Clinical trial • Phase II • Neurology|Rare Disease

VHB937 for Amyotrophic lateral sclerosis

Phase II trial of VHB937 for Amyotrophic lateral sclerosis.

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Amyotrophic lateral sclerosis
Trial Stage
Phase II
Drug Modality
Monoclonal antibody

Key dates

Initial CTIS Submission Date
11-09-2024
First CTIS Authorization Date
15-01-2025

Trial design

Randomised, open-label, placebo 00 mg/ 00ml, concentrate for solution for infusion (placebo to vhb937); dose/schedule not specified in available documents.-controlled Phase II trial in Finland, Denmark, Spain and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo 00 mg/ 00mL, concentrate for solution for infusion (Placebo to VHB937); dose/schedule not specified in available documents.
Target Sample Size
104
Trial Duration For Participant
700

Eligibility

Recruits 104 Signed informed consent must be obtained prior to participation in the study. Participants with active moderate or severe psychiatric disease, cognitive impairment, neurological disease other than ALS, dementia or substance abuse that would impair ability of the participant to provide informed consent are excluded, in the investigator’s opinion. (Consent/ICF and caregiver/assistance arrangements are provided in subject information and informed consent forms.).

Pregnancy Exclusion
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 24 weeks after stopping study medication.
Vulnerable Population
Signed informed consent must be obtained prior to participation in the study. Participants with active moderate or severe psychiatric disease, cognitive impairment, neurological disease other than ALS, dementia or substance abuse that would impair ability of the participant to provide informed consent are excluded, in the investigator’s opinion. (Consent/ICF and caregiver/assistance arrangements are provided in subject information and informed consent forms.)

Inclusion criteria

  • {"criterion_text":"- Signed informed consent must be obtained prior to participation in the study.\n- Sporadic or familial ALS diagnosed using the revised El Escorial criteria as clinically possible, probable, lab-supported probable, or definite ALS.\n- Age 18 years or older (19 years of age for S. Korea population according to the local adult age standard).\n- Onset of ALS symptoms within 24 months.\n- Slow Vital Capacity (SVC) greater or equal to 60% of predicted capacity for age, height, and sex.\n- ALSFRS-R total score greater or equal to 30\n- Treated or untreated with SoC therapy for ALS. If treated, must be on a stable dose of riluzole, for ≥ 30 days and/or edaravone oral or i.v. having completed at least one on-drug cycle prior to randomization.\n- Able and willing to travel to the site with or without assistance from their support network."}

Exclusion criteria

  • {"criterion_text":"- Use of other investigational drugs within 5 half-lives of screening, or within 30 days (e.g., small molecules) / or until the expected pharmacodynamic effect has returned to baseline (e.g., biologics), whichever is longer; or longer if required by local regulations.\n- Taking any prohibited medications as listed on Table 6-4 of the protocol.\n- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 24 weeks after stopping study medication.\n- History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study.\n- Clinical evidence of liver or renal disease/injury.\n- Laboratory evidence of hematological abnormalities\n- Active moderate or severe psychiatric disease, cognitive impairment, neurological disease other than ALS, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the investigator’s opinion.\n- Participants that reported 'yes' on any suicidal ideation section except for the “Non- Suicidal Self-Injurious Behavior” in the past 2 years as per C-SSRS.\n- Presence of cancer, HIV, uncontrolled diabetes\n- History of active severe respiratory disease."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS) [Time Frame: Baseline to DB Week 40]","definition_or_measurement_approach":"Analysis method: Combined Assessment of Function and Survival (CAFS). Time frame: Baseline to Double-Blind (DB) Week 40; composite of permanent assisted ventilation (PAV)-free survival and change in ALSFRS-R."}

Secondary endpoints

  • {"endpoint_text":"- ALSFRS-R total score [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)]","definition_or_measurement_approach":"ALSFRS-R total score; time frames: Baseline to DB Week 40 (or until death/PAV) and Baseline to OLE Week 100 (or until death/PAV)."}
  • {"endpoint_text":"- Slow Vital Capacity (SVC) (% of predicted normal value) [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)]","definition_or_measurement_approach":"SVC expressed as % of predicted normal value; same time frames as ALSFRS-R."}
  • {"endpoint_text":"- Ratio to baseline in Neurofilament Light (NfL) concentration in serum [Timeframe: DB up to Week 40; DB and OLE up to Week 100]","definition_or_measurement_approach":"Change (ratio to baseline) in serum NfL concentration; timeframes: DB up to Week 40 and DB+OLE up to Week 100."}
  • {"endpoint_text":"- Safety and tolerability parameters including adverse events, laboratory data, vital signs, electrocardiogram (ECG), Columbia Suicide Severity Rating Scale (C-SSRS) [Time Frame: Baseline to DB Week 40, Baseline to OLE Week 100, and Baseline to last scheduled visit or end of study]","definition_or_measurement_approach":"Standard safety assessments: AEs, labs, vitals, ECG, C-SSRS. Time frames: Baseline to DB Week 40; Baseline to OLE Week 100; Baseline to last scheduled visit/end of study."}
  • {"endpoint_text":"- Time to death and Time to event (death or PAV, whichever comes first). [Timeframe: Baseline to DB Week 40]","definition_or_measurement_approach":"Time-to-event analyses for death and combined event (death or PAV); timeframe Baseline to DB Week 40."}
  • {"endpoint_text":"- Time to death and Time to event (death or PAV, whichever comes first) – endpoints referring to treatment policy estimand [Time Frame: Baseline to OLE Week 100, and Baseline to end of study]","definition_or_measurement_approach":"Time-to-event endpoints considered under a treatment-policy estimand; timeframe Baseline to OLE Week 100 and Baseline to end of study."}
  • {"endpoint_text":"- Clinician and Patient Global Impression of change in functional ability and ALS symptom severity (CGI-C and PGI-C) [Time Frame: DB up to Week 40; DB and OLE up to Week 100]","definition_or_measurement_approach":"CGI-C and PGI-C assessments of change in function and symptom severity; timeframes DB up to Week 40 and DB+OLE up to Week 100."}
  • {"endpoint_text":"- Change in QoL from baseline as measured with Amyotrophic Lateral Sclerosis Assessment Questionnaire -5 (ALSAQ-5), EuroQoL 5 Dimension 5 Level (EQ-5D-5L), 12-item Short form health survey (SF-12) [Time Frame: DB up to Week 40; DB and OLE up to Week 100]","definition_or_measurement_approach":"Quality of life measures: ALSAQ-5, EQ-5D-5L, SF-12; timeframes DB up to Week 40 and DB+OLE up to Week 100."}
  • {"endpoint_text":"- PK in serum and CSF and Anti-VHB937 antibodies (ADAs) in serum [Time Frame for serum: DB up to Week 40, DB and OLE up to Week 100; DB and OLE up to last scheduled visit; For CSF: DB Week 12]","definition_or_measurement_approach":"Pharmacokinetics in serum and CSF; anti-VHB937 antibodies in serum. Serum: DB up to Week 40 and DB+OLE up to Week 100 and up to last scheduled visit. CSF PK at DB Week 12."}

Recruitment

Registry Or Advocacy Recruitment
True, TRICALS (document titled "K2_CVHB937B12201_TRICALS Recruitment text" is present).
Planned Sample Size
104
Recruitment Window Months
41
Consent Approach
Signed informed consent must be obtained prior to participation. Subject information and ICF documents (main ICF, pregnancy ICF, optional genetics ICF, participant study guides and other materials) are provided in country- and language-specific versions (examples present: EN, ES, FR, NL, PL, IT, DE, SE, FI). ICFs and supporting documents are available per participating country and are required before enrollment.

Methods

  • Doctor-to-Participant Letter – HCP-to-patient referral material (country-specific versions listed in documents).
  • Participant Brochure – patient-facing brochure distributed to potential participants (country-specific versions).
  • Clinical Studies Brochure – recruitment brochure for study information (country-specific versions).
  • Physician Referral Letter – materials to inform referring physicians about the study.
  • Patient advertisement / Patient advertisement ASTRALS – patient-facing adverts (country-specific versions).
  • K1 Recruitment arrangements and local recruitment procedure documents (country-specific).

Geography

Total Number Of Sites
43
Total Number Of Participants
121

Finland

Earliest CTIS Part Ii Submission Date
29-11-2024
Latest Decision Or Authorization Date
21-01-2025
Processing Time Days
53
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Turku University Hospital
Department Name
Turku University Hospital, Department of Neurology
Contact Person Name
Manu Jokela
Contact Person Email
manu.jokela@varha.fi

Denmark

Earliest CTIS Part Ii Submission Date
19-12-2024
Latest Decision Or Authorization Date
18-01-2025
Processing Time Days
30
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Aalborg University Hospital
Department Name
Department of Neurology
Contact Person Name
Izabella Obal
Contact Person Email
i.obal@rn.dk
Site Name
Region Hovedstaden
Department Name
Department of Neurology
Contact Person Name
Lisette Salvesen
Contact Person Email
lisette.salvesen@regionh.dk

Spain

Earliest CTIS Part Ii Submission Date
27-12-2024
Latest Decision Or Authorization Date
15-01-2025
Processing Time Days
19
Number Of Sites
8
Number Of Participants
25

Sites

Site Name
Bellvitge University Hospital
Department Name
Neurology Service
Contact Person Name
Monica Povedano Panades
Site Name
Hospital Carlos III
Department Name
Neurology Service
Contact Person Name
Maria Hernandez Barral
Contact Person Email
maryh210@msn.com
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology Service
Contact Person Name
Virginia Reyes Garrido
Contact Person Email
v.reyes.eecc@gmail.com
Site Name
Hospital Universitario Donostia
Department Name
Neurology Service
Contact Person Name
Adolfo Lopez de Munain Arregui
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Neurology Service
Contact Person Name
Beatrice Canneti Heredia
Contact Person Email
canneti.heredia@gmail.com
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology Service
Contact Person Name
Juan Francisco Vazquez Costa
Contact Person Email
juan.vazquez.neuro@gmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology Service
Contact Person Name
Raul Juntas Morales
Contact Person Email
raul.juntas@vallhebron.cat
Site Name
Hospital Del Mar
Department Name
Neurology Service
Contact Person Name
Miguel Angel Rubio Perez
Contact Person Email
marubio@psmar.cat

Sweden

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
20-01-2025
Processing Time Days
116
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Region Stockholm – SLSO
Department Name
Studieenheten Akademiskt Specialistcentrum, ME Neurlogi
Contact Person Name
Caroline Ingre
Contact Person Email
Caroline.ingre@ki.se
Site Name
Region Vaesterbotten
Department Name
Neurologiska klinikken, NHHC och Institutionen for vetenskap, neurovetenskaper
Contact Person Name
Peter Andersen
Contact Person Email
peter.andersen@umu.se
Site Name
University Of Skane
Department Name
Neurologimottagningen, VE Neurologi
Contact Person Name
Christer Nilsson
Contact Person Email
christer.f.nilsson@skane.se

Netherlands

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
16-01-2025
Processing Time Days
112
Number Of Sites
1
Number Of Participants
7

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
Neurology
Contact Person Name
Leonard van den Berg
Contact Person Email
L.H.vandenBerg@umcutrecht.nl

Belgium

Earliest CTIS Part Ii Submission Date
16-12-2024
Latest Decision Or Authorization Date
16-01-2025
Processing Time Days
31
Number Of Sites
2
Number Of Participants
7

Sites

Site Name
UZ Leuven
Department Name
Neurology
Contact Person Name
Philip Van Damme
Contact Person Email
philip.vandamme@uzleuven.be
Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Neurology
Contact Person Name
Stephanie Delstanche

France

Earliest CTIS Part Ii Submission Date
16-12-2024
Latest Decision Or Authorization Date
20-01-2025
Processing Time Days
35
Number Of Sites
7
Number Of Participants
16

Sites

Site Name
Centre Hospitalier Et Universitaire De Limoges
Department Name
Neurologie
Contact Person Name
Philippe Couratier
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Explorations Neurologiques et centre de reference SLA
Contact Person Name
Elisa De La Cruz
Contact Person Email
e-delacruz@chu-montpellier.fr
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Neurologie
Contact Person Name
Philippe Corcia
Contact Person Email
corcia@med.univ-tours.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Centre SLA - Neurologie
Contact Person Name
Veronique Danel-Brunaud
Contact Person Email
Veronique.danel@chu-lille.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neurologie
Contact Person Name
Gaelle Bruneteau
Contact Person Email
gaelle.bruneteau@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Neurologie, CRMR SLA
Contact Person Name
Marie-Helene Soriani
Contact Person Email
soriani.mh@chu-nice.fr
Site Name
Hospices Civils De Lyon
Department Name
Centre SLA Lyon - Neurologie C
Contact Person Name
Emilien Bernard
Contact Person Email
emilien.bernard@chu-lyon.fr

Germany

Earliest CTIS Part Ii Submission Date
16-12-2024
Latest Decision Or Authorization Date
17-01-2025
Processing Time Days
32
Number Of Sites
9
Number Of Participants
10

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Neurologie
Contact Person Name
Andre Maier
Contact Person Email
andre.maier@charite.de
Site Name
Universitaet Muenster
Department Name
Klinik für Neurologie
Contact Person Name
Matthias Pawlowski
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Studienzentrum Neurologie
Contact Person Name
Jochen Weishaupt
Contact Person Email
jochen.weishaupt@rku.de
Site Name
Heidelberg University
Department Name
Neurologische Klinik
Contact Person Name
Julian Conrad
Contact Person Email
julian.conrad@umm.de
Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Neurologische Klinik
Contact Person Name
Julian Grosskreutz
Site Name
DIAKOVERE Krankenhaus gGmbH
Department Name
Klinik für Neurologie und Neurophysiologie
Contact Person Name
Susanne Petri
Contact Person Email
susanne.petri@diakovere.de
Site Name
Rostock University Medical Center
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Andreas Hermann
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Neurologische Klinik und Poliklinik
Contact Person Name
Daniel Zeller
Contact Person Email
Zeller_D@ukw.de
Site Name
Klinikum rechts der Isar der TU Muenchen AöR
Department Name
Klinik für Neurologie
Contact Person Name
Paul Lingor
Contact Person Email
paul.lingor@mri.tum.de

Ireland

Earliest CTIS Part Ii Submission Date
12-12-2024
Latest Decision Or Authorization Date
17-01-2025
Processing Time Days
36
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Beaumont Hospital
Department Name
Neurology
Contact Person Name
Orla Hardiman
Contact Person Email
hardimao@tcd.ie

Italy

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
17-01-2025
Processing Time Days
114
Number Of Sites
4
Number Of Participants
10

Sites

Site Name
Azienda Ospedaliero Universitaria Pisana
Department Name
Neurological Clinic
Contact Person Name
Francesca Bianchi
Contact Person Email
f.bianchi@ao-pisa.toscana.it
Site Name
Istituti Clinici Scientifici Maugeri S.p.A.
Department Name
Neurohabilitation
Contact Person Name
Christian Lunetta
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Neuroscience and mental Healh
Contact Person Name
Adriano Chiò
Contact Person Email
adriano.chio@unito.it
Site Name
Azienda Ospedaliero Universitaria Di Modena
Department Name
Dipartimento di Scienze Biomediche, Metaboliche e Neuroscienze
Contact Person Name
Jessica Mandrioli
Contact Person Email
jmandrio@unimore.it

Poland

Earliest CTIS Part Ii Submission Date
19-12-2024
Latest Decision Or Authorization Date
21-01-2025
Processing Time Days
33
Number Of Sites
5
Number Of Participants
27

Sites

Site Name
Centrum Medyczne Neuromed Sp. z o.o.
Contact Person Name
Paweł Lisewski
Contact Person Email
lisewski.p@gmail.com
Site Name
City Clinic Research Sp. z o.o.
Contact Person Name
Magdalena Kuzma-Kozakiewicz
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Oddział Kliniczny Neurologii
Contact Person Name
Agnieszka Słowik
Contact Person Email
neurologiabk@su.krakow.pl
Site Name
Neuroprotect Sp. z o.o.
Contact Person Name
Mariusz Grudniak
Site Name
Linden Sp. z o.o. sp.k.
Contact Person Name
Jakub Antczak
Contact Person Email
jacob.antczak@gmail.com

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Parexel International (IRL) Limited
Responsibilities
Sponsor duty code: 12 (specific duty description not provided)
Name
IQVIA Limited
Responsibilities
Regulatory Document Collection, IRB/EC Submissions, Site Identification and Selection, Investigator Payments, Local ICF Development, Patient Recruitment, TMF.
Name
WCG Clinical Inc.
Responsibilities
Scale Management - Licensing, Development and Translation Activities, Virgil® Study Configuration
Name
Eresearchtechnology Inc.
Responsibilities
ECG and Spirometry
Name
Labcorp Central Laboratory Services LP
Responsibilities
Laboratory services
Name
Greenphire LLC
Responsibilities
Concierge service and patient reimbursement.

Third parties

  • {"country":"United States","full_name":"Linus Health Inc.","duties_or_roles":"Speech Recording","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Ancillary supplies","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Scale Management - Licensing, Development and Translation Activities, Virgil® Study Configuration","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Sponsor duty code: 12 (specific duty description not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG and Spirometry","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Regulatory Document Collection, IRB/EC Submissions, Site Identification and Selection, Investigator Payments, Local ICF Development, Patient Recruitment, TMF.","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Concierge service and patient reimbursement.","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Pharma Bio-Research Group","duties_or_roles":"Sponsor duty code: 4 (specific duty description not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Myonex LLC","duties_or_roles":"Ancillary supplies","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Sponsor duty code: 4 (laboratory services)","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"SGS France","duties_or_roles":"Sponsor duty code: 4 (specific duty description not provided)","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
VHB937
Active Substance
VHB937
Modality
Monoclonal antibody
Routes Of Administration
Intravenous
Route
INTRAVENOUS
Investigational Product Name
Placebo 00 mg/ 00mL, concentrate for solution for infusion (Placebo to VHB937)
Modality
Other

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