Niemann-Pick DiseasesNCT04469894
Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick
An observational study of Niemann-Pick Diseases, run by National Niemann-Pick Disease Foundation at 1 site in United States.
Trial summary
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Niemann-Pick Diseases
- How many people
- 76 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2020to 21 Sep 2020
- 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: Within the United States, patients or the parent/caregiver of patients who have been diagnosed with any of the 7 known subtypes of Niemann-Pick disease
Who may be able to join
- Participant must be a person with Niemann-Pick disease who is 18 years or older or The parent/legal guardian of a person with Niemann-Pick disease. Please note: Parents whose child has passed on are able to participate in the RSVP. Parents whose child has passed on in the last two years are also able to participate in the interview. Please note that only one family member will be eligible to complete the RSVP and interview for one family.
- Confirmed diagnosis of Niemann-Pick disease, confirmed by membership in the NNPDF or by provision of a proof of disease form
- Able to read, write and communicate in English
- Able to grant informed consent
- Willing to complete a survey and RSVP, and to participate in a 30-minute telephone interview
- Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail)
Who cannot join
- Inability to meet any of the above 6 criteria
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: Understanding Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick Disease Through the Eyes of Patients and Families
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.
The core research objectives are to understand the following from Niemann-Pick patients and their families in the US
Read the detailed description
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.
The core research objectives are to understand the following from Niemann-Pick patients and their families in the US;
- Level of health insurance literacy through assessment of health insurance terminology, and self-reported insights regarding information seeking, document literacy and cognitive skills
- If they have healthcare insurance, and if not, why not
- Types (including name) of insurance by which Niemann-Pick families are covered
- Information regarding Medicaid Waivers
- Attributes of insurance plans including, but not limited to;
- Deductible amounts for individual and family
- Out of pocket maximums for individual and family
- Insurance premiums
- Associated Health Savings Accounts
- Associated Cost Sharing requirements
- Services, drugs and assistive devices covered
- What the perceived out-of-pocket costs are for Niemann-Pick families (annually)
- When out-of-pocket maximums are perceived to be met for Niemann-Pick families
- Patient and HCP experience in terms of challenges with accessing desired care and coverage
- Where are families experiencing challenges with accessing desired care and coverage? (drugs, assistive devices, services) Is it different for the different phenotypes of the disease?
- What is the challenge? (financial, time, stress, others)
- What is the life impact of that challenge? (work and educational impact, lack or delay of treatment, others)
- Conditions
- Niemann-Pick Diseases
- Medical subject headings
- Niemann-Pick Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Niemann-Pick Type A | Also referred to as Infantile Neurovisceral ASMD | |
| Niemann-Pick Type A/B | Also referred to as Intermediate form or Chronic Neurovisceral ASMD | |
| Niemann-Pick Type B | Also referred to as Chronic Visceral ASMD | |
| Niemann-Pick Type C (Early Infantile) | Onset at less than 2 years of age | |
| Niemann-Pick Type C (Late Infantile) | Neurodegenerative form (late-infantile) onset at 2-6 years of age | |
| Niemann-Pick Type C (Juvenile) | Neurodegenerative form (juvenile) onset at 6-15 years of age | |
| Niemann-Pick Type C (Adult) | Psychiatric neurodegenerative form (adult) onset at greater than 15 years of age |
What is being measured
The primary outcome is the main question the study is designed to answer.
Health Insurance Literacy
Health insurance literacy will be measured by a score on a brief quiz regarding health insurance key terms as well as self-ratings regarding various aspects of the participants' ability to navigate and understand insurance
Measured: July 2020- September
Health Insurance Coverage
Health insurance coverage will be measured by the different multiple choice and open-ended questions based on the services available under the participants' insurance plan as well as their reported difficulty with navigating their plan and obtaining what they need for Niemann-Pick
Measured: July 2020- September
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2020
- Study start15 Jun 2020
- Primary completion21 Sep 2020
- Study completion21 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Niemann-Pick DiseasesStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Niemann-Pick Disease Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- Engage Health Inc., Icahn School of Medicine at Mount Sinai
- Responsible party
- Sponsor
- Investigators
- George A Diaz, MD, PhD, Principal investigator, Icahn School of Medicine at Mount SinaiJustin Hopkin, MD, Study chair, National Niemann-Pick Disease Board Chair
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04469894
- Sponsor's protocol ID
- NNPDF-BUR001
- Status last verified
- Oct 2020
- First submitted
- 10 Jul 2020
- Last updated
- 6 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data for each question in the study will be analyzed and reported by Engage Health using descriptive statistics as appropriate for the nature of the data collected-frequency distributions, cross tabulations and measures of central tendency and dispersion.
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Understanding Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick Disease Through the Eyes of Patients and Families
- ClinicalTrials.gov ID
- NCT04469894
- Acronym / sponsor ID
- NNPDF-BUR001
- Conditions
- Niemann-Pick Diseases
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- National Niemann-Pick Disease FoundationUniversity, hospital or other
- Collaborators
- Engage Health Inc., Icahn School of Medicine at Mount Sinai
- Study start
- 15 Jun 2020 (actual)
- Primary completion
- 21 Sep 2020 (actual)
- Study completion
- 21 Sep 2020 (actual)
- First posted
- 14 Jul 2020 (actual)
- 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.
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.
The core research objectives are to understand the following from Niemann-Pick patients and their families in the US
Detailed description
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.
The core research objectives are to understand the following from Niemann-Pick patients and their families in the US;
- Level of health insurance literacy through assessment of health insurance terminology, and self-reported insights regarding information seeking, document literacy and cognitive skills
- If they have healthcare insurance, and if not, why not
- Types (including name) of insurance by which Niemann-Pick families are covered
- Information regarding Medicaid Waivers
- Attributes of insurance plans including, but not limited to;
- Deductible amounts for individual and family
- Out of pocket maximums for individual and family
- Insurance premiums
- Associated Health Savings Accounts
- Associated Cost Sharing requirements
- Services, drugs and assistive devices covered
- What the perceived out-of-pocket costs are for Niemann-Pick families (annually)
- When out-of-pocket maximums are perceived to be met for Niemann-Pick families
- Patient and HCP experience in terms of challenges with accessing desired care and coverage
- Where are families experiencing challenges with accessing desired care and coverage? (drugs, assistive devices, services) Is it different for the different phenotypes of the disease?
- What is the challenge? (financial, time, stress, others)
- What is the life impact of that challenge? (work and educational impact, lack or delay of treatment, others)
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Niemann-Pick Type A | – | –Also referred to as Infantile Neurovisceral ASMD |
| Niemann-Pick Type A/B | – | –Also referred to as Intermediate form or Chronic Neurovisceral ASMD |
| Niemann-Pick Type B | – | –Also referred to as Chronic Visceral ASMD |
| Niemann-Pick Type C (Early Infantile) | – | –Onset at less than 2 years of age |
| Niemann-Pick Type C (Late Infantile) | – | –Neurodegenerative form (late-infantile) onset at 2-6 years of age |
| Niemann-Pick Type C (Juvenile) | – | –Neurodegenerative form (juvenile) onset at 6-15 years of age |
| Niemann-Pick Type C (Adult) | – | –Psychiatric neurodegenerative form (adult) onset at greater than 15 years of age |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Within the United States, patients or the parent/caregiver of patients who have been diagnosed with any of the 7 known subtypes of Niemann-Pick disease
Key inclusion criteria (6 listed)
- Participant must be a person with Niemann-Pick disease who is 18 years or older or The parent/legal guardian of a person with Niemann-Pick disease. Please note: Parents whose child has passed on are able to participate in the RSVP. Parents whose child has passed on in the last two years are also able to participate in the interview. Please note that only one family member will be eligible to complete the RSVP and interview for one family.
- Confirmed diagnosis of Niemann-Pick disease, confirmed by membership in the NNPDF or by provision of a proof of disease form
- Able to read, write and communicate in English
- Able to grant informed consent
- Willing to complete a survey and RSVP, and to participate in a 30-minute telephone interview
Key exclusion criteria (1 listed)
- Inability to meet any of the above 6 criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Health Insurance Literacy
Time frame: July 2020- September
Health insurance literacy will be measured by a score on a brief quiz regarding health insurance key terms as well as self-ratings regarding various aspects of the participants' ability to navigate and understand insurance
Health Insurance Coverage
Time frame: July 2020- September
Health insurance coverage will be measured by the different multiple choice and open-ended questions based on the services available under the participants' insurance plan as well as their reported difficulty with navigating their plan and obtaining what they need for Niemann-Pick
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 6 Oct 2020.
- Individual participant dataSharing planned: Yes. The data for each question in the study will be analyzed and reported by Engage Health using descriptive statistics as appropriate for the nature of the data collected-frequency distributions, cross tabulations and measures of central tendency and dispersion.
Related trials for Niemann-Pick Diseases
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RecruitingObservational study
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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.
George A Diaz, MD, PhD principal investigator
Icahn School of Medicine at Mount Sinai
Justin Hopkin, MD study chair
National Niemann-Pick Disease Board Chair
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 Niemann-Pick Diseases, run by National Niemann-Pick Disease Foundation 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 6 inclusion and 1 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 6 Oct 2020. 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?
Health Insurance Literacy (time frame: July 2020- September). The study lists 2 primary outcomes in all. 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 6 Oct 2020.