Online survey study to explore patient and caregiver preferences for different spinal muscular atrophy treatments

ISRCTN	ISRCTN11372270
DOI	https://doi.org/10.1186/ISRCTN11372270
Secondary identifying numbers	ALC 1079

Submission date: 08/01/2021
Registration date: 15/01/2021
Last edited: 18/07/2022

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Musculoskeletal Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Background and study aims
Spinal muscular atrophy (SMA) is a genetic condition that makes the muscles weaker and causes problems with movement. It's a serious condition that gets worse over time, but there are treatments to help manage the symptoms.
This is a non-treatment, online survey study designed to explore patient and caregiver preferences for different treatment attributes for SMA Type II and non-ambulatory Type III. The study will be conducted in five European countries: the Netherlands, Finland, Ireland, Portugal and Belgium.

Who can participate?
Parents/caregivers of patients aged <18 years and adult patients living with SMA type II and non-ambulatory type III.

What does the study involve?
Patient/caregiver participants will be recruited through patient associations in each of the five target countries. Potential participants will be sent a link to the survey via email. Participants will first be asked some screening questions to determine their eligibility. If eligible, participants will be given further information about the study and asked to give their informed consent online before proceeding to the survey.
In the survey, participants will be asked a series of clinical questions about their or their child’s SMA diagnosis and treatment alongside demographic and background questions in relation to the SMA patient (either themselves or their child) and, if applicable, the caregiver themselves (caregiver participants only). This will be followed by a presentation of treatment attributes and a DCE survey to assess their preferences for SMA treatments.
The survey will last approximately 20-30 minutes.

What are the possible benefits and risks of participating?
No treatment will be provided in this study; therefore, there are no known physical risks. However, during or following participation in the study, patient and caregiver participants may be reminded and become more aware of their or their child’s condition. There will be no direct benefit to the participant.
The hope is that study results will lead to a robust assessment of patient and caregiver preferences for SMA treatment attributes.

Where is the study run from?
This is an online study in the Netherlands, Finland, Ireland, Portugal and Belgium.

When is the study starting and how long is it expected to run for?
May 2020 to APril 2021.

Who is funding the study?
F. Hoffmann-La Roche Ltd (USA)

Who is the main contact?
global-roche-genentech-trials@gene.com, reference Study ID ALC 1079

Contact information

Mr Clinical Trials
Public

1 DNA Way
South San Francisco
94080
United States of America

Phone	+1 888-662-6728
Email	global-roche-genentech-trials@gene.com

Study information

Study design	Non-interventional online survey study
Primary study design	Observational
Secondary study design	Cross sectional study
Study setting(s)	Internet/virtual
Study type	Other
Participant information sheet	No participant information sheet available to the public, informed consent form intended for participants only.
Scientific title	Patient and carer preference weights in spinal muscular atrophy: a discrete choice experiment (DCE) survey in five European countries
Study objectives	To explore and quantify the value of, or strength of preference for Risdiplam’s treatment characteristics compared to the treatment characteristics of Spinraza and Zolgensma among parents/caregivers of patients aged <18 and adult patients living with SMA type II and non-ambulatory type III.
Ethics approval(s)	Date of IRB exemption status determination: 19/08/2020. Western Institutional Review Board. This is a non-interventional study that does not utilize drugs, and will not take place at any centres. It is strictly an online survey.
Health condition(s) or problem(s) studied	SMA Type II and non-ambulatory Type III
Intervention	Approximately 70 survey participants will be recruited through participant associations within the five target countries. Potential participants will be contacted by e-mail with information about the study. If interested, participants will complete a brief screener questionnaire to check whether they meet the inclusion criteria. Eligible participants will be provided with an information sheet about the study describing the study content, study procedures and their rights as participants and asked to complete a consent form in order to take part in the survey should they wish to do so. Participants will then proceed to the main survey. The survey consists of two parts: the first part of the survey will collect clinical and demographic details of the patient and, if applicable, demographics of the caregiver (caregiver participants only); the second part of the survey will be a DCE consisting of a series of choices between hypothetical treatments varying in terms of treatment attributes, including treatment benefits, side effects, mode of administration, and level of evidence available for different treatment options. The survey is expected to take approximately 20-30 minutes to complete. Participants will not receive a study remuneration for their participation in the study.
Intervention type	Other
Primary outcome measure	Relative importance of treatment characteristics measured using a DCE survey at a single time point
Secondary outcome measures	There are no secondary outcome measures
Overall study start date	28/05/2020
Completion date	30/04/2021

Eligibility

Participant type(s)	Patient
Age group	Mixed
Sex	Both
Target number of participants	70
Key inclusion criteria	1. A patient self-reported or caregiver proxy-reported diagnosis of SMA Type II: age of onset between 7 and 18 months OR SMA Type III: age of onset 18+ months and 17 years 2. Non-ambulatory, defined as unable to walk more than ten steps without a form of assistance 3. Adult patients aged 18+ OR caregivers of patients aged <18 years 4. Residents of the Netherlands, Finland, Ireland, Portugal or Belgium 5. Willing and able to give their informed consent
Key exclusion criteria	Does not meet inclusion criteria
Date of first enrolment	01/10/2020
Date of final enrolment	31/03/2021

Locations

Countries of recruitment

Belgium
Finland
Ireland
Netherlands
Portugal
United States of America

Study participating centre

Online survey administered by Genentech, Inc.

1 DNA Way
San Francisco
94080
United States of America

Sponsor information

Roche (United States)
Industry

Genentech, Inc.
1 DNA Way
San Francisco
94080
United States of America

Phone	+1 888-662-6728
Email	global-roche-genentech-trials@gene.com
Website	https://www.roche.com/about_roche/roche_worldwide.htm
"ROR"	https://ror.org/011qkaj49

Funders

Funder type

Industry

Genentech
Private sector organisation / For-profit companies (industry)

Alternative name(s): Genentech, Inc., Genentech USA, Inc., Genentech USA
Location: United States of America

Results and Publications

Intention to publish date	30/04/2022
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not expected to be made available
Publication and dissemination plan	Peer-reviewed manuscript and/or conference poster of study results.
IPD sharing plan	The datasets generated during and/or analysed during the current study are not expected to be made available as they are not required to be made available.

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article		13/12/2021	18/07/2022	Yes	No

Editorial Notes

18/07/2022: Publication reference added.
13/01/2021: Trial’s existence confirmed by Acaster Lloyd Consulting Ltd.