Clinical study on the efficacy and tolerabilty of Guabenz in Amiotropic Lateral Sclerosis

PROTEIN MISFOLDING, AMYOTROPHIC LATERAL SCLEROSIS AND GUANABENZ: A PHASE II RCT WITH FUTILITY DESIGN - PROMISE

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-005367-32-IT

Enrollment

208

Registered

2017-03-17

Start date

2016-02-29

Completion date

Unknown

Last updated

2025-01-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amiotrophic Lateral Sclerosis MedDRA version: 19.1 Level: PT Classification code 10028003 Term: Motor neurone disease System Organ Class: 10029205 - Nervous system disorders

Interventions

Product Name: GUANABENZ ACETATO Product Code: GUANA2015 Pharmaceutical Form: Tablet INN or Proposed INN: GUANABENZ CAS Number: 23256-50-0 Current Sponsor code: GUANA2015 Other descriptive name: GUANAB

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Diagnosis of, probable, probable laboratory supported or definite sporadic (SALS) or familiar (FALS) ALS according to the revised El Escorial criteria; 2) age >18 yrs; 3) onset =18 months before randomization; 4) sVC =70% in spinal onset; 5) riluzole 100 mg/day or no riluzole; 6) written informed consent Are the trial subjects under 18? no Number of subjects for this age range: 1 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 166 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 42

Exclusion criteria

Exclusion criteria: 1) PEG, NIV or tracheotomy; 3) known heart, renal or liver failure; 3) known intolerance to alfa2agonists; 4) known conditions at risk for cardiovascular disorders or symptomatic hypotension; 5) participation in a clinical trial within 3 months prior to the screening; 6) cognitive impairment defined by the Edinburg Cognitive and Behavior ALS Screen (ECAS) scale

Design outcomes

Primary

Measure	Time frame
Main Objective: To investigate the effect of guanabenz as add-on treatment compared to riluzole alone in reducing the proportion of patients progressed to higher stages of disease over 6 months in sporadic (SALS) or familial ALS (FALS) patients.;Secondary Objective: To assess safety and tolerability related to alpha2agonist activity of guanabenz. To explore serum biomarkers of ALS progression (creatinine, albumin tau, pNfH, TDP43, cystatin C, fetuin A, transthyretin, and CD14/S100ß and pNfH/C3) in guanabenz and riluzole alone arms at baseline and study end. In patients giving consent the biomarkers will be explored also in the cerebrospinal fluid.;Primary end point(s): Proportion of patients progressed to higher stages of disease at 6 months (measured by the ALS-MITOS system) by at least 35% in the guanabenz arm compared to their baseline stage and to the placebo arm (null hypothesis in the futility design). ;Timepoint(s) of evaluation of this end point: 6 months	—

Measure

Time frame

Main Objective: To investigate the effect of guanabenz as add-on treatment compared to riluzole alone in reducing the proportion of patients progressed to higher stages of disease over 6 months in sporadic (SALS) or familial ALS (FALS) patients.;Secondary Objective: To assess safety and tolerability related to alpha2agonist activity of guanabenz. To explore serum biomarkers of ALS progression (creatinine, albumin tau, pNfH, TDP43, cystatin C, fetuin A, transthyretin, and CD14/S100ß and pNfH/C3) in guanabenz and riluzole alone arms at baseline and study end. In patients giving consent the biomarkers will be explored also in the cerebrospinal fluid.;Primary end point(s): Proportion of patients progressed to higher stages of disease at 6 months (measured by the ALS-MITOS system) by at least 35% in the guanabenz arm compared to their baseline stage and to the placebo arm (null hypothesis in the futility design). ;Timepoint(s) of evaluation of this end point: 6 months

—

Secondary

Measure	Time frame
Secondary end point(s): 3) Difference in at least one serum and/or CSF biomarker of neurodegeneration (creatinine, albumin, tau, pNfH, TDP43, cystatin C, fetuin A, transthyretin, and CD14/S100ß and pNfH/C3 assayed by ELISA and Western blot) comparing baseline and study end between guanabenz and placebo arm.;Timepoint(s) of evaluation of this end point: At baseline and at the end of the study	—

Countries

Italy

Contacts

Public ContactSERVIZIO RICERCA E SVILUPPO CLINICO

FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA

crc@istituto-besta.it+390223942321

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026