Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

NCT04469894 | Completed

• 1 other identifier: NNPDF-BUR001
• Study Type: observational
• Target: 76
• Locations: 1 country
• Sites: 1

Brief 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. 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

Conditions

Niemann-Pick Diseases

Outcome Measures

Primary Outcomes (2)

• 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

  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

  July 2020- September

Study Arms (7)

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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

You may qualify if:

• 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)

You may not qualify if:

• \- Inability to meet any of the above 6 criteria

Sponsors & Collaborators

• National Niemann-Pick Disease Foundation: lead
• Engage Health Inc.: collaborator
• Icahn School of Medicine at Mount Sinai: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Niemann-Pick Diseases

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• George A Diaz, MD, PhD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Justin Hopkin, MD

  National Niemann-Pick Disease Board Chair

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 10, 2020
• First Posted: July 14, 2020
• Study Start: June 15, 2020
• Primary Completion: September 21, 2020
• Study Completion: September 21, 2020
• Last Updated: October 6, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, ANALYTIC CODE
• Time Frame: September 2020

Locations

US (1)