Ignite Creation Date:
2025-12-24 @ 11:42 PM
Ignite Modification Date:
2026-01-01 @ 1:35 PM
Study NCT ID:
NCT05033951
Status:
RECRUITING
Last Update Posted:
2024-05-16
First Post:
2021-07-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Optimizing INITIation of Non-invasive Ventilation in ALS Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D059039', 'term': 'Standard of Care'}], 'ancestors': [{'id': 'D019984', 'term': 'Quality Indicators, Health Care'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D006298', 'term': 'Health Services Administration'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 250}, 'targetDuration': '42 Months', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-08-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-05', 'completionDateStruct': {'date': '2025-10-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-05-14', 'studyFirstSubmitDate': '2021-07-07', 'studyFirstSubmitQcDate': '2021-08-27', 'lastUpdatePostDateStruct': {'date': '2024-05-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-09-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-04-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Health related Quality of Life in patients with ALS', 'timeFrame': '15 months', 'description': 'Assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) Min score: 0 Max score: 160 Higher scores mean a worse outcome'}], 'secondaryOutcomes': [{'measure': 'Survival', 'timeFrame': '15 months', 'description': 'Determine the effect of NIV on survival in patients with different ALS phenotypes.'}, {'measure': 'Overall function', 'timeFrame': '15 months', 'description': 'Overall function decline based on the ALS Functional Rating Scale Revised (ALS-FRS-R).\n\nMin score: 0 Max score: 48 Higher scores mean a worse outcome'}, {'measure': 'Forced vital capacity', 'timeFrame': '15 months', 'description': 'Pulmonary function based on FVC Min score: 0% Max score: 100% Lower scores mean a worse outcome'}, {'measure': 'Carbondioxide', 'timeFrame': '15 months', 'description': 'Pulmonary function based on carbondioxide pCO2 in capillary bloodgas Min score: 4 kPa Max score: 9 kPa Higher score means a worse outcome'}, {'measure': 'Anamnestic questions', 'timeFrame': '15 months', 'description': 'Pulmonary function based on anamnestic questions, Borg scale Min score: 0 Max score: 40 Higher score means a worse outcome'}, {'measure': 'Bulbar function', 'timeFrame': '15 months', 'description': 'Bulbar function based on questionnaire Center for Neurological Study Bulbar Functional Scale (CNS BFS).\n\nMin score: 21 Max score: 105 Higher scores means a worse outcome'}, {'measure': 'Weight', 'timeFrame': '15 months', 'description': 'Weight in kg Min score: 40 Max score 130 Lower scores mean a worse outcome'}, {'measure': 'General health Quality of Life in patients with severe respiratory insufficiency', 'timeFrame': '15 months', 'description': 'Assessed by severe respiratory insufficiency questionnaire (SRI) Min score: 49 Max score: 245 Higher scores mean worse outcome'}, {'measure': 'General health Quality of Life', 'timeFrame': '15 months', 'description': 'Assessed by in Short Form Health Survey (SF-36) Min score: 45 Max score: 198 Higher score means a worse outcome'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Noninvasive ventilation', 'Quality of life'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'Objective: The primary objective in this study is to identify which (pheno)type of ALS patient has the most benefit from NIV in improving quality of life.\n\nStudy population: Adult patients with ALS, PLS (Primary Lateral Sclerosis) of PSMA (Progressive Spinal Muscular Atrophy) in the Netherlands. Patients will be included during their first visit to one of the HMV centres in the Netherlands.\n\nMain study parameters/endpoints: The main study parameter is change in Quality of Life (QoL) defined as change in ALS Assessment Questionnaire (ALSAQ-40) in patients with ALS after initiation of NIV.\n\nDesign: Multi-centre prospective cohort study consisting of 2 non-randomized cohorts, i.e. ALS patients who start NIV and ALS patients who do not start NIV at the time of inclusion in the present study.\n\nDuration: The total duration of the study: 3,5 years. Inclusion period: 30 months. Follow up time after initiation of NIV: 9 months. Thereafter, 6 months will be used for data analysis.\n\nSetting: Involvement of all (4) HMV centres in the Netherlands (Groningen, Maastricht, Rotterdam, Utrecht).\n\nProcedure: 250 ALS patients will be included. During the first regular visit to the HMV centre patients will be asked to participate in the study. Data will be recorded during regular visits to the HMV centre.', 'detailedDescription': 'First visit:\n\nAfter informed consent the following data which are gathered as parts of usual care will be recorded in the case report form (CRF):\n\n* Pulmonary function:\n\n * FVC ( % predicted) upright and supine\n * capillary blood gas analysis, room air (pH, pCO2, HCO3-, pO2)\n * anamnestic questions (Borg scale 0-4)\n* Gender\n* Age\n* Percutaneous Endoscopic Gastrostomy (PEG)/ Percutaneous Radiological Gastrostomy (PRG) tube: yes/no\n* Medication\n* Weight/ BMI\n* Living situation\n* Civil status\n* The following clinical characteristics from their medical record (from neurologist or rehabilitation specialist):\n\n * Date of diagnosis\n * Type of ALS at onset\n * ALSFRS-R at the time of diagnosis\n * Pulmonary function at the time of diagnosis\n * Cognitive status (ECAS, ALS-FTD-Q)\n * Medical history\n* Validated questionnaires (online):\n\n * Quality of life questionnaires (SF 36, ALSAQ-40)\n * Respiratory insufficiency questionnaire (SRI)\n * ALS FRS R (patient version)\n * Bulbar symptoms questionnaire (CNS BFS)\n\nAfter the first visit patients will participate in one of the two cohorts (non-randomized):\n\n* Cohort 1: patients who start NIV in the first two months after the first visit to the HMV\n* Cohort 2: patients who do not start NIV in the first two months after the first visit to the HMV.\n\nCohort 1:\n\nData will be recorded at the moment of initiation of NIV and 3, 6 and 9 months after initiation. Data will be recorded during regular visits (to the HMV centre or at the patients home).\n\n* Pulmonary function:\n\n * capillary blood gas, room air (pH, pCO2 (kPa/ mmHg), HCO3- (mmol/l), SaO2 (%))\n * anamnestic questions (Borg scale 0-4)\n * Nocturnal gas exchange by transcutaneous measurement, at the patients home - Mean tcpCO2 (kPa), Maximal tcpCO2 (kPa), mean oxygen saturation (%), lowest oxygen saturation (%)\n* Data from NIV equipment (compliance, modus, IPPA, EPAP, freq, mask type)\n* Use of NIV\n\n * hours a day/night\n * In case of no use: why not?\n* PEG/ PRG tube: yes/no\n* Survival\n* Tracheostomy or not\n* Weight\n* Validated online questionnaires:\n\n * Quality of life questionnaires (SF 36, ALSAQ-40)\n * Respiratory insufficiency questionnaire (SRI)\n * ALS FRS R (patient version)\n * Bulbar symptoms questionnaire (CNS BFS)\n* Nocturnal gas exchange, transcutaneous, at the patients home\n\nCohort 2:\n\nData will be recorded every 3 months until initiation of NIV or death or max 1,5 years after the first visit to the HMV.\n\nData will be recorded during regular visits (to the HMV centre or at the patients home).\n\n* Pulmonary function:\n\n * Capillary blood gas (pH, pCO2 (kPa/ mmHg), HCO3- (mmol/l), SaO2 (%))\n * anamnestic questions (Borg scale 0-4)\n * Nocturnal gas exchange by transcutaneous measurement, at the patients home - tcpCO2 (kPa), Maximal tcpCO2 (kPa), oxygen saturation (%), lowest oxygen saturation (%)\n* PEG/ PRG tube: yes/no\n* Survival\n* Tracheostomy or not\n* Weight\n* Cognitive status (from medical record)\n* Reason for not starting NIV\n* Validated online questionnaires:\n\n * Quality of life questionnaires (SF 36, ALSAQ-40)\n * Respiratory insufficiency questionnaire (SRI)\n * ALS FRS R (patient version)\n * Bulbar symptoms questionnaire (CNS BFS)\n* Nocturnal gas exchange, transcutaneous, at the patients home\n\nIf a patient in cohort 2 will start with NIV, the patient will be transferred to cohort 1.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients must be diagnosed with Amyotrophic Lateral Sclerosis (ALS), Progressive Spinal Muscular Atrophy (PSMA) or Primary Lateral Sclerosis (PLS) by a neurologist.\n\nThe patients will be included (after signing informed consent) during the first visit to one of the HMV centres in the Netherlands. Patients must to be able to participate and at least 18 years old', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age \\> 18 years\n* Diagnosis of ALS, PLS or PSMA\n* Ability to give informed consent.\n* Ability to fill in the questionnaires independently or with assistance of a caregiver.\n\nExclusion Criteria:\n\n* Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.\n* Pregnancy'}, 'identificationModule': {'nctId': 'NCT05033951', 'acronym': 'INITIALS', 'briefTitle': 'Optimizing INITIation of Non-invasive Ventilation in ALS Patients', 'organization': {'class': 'OTHER', 'fullName': 'University Medical Center Groningen'}, 'officialTitle': 'Optimizing INITIation of Non-invasive Ventilation in ALS Patients', 'orgStudyIdInfo': {'id': '202000124'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Cohort 1', 'description': 'Patients who start NIV in the first two months after the first visit to the HMV', 'interventionNames': ['Other: Standard care']}, {'label': 'Cohort 2', 'description': 'Patients who do not start NIV in the first two months after the first visit to the HMV.', 'interventionNames': ['Other: Standard care']}], 'interventions': [{'name': 'Standard care', 'type': 'OTHER', 'description': 'Both groups: standard care+ questionnaires', 'armGroupLabels': ['Cohort 1', 'Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '9700RB', 'city': 'Groningen', 'status': 'RECRUITING', 'country': 'Netherlands', 'contacts': [{'name': 'Peter wijkstra, MD PhD', 'role': 'CONTACT'}], 'facility': 'University medical centre Groningen', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}], 'centralContacts': [{'name': 'Rineke Jaspers Focks, MD', 'role': 'CONTACT', 'email': 'r.jaspersfocks@roessingh.nl', 'phone': '+31534875424'}, {'name': 'Peter Wijkstra, MD, PhD', 'role': 'CONTACT', 'email': 'p.j.wijkstra@umcg.nl', 'phone': '+31503613200'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Medical Center Groningen', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Prof. Dr. P.J. Wijkstra', 'investigatorFullName': 'Peter Wijkstra', 'investigatorAffiliation': 'University Medical Center Groningen'}}}}