Study Purpose:
The primary objective is to evaluate the safety and tolerability of AMX0035 over 108 weeks of open label treatment for participants previously enrolled in Study A35-004 (PHOENIX).Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
AMX0035
Placebo:
No
Phase:
Phase 3
Study Chair(s)/Principal Investigator(s):
Lahar Mehta, MD, Head, Global Clinical Development
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Full Study Summary:
Study Sponsor:
Amylyx Pharmaceuticals Inc.
Estimated Enrollment:
600
Estimated Study Start Date:
12 / 29 / 2022
Estimated Study Completion Date:
08 / 01 / 2026
Posting Last Modified Date:
05 / 08 / 2023
Date Study Added to neals.org:
11 / 17 / 2022
Minimum Age:
18 Years
Maximum Age:
N/A
Inclusion Criteria:1. Previous participation in Study A35-004 (PHOENIX), including completion of the randomized controlled phase through Week 48 (this timepoint may be upcoming at the time of screening). Participants who do not complete randomized-controlled phase through Week 48 for medical reasons may be included on a case-by-case basis, in consultation with the Sponsor;
2. Capable of providing informed consent;
3. Capable and willing to follow trial procedures including visits to the trial clinic, remote visits, and survival status reporting requirements;
4. Women of childbearing potential (WOCBP; e.g., not post-menopausal for at least one year or surgically sterile must agree to use adequate birth control for the duration of the trial and 3 months after the last dose of AMX0035;
1. 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum Follicle-stimulating hormone (FSH) levels > 40 mIU/ml (milli-international units per milliliter) or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy.
2. Acceptable contraception methods for use in this trial are:
- Hormonal methods, such as birth control pills, patches, injections, vaginal ring, or implants;
- Barrier methods (such as a condom or diaphragm) used with a spermicide (a foam, cream, or gel that kills sperm);
- Intrauterine device (IUD);
- Abstinence (no heterosexual sex);
- Unique partner who is surgically sterile (men) or not of childbearing potential (female).
5. Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of AMX0035;
6. Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of AMX0035;
7. Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of AMX0035
Exclusion Criteria:
1. History of known allergy to phenyl butyrate or bile salts;
2. Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 5 times the upper limit of the normal (obtained within 12 weeks from first dose);
3. Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 normal (obtained within 12 weeks from first dose);
4. Pregnant women or women currently breastfeeding;
5. Current severe biliary disease which may result in the Investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the gallbladder, abscess of the gallbladder;
6. History of Class III/IV heart failure (per New York Heart Association - NYHA);
7. Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment;
8. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment;
9. Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, severe laboratory test anomaly or clinically significant electrocardiogram [ECG] changes) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment;
10. Currently enrolled in another trial (excluding Study A35-004 (PHOENIX)) involving use of an investigational therapy (or within 5 plasma half-lives) prior to first dose at Baseline Visit;
11. Implantation of Diaphragm Pacing System (DPS);
12. Currently or previously treated within the last 30 days (or 5 half-lives, whichever is longer) from first dose at the Baseline Visit or planned exposure during the treatment period to any prohibited medications listed in Section 6.7 of the protocol.
University Hospitals Leuven
Leuven
Belgium
Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC)
Bron
France
Hopital Gabriel Montpied Service de Neurologie
Clermont-Ferrand
France
CHRU de Lille - Hôpital Roger Salengro
Lille
France
CHU de Limoges - Hôpital Dupuytren
Limoges
France
Hôpitaux Universitaires de Marseille Timone
Marseille
France
Gui de Chauliac
Montpellier Cedex 5
France
CHU de Montpellier
Montpellier
France
CHU Nice
Nice
France
Hôpital de la Salpêtrière
Paris
France
Le Centre Hospitalier Régional Universitaire de Tours
Tours
France
Uniklinikum Dresden
Dresden
Germany
Hannover Medical School
Hannover
Germany
Jena University Hospital
Jena
Germany
Medizinische Fakultät Mannheim der Universität Heidelberg
Mannheim
Germany
University Medical Center Rostock
Rostock
Germany
Ulm University Medical Centre
Ulm
Germany
Trinity College Dublin/Beaumont Hospital
Dublin
Ireland
Università degli Studi di Bari Aldo Moro
Bari
Italy
IRCCS - Istituto Auxologico italiano
Milano
Italy
Centro Clinico NEMO
Milan
Italy
IRCCS - Ospedale San Raffaele
Milan
Italy
University of Milan Medical School
Milan
Italy
Azienda Ospedaliero Universitaria Di Modena
Modena
Italy
Università degli Studi della Campania Luigi Vanvitelli
Napoli
Italy
University of Padua
Padova
Italy
Università degli Studi di Bari Aldo Moro
Tricase
Italy
University of Torino
Turin
Italy
University Medical Center Utrecht
Utrecht
Netherlands
Centrum Medyczne Linden
Kraków
Poland
City Clinic Warsaw
Warsaw
Poland
Centro Hospitalar Universitário Lisboa-Norte
Lisbon
Portugal
Hospital del Mar
Barcelona
Spain
Hospital Universitari de Bellvitge-IDIBELL
Barcelona
Spain
Hospital Universitario de Basurto
Bilbao
Spain
Hospital San Rafael
Madrid
Spain
Biodonostia Health Research Institute; Hospital Universitario Donostia
San Sebastián
Spain
Hospital Universitario y Politécnico La Fe
Valencia
Spain
Karolinska Institutet
Stockholm
Sweden
Umeå University Hospital
Umeå
Sweden
The Walton Centre NHS Trust
Liverpool
United Kingdom
King's College London
London
United Kingdom
UCL Queen Square Institute of Neurology
London
United Kingdom
University of Plymouth
Plymouth
United Kingdom
Salford Royal Hospital Barnes Clinical Research Team
Salford
United Kingdom
Sheffield Institute for Translational Neuroscience (SITraN)
Sheffield
United Kingdom