## Protocol Section
### Identification Module
**NCT ID:** NCT06426030

**Brief Title:** Validation of Czech Language Versions of Questionnaires for ALS Patients' Functional Status and Biomarker Long-term Follow-up

**Official Title:** Validation of Czech Language Versions of Questionnaires Most Frequently Used for Functional Status Monitoring in Patients With Amyotrophic Lateral Sclerosis and the Long-term Follow-up of Biomarkers of the Disease in These Patients.

#### Organization Study ID Info
**ID:** VALID-ALS-QUEST-CZ
#### Organization
**Class:** OTHER
**Full Name:** Masaryk University
### Status Module
#### Completion Date
**Date:** 2024-09-30

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-23

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-17

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2024-09-30

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-05-17

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-23
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-17
**Study First Submit QC Date:** 2024-05-17
### Sponsor Collaborators Module

#### Collaborators

**Class:** OTHER

**Name:** Brno University Hospital

**Class:** OTHER

**Name:** University Hospital, Motol

**Class:** OTHER

**Name:** University Hospital, Martin

**Class:** OTHER

**Name:** University Hospital Bratislava

#### Lead Sponsor

**Class:** OTHER

**Name:** Masaryk University

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** Questionnaires and scales used to assess the clinical status and quality of life of patients with amyotrophic lateral sclerosis (ALS) are an important tool to monitor the disease progression and current needs of patients. The use of these tools (and in particular their combination) allows to cover the whole spectrum of potential patient difficulties and thus significantly facilitates the process of individualisation and optimisation of care. The aim of the study was to create and validate the Czech language versions of the following questionnaires or scales: (1) ALSFRS-R (ALS Functional Rating Scale - Revised Version) and (2) ALSFRS-EX (EXtended, i.e. extended, version of the same scale), both in the self-assessment version (incl. (3) the ALSAQ-40 (ALS Assessment Questionnaire including 40 questions), (4) the DYALS (Dysphagia in ALS), and (5) the Borg Dyspnoea Rating Scale.

All questionnaires were translated using the forward-backward translation method. The scales and questionnaires were administered to ALS patients repeatedly at one-week intervals, first in writing during routine patient follow-up at the Neuromuscular Centre of the University Hospital Brno, and during repeated administrations by telephone.

**Detailed Description:** The first step of the study (before patient recruitment began) was the linguistic validation of all the scales and questionnaires used. Initially, Czech language versions were created using the forward-backward translation method. These created versions were then discussed by an expert panel consisting of 2 amyotrophic lateral sclerosis experts and 2 translators (one native speaker bilingual for both Czech and English, one professional translator specialized in medical English).

Patients potentially meeting the entry criteria will be informed about the purpose and conduct of the study and will sign an informed consent if they agree to participate in the study.

The first administration of all questionnaires (including the self-assessment version of the ALSFRS-R, ALSFRS-Ex, ALSAQ-40, DYALS and Borg Scale for lying, standing and moving positions) will be performed as part of the patient's routine clinical follow-up at the neuromuscular centre. The other two administrations of the questionnaires will be done by telephone one and two weeks after inclusion, respectively. The one-week interval was chosen in line with the approach of similar foreign validation studies of other languages, so that there is a high probability of no significant change in the patient's clinical condition between repeated administrations and, on the other hand, that the patient no longer remembers in detail the answers from the previous administration. At the first telephone readministration (after one week), patients will complete the full range of questionnaires as they did at the first administration of the questionnaires at the centre. The second telephone readministration (after an additional week) will involve completion of the ALSFRS-R and -EX questionnaires only and will use the standard version of both questionnaires administered by an assessor certified to use this scale.

For patients with significantly limited verbal communication skills, it is acceptable to have the patient's caregiver mediate the responses during the telephone administration and/or to send the completed questionnaires during the follow-up administrations by mail or electronically (e-mail). In these cases, patients will be invited to complete the readministration questionnaires by email or telephone (depending on their preference) at a time that would be consistent with normal telephone readministrations for other patients (to maintain an identical time interval between readministrations). Similarly, for patients with limited ability to grasp writing instruments, it will be permissible to complete questionnaires in collaboration with the caregiver, but always on the basis of patient-reported data.

At the first administration of all questionnaires (including the self-assessment version of the ALSFRS-R questionnaire) at the centre, the patient will be asked to complete the questionnaires without further clarification from the investigator. Similarly, during the first telephone readministration involving only the self-assessment questionnaires, the individual questions and the options included in them will be read to the patient only, with no opportunity for the patient to ask additional questions, in order to maintain the self-assessment nature of all tests used.

The standard (non-self-assessment) version of the ALSFRS-R questionnaire will be administered at the second telephone follow-up by TRICALS (Treatment Research Initiative to Cure ALS, an organisation authorised to use the questionnaire in a certified manner) certified assessors and will be administered in accordance with the training provided by this initiative. Initial and repeat administrations will be conducted by different raters. Evaluators conducting telephone readministrations will not be aware of patients' initial test results at the first administration or their clinical status.

The procedures for working with human subjects were approved by the Ethics Committee of the Brno University Hospital on 11.05.2023, reference number 06-110522/EK, project number 85/22.

Statistical data processing will be performed using SPSS 29 statistical software (IBM Corporation, 2020, Armonk, New York, USA).

### Conditions Module

**Conditions:**

- Amyotrophic Lateral Sclerosis

**Keywords:**

- Amyotrophic lateral sclerosis

- ALS

- ALSFRS-R

- ALSFRS-EX

- ALSAQ-40

- Borg scale

- Frontal assessment battery

- Scale

- Questionnaire

### Design Module

#### Design Info

**Observational Model:** COHORT

**Time Perspective:** PROSPECTIVE

#### Enrollment Info

**Count:** 100

**Type:** ESTIMATED

**Study Type:** OBSERVATIONAL

### Outcomes Module

#### Primary Outcomes

**Description:** Content Validity: Ensures the questionnaire comprehensively covers the construct it intends to measure.

**Measure:** Questionnaire validity (ALSFRS-R(-EX), ALSAQ-40, DYALS)

**Time Frame:** 01.09.2024

**Description:** Test-Retest Reliability:

Intraclass Correlation Coefficient (ICC) Pearson or Spearman Correlation Coefficient Cohen's Kappa (κ) Bland-Altman Plot

**Measure:** Questionnaire reproducibility (Czech versions of ALSFRS-R(-EX), ALSAQ-40, DYALS)

**Time Frame:** 01.09.2024

**Description:** Test-Retest Reliability:

Intraclass Correlation Coefficient (ICC) Pearson or Spearman Correlation Coefficient Cohen's Kappa (κ) Bland-Altman Plot

**Measure:** Agreement between self reported and non-self reported version of ALSFRS-R

**Time Frame:** 01.09.2024

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. diagnosed amyotrophic lateral sclerosis (ALS) meeting the EMG (electromyographic) criteria:

   * Gold Coast criteria or
   * at least clinically probable ALS according to Awaji-Shima criteria
2. willing and able to comply with all protocol procedures

Exclusion Criteria:

* none

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

**Study Population:** ALS patients in colaborating Centres - University Hospital Brno (CZ), Motol University Hospital (CZ), Martin University Hospital (SK), University Hospital Bratislava (SK) - who are willing to participate in study.

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** betik.adam@fnbrno.cz

**Name:** Adam Betik, MD

**Phone:** +420532232503

**Role:** CONTACT

**Contact 2:**

**Email:** vlckova.eva@fnbrno.cz

**Name:** Eva Vlckova, doc,MD,PhD

**Phone:** +420532233221

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Brno

**Contacts:**

***Contact 1:***

- **Email:** betik.adam@fnbrno.cz

- **Name:** Adam Betik, MD

- **Phone:** +420532232503

- **Role:** CONTACT

***Contact 2:***

- **Email:** vlckova.eva@fnbrno.cz

- **Name:** Eva Vlckova, doc,MD,PhD

- **Phone:** +420532233221

- **Role:** CONTACT

**Country:** Czechia

**Facility:** University Hospital Brno

**State:** Czech Republic

**Status:** RECRUITING

**Zip:** 62500

#### Overall Officials

**Official 1:**

**Affiliation:** Masaryk University, University Hospital Brno.

**Name:** Eva Vlckova, doc,MD,PhD

**Role:** STUDY_CHAIR

### IPD Sharing Statement Module

**IPD Sharing:** UNDECIDED

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000010335

- Term: Pathologic Processes

- ID: D000019636

- Term: Neurodegenerative Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000013118

- Term: Spinal Cord Diseases

- ID: D000002493

- Term: Central Nervous System Diseases

- ID: D000057177

- Term: TDP-43 Proteinopathies

- ID: D000057165

- Term: Proteostasis Deficiencies

- ID: D000008659

- Term: Metabolic Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M15415

- Name: Sclerosis

- Relevance: HIGH

- As Found: Sclerosis

- ID: M18879

- Name: Motor Neuron Disease

- Relevance: HIGH

- As Found: Lateral Sclerosis

- ID: M4024

- Name: Amyotrophic Lateral Sclerosis

- Relevance: HIGH

- As Found: Amyotrophic Lateral Sclerosis

- ID: M21558

- Name: Neurodegenerative Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M15915

- Name: Spinal Cord Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5742

- Name: Central Nervous System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M28759

- Name: TDP-43 Proteinopathies

- Relevance: LOW

- As Found: Unknown

- ID: M28747

- Name: Proteostasis Deficiencies

- Relevance: LOW

- As Found: Unknown

- ID: M11639

- Name: Metabolic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T349

- Name: Amyotrophic Lateral Sclerosis

- Relevance: HIGH

- As Found: Amyotrophic Lateral Sclerosis

- ID: T4699

- Name: Primary Lateral Sclerosis

- Relevance: HIGH

- As Found: Lateral Sclerosis

### Condition Browse Module - Meshes

- ID: D000016472

- Term: Motor Neuron Disease

- ID: D000000690

- Term: Amyotrophic Lateral Sclerosis

- ID: D000012598

- Term: Sclerosis

### Misc Info Module

- Version Holder: 2024-05-31
