ISM | – | Other: Interview |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Residents of the United States with advanced systemic mastocytosis who volunteer to participate in the study
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InterviewSingle interview lasting approximately one hour | Other | ASM, ISM, MCL, SM-AHNMD, SSM |
Primary outcome
Collection of qualitative interview data on the symptoms of advanced systemic mastocytosis
Time frame: Once
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Aggressive Systemic Mastocytosis (ASM) and SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), run by Adelphi Values LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Collection of qualitative interview data on the symptoms of advanced systemic mastocytosis (time frame: Once). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2017.