Last updated: 02/13/2019 20:00:18
Qualitative Research in Patients with Systemic Amyloidosis: Interviews on Symptoms and Impact of Disease
Clinicaltrials.gov ID
Not applicable
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
Study complete
Study complete
Trial overview
Official title: Qualitative Research in Patients with Systemic Amyloidosis: Interviews on Symptoms and Impact of Disease
Trial description: GlaxoSmithKline (GSK) is currently in Phase II of development of a novel therapy for systemic amyloidosis which directly targets the amyloid deposits themselves through a mechanism directed towards serum amyloid P component (SAP). It is envisaged that the proposed anti-SAP treatment would be used in patients with systemic amyloidosis to improve or at least stabilize key organ function. The goal of this project is to identify an existing tool, or modify/develop a content-valid instrument for exploring the efficacy of the proposed anti-SAP treatment with regard to assessing the symptoms associated with the condition. This is a qualitative study involving one-time, one-on-one, phone interviews with patients diagnosed with Amyloid Light-chain (AL), Transthyretin (TTR), or Amyloid A (AA) systemic amyloidosis. The study will use a combined concept elicitation and cognitive debriefing interview guide. The concept elicitation portion of the interviews aims to understand patient experiences of systemic amyloidosis symptoms, symptom impacts and treatment, so as to refine the conceptual framework of the effects of systemic amyloidosis. The cognitive debriefing questions aim to ensure the content of the selected patient-reported outcome (PRO) instruments is appropriate for the target patient population to be studied in the Phase III clinical trials, as well as evaluate the patients’ ability to understand and complete the PRO measures selected for the Phase III study. Approximately 45 patients with systemic amyloidosis will be interviewed. Of these, approximately 15 patients will have AL subtype of systemic amyloidosis, 15 will have TTR (systemic and wild-type) subtypes, and 15 will have AA subtype.
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:
Determination of concepts important in systemic amyloidosis as described by patients with different levels of severities of illness and organ involvement
Timeframe: Day 1
Determination of fitness for purpose of currently available PRO tools for the measurement of symptoms in patients with different levels of amyloid load, illness severity and organ involvement
Timeframe: Day 1
Development or adaptation of a ‘fit for purpose’ measure of symptoms and impacts of disease and treatment for possible use as a key secondary endpoint in a pivotal trial with systemic amyloidosis patients
Timeframe: Day 1
Secondary outcomes:
Not applicable
Interventions:
Enrollment:
0
Primary completion date:
2018-15-02
Observational study model:
Case-Only
Time perspective:
Cross-Sectional
Clinical publications:
Not applicable
Medical condition
Amyloidosis
Product
dezamizumab, miridesap
Collaborators
None
Study date(s)
June 2016 to February 2018
Type
Observational
Phase
Not applicable
Participation criteria
Sex
Female & Male
Age
18 - 82 Years
Accepts healthy volunteers
Not applicable
- For AL patients: Mayo classification II and III with heart involvement (New York Heart Association [NYHA] stage II or III); Patient currently having chemotherapy, or 6 months post-chemotherapy
- For TTR Cardiomyopathy (CM) patients: Confirmed diagnosis of TTR-related systemic amyloidosis (ATTR) cardiomyopathy, NYHA stage II or III; Adequate written and oral fluency in English
- For AL patients: Post stem cell transplant (autologous stem cell transplant)
- For TTR CM patients: On heart transplant waiting list; Requirement for long term left ventricular assist device (LVAD); Requirement for LVAD as a peri-heart transplant bridge
Inclusion and exclusion criteria
Inclusion criteria:
- For AL patients: Mayo classification II and III with heart involvement (New York Heart Association [NYHA] stage II or III); Patient currently having chemotherapy, or 6 months post-chemotherapy
- For TTR Cardiomyopathy (CM) patients: Confirmed diagnosis of TTR-related systemic amyloidosis (ATTR) cardiomyopathy, NYHA stage II or III; Adequate written and oral fluency in English
- For all patients: Aged 18-82 years old; Clinic-confirmed or self-reported diagnosis of AL, TTR, or AA systemic amyloidosis; Willing and able to participate in a seventy-five minute telephone interview; Willing to have the interview audio-recorded; Willing to provide written informed consent
Exclusion criteria:
- For AL patients: Post stem cell transplant (autologous stem cell transplant)
- For TTR CM patients: On heart transplant waiting list; Requirement for long term left ventricular assist device (LVAD); Requirement for LVAD as a peri-heart transplant bridge
- For all patients: Patient does not meet the inclusion criteria specified above; NYHA stage I or IV; Any serious psychiatric disorder or other condition (e.g. Alzheimer’s disease, dementia, schizophrenia, or other disorders that impair memory or cognitive function) that could interfere with subject providing informed consent, completing questionnaires, or participating in an interview to discuss their health
Trial location(s)
This study does not involve prospective enrollment of participants.
Study documents
Scientific result summary
Available language(s): English
Protocol
Available language(s): English
If you wish to request for full study report, please contact - [email protected]
Results overview
Refer to study documents
Recruitment status
Study complete
Actual primary completion date
2018-15-02
Actual study completion date
2018-15-02
Plain language summaries
Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.
Additional information about the trial
Not applicable
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