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NCT05866926: ADOREXT

Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis

Terminated Phase 3 Results posted Last updated 4 March 2025
What this trial tests

Phase 3 trial testing FAB122 in Amyotrophic Lateral Sclerosis in 201 participants. Terminated before completion.

Timeline
6 March 2023
Primary endpoint
9 January 2024
22 February 2024

Quick facts

Lead sponsorFerrer Internacional S.A.
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationnon randomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment201
Start date6 March 2023
Primary completion9 January 2024
Estimated completion22 February 2024
Sites1 location across Spain

Drugs / interventions tested

Conditions studied

Sponsor

Ferrer Internacional S.A. — full company profile →

Who can join

Adults 18 to 80, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants Experiencing Treatment Emergent Adverse Events Primary · approximately 44 weeks

To evaluate the long-term safety of FAB122 in patients with ALS by assessing Number of Participants experiencing Treatment Emergent Adverse Events, evaluating nature and severity. The study duration for these subjects, and therefore the duration of FAB122 treatment, was variable depending on the subject's start date, ranging from 3 to approximately 44 weeks.

Any TEAE
GroupValue95% CI
FAB12277
Placebo41
Any Serious AE
GroupValue95% CI
FAB12229
Placebo15
Any Serious TEAE
GroupValue95% CI
FAB12229
Placebo15
Any TEAE leading to treatment discontinuation
GroupValue95% CI
FAB1227
Placebo5
Any AE leading to death
GroupValue95% CI
FAB12216
Placebo9
TEAE by relationship - Unrelated
GroupValue95% CI
FAB12275
Placebo40
TEAE by relationship - Related
GroupValue95% CI
FAB1226
Placebo3
TEAE by Severity - Mild
GroupValue95% CI
FAB12252
Placebo31
The Secondary Efficacy Objective to Evaluate the Effect of Treatment With FAB122 Based on Change From Baseline in ALSFRS-R Until End of Study Secondary · 45 weeks

Change from baseline in ALSFRS-R total score until the end of the study. Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Maximum value is 48 points and represents better outcome. Minimum value is 0 and represents worse outcome.

GroupValue95% CI
FAB122-4.4± 4.8
Placebo-3.8± 5

Adverse events — posted to ClinicalTrials.gov

Time frame: 45 weeks. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

FAB122
Serious: 29/135 (21%)
Deaths: 16/135
Placebo
Serious: 15/66 (23%)
Deaths: 9/66

Serious adverse events (25 terms)

ReactionSystemFAB122Placebo
Respiratory FailureRespiratory, thoracic and mediastinal disorders
PneumoniaInfections and infestations
DysponeaRespiratory, thoracic and mediastinal disorders
Chronic respiratory failureRespiratory, thoracic and mediastinal disorders
Pneumonia aspirationInfections and infestations
Cardiac arrestCardiac disorders
DeathGeneral disorders
COVID-19Infections and infestations
Lower respiratory track infectionInfections and infestations
Craniocerebral injuryInjury, poisoning and procedural complications
Amyotrophic Lateral SclerosisNervous system disorders
HospitalizationSurgical and medical procedures
Hypertensive crisisVascular disorders
Accute respiratory failureRespiratory, thoracic and mediastinal disorders
Lung disorderRespiratory, thoracic and mediastinal disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
Tooth infectionInfections and infestations
Cardiac failureCardiac disorders
Myocardial infarctionCardiac disorders
FallInjury, poisoning and procedural complications
Subdural haematomaInjury, poisoning and procedural complications
Lypoprotein increasedInvestigations
Weight decreasedInvestigations
Cognitive disorderNervous system disorders
ApathyPsychiatric disorders
Other adverse events (25 terms — click to expand)

ReactionSystemFAB122Placebo
Urinary track infectiousInfections and infestations
Respiratory failureRespiratory, thoracic and mediastinal disorders
DysphagiaGastrointestinal disorders
PneumoniaInfections and infestations
FallInjury, poisoning and procedural complications
COVID-19Infections and infestations
ConstipationGastrointestinal disorders
FatigueGeneral disorders
Upper respiratory tract infectionInfections and infestations
NauseaGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
CoughRespiratory, thoracic and mediastinal disorders
Gamma-glutamyltransferase increasedInvestigations
InsomniaPsychiatric disorders
Renal ColicRenal and urinary disorders
ArthralgiaMusculoskeletal and connective tissue disorders
HypertensionVascular disorders
InfluenzaInfections and infestations
Lower respiratory track infectionInfections and infestations
NasopharyngitisInfections and infestations
DyspnoeaRespiratory, thoracic and mediastinal disorders
ContusionInjury, poisoning and procedural complications
Skin lacerationInjury, poisoning and procedural complications
SyncopeNervous system disorders
DeathGeneral disorders

Most-reported serious reactions: Respiratory Failure, Pneumonia, Dysponea, Chronic respiratory failure, Pneumonia aspiration, Cardiac arrest, Death, COVID-19.

Data from ClinicalTrials.gov NCT05866926 adverse events section.

Sponsor's own description

A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. CRISPR/Cas9: implication for modeling and therapy of amyotrophic lateral sclerosis.
    Shi Y, Zhao Y, Lu L, Gao Q, et al · · 2023 · cited 10× · PMID 37483353 · DOI 10.3389/fnins.2023.1223777
  2. Drug repurposing in amyotrophic lateral sclerosis (ALS).
    Carroll E, Scaber J, Huber KVM, Brennan PE, et al · · 2025 · cited 3× · PMID 40029669 · DOI 10.1080/17460441.2025.2474661
  3. Amyotrophic Lateral Sclerosis: Pathophysiological Mechanisms and Treatment Strategies (Part 2).
    Tolochko C, Shiryaeva O, Alekseeva T, Dyachuk V. · · 2025 · cited 2× · PMID 40508048 · DOI 10.3390/ijms26115240

Verify or expand the search:

Other trials of FAB122

Trials testing the same drug.

Other recruiting trials for Amyotrophic Lateral Sclerosis

Currently open trials in the same condition.

Other Ferrer Internacional S.A. trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing