Amyotrophic Lateral SclerosisPhase 3NCT05619783
Extension Study Evaluating The Safety And Tolerability of AMX0035
A Phase 3 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 46 sites in 11 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- AMX0035
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 352 participants
- Where
- 46 sitesin 11 countries
- Study dates
- 29 Dec 2022to 30 Oct 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
- Capable of providing informed consent;
- Capable and willing to follow trial procedures including visits to the trial clinic, remote visits, and survival status reporting requirements;
- 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;
- 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.
- Acceptable contraception methods for use in this trial are:
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- 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).
- Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of AMX0035;
- Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of AMX0035;
- 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
Who cannot join
- History of known allergy to phenyl butyrate or bile salts;
- 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);
- Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 normal (obtained within 12 weeks from first dose);
- Pregnant women or women currently breastfeeding;
- 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;
- History of Class III/IV heart failure (per New York Heart Association - NYHA);
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- Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment;
- 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;
- 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;
- 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;
- Implantation of Diaphragm Pacing System (DPS);
- 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.
Trial locations and contacts
46 sites in 11 countries.
46 sites in 11 countries
France10 sites
Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC)
Bron
Hopital Gabriel Montpied Service de Neurologie
Clermont-Ferrand
CHRU de Lille - Hôpital Roger Salengro
Lille
CHU de Limoges - Hôpital Dupuytren
Limoges
Hôpitaux Universitaires de Marseille Timone
Marseille
CHU de Montpellier
Montpellier
Gui de Chauliac
Montpellier
CHU Nice
Nice
Hôpital de la Salpêtrière
Paris
Le Centre Hospitalier Régional Universitaire de Tours
Tours
Italy10 sites
Università degli Studi di Bari Aldo Moro
Bari
Centro Clinico NEMO
Milan
IRCCS - Ospedale San Raffaele
Milan
University of Milan Medical School
Milan
IRCCS - Istituto Auxologico italiano
Milan
Azienda Ospedaliero Universitaria Di Modena
Modena
Università degli Studi della Campania Luigi Vanvitelli
Naples
University of Padua
Padova
Università degli Studi di Bari Aldo Moro
Tricase
University of Torino
Turin
Germany6 sites
Uniklinikum Dresden
Dresden
Hannover Medical School
Hanover
Jena University Hospital
Jena
Medizinische Fakultät Mannheim der Universität Heidelberg
Mannheim
University Medical Center Rostock
Rostock
Ulm University Medical Centre
Ulm
Spain6 sites
Hospital del Mar
Barcelona
Hospital Universitari de Bellvitge-IDIBELL
Barcelona
Hospital Universitario de Basurto
Bilbao
Hospital San Rafael
Madrid
Biodonostia Health Research Institute; Hospital Universitario Donostia
San Sebastián
Hospital Universitario y Politécnico La Fe
Valencia
United Kingdom6 sites
The Walton Centre NHS Trust
Liverpool
King's College London
London
UCL Queen Square Institute of Neurology
London
University of Plymouth
Plymouth
Salford Royal Hospital Barnes Clinical Research Team
Salford
Sheffield Institute for Translational Neuroscience (SITraN)
Sheffield
About this study
Official title: A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants With Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)
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).
Read the detailed description
All participants will receive open-label treatment with AMX0035, starting on Day 1 with twice a day oral dosing (once in the morning and once in the evening) for the duration of the study. After the Baseline Visit (Day 1), enrolled participants will complete visits approximately every 12 weeks (± 2 weeks), until Week 108 or the end of treatment (EOT) visit, followed by a safety follow-up approximately 28 days after the last dose. A survival follow-up assessment will be completed every 12 weeks following the EOT visit until time of death or end of study (EOS).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- AMX0035
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMX0035Drug
Also called RELYVRIO
Combination of 3 g phenylbutyrate and 1 g taurursodiol
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | All participants will be treated with oral (or feeding tube) AMX0035 (a fixed-dose combination of Sodium Phenylbutyrate (PB) and taurursodiol). All participants will take 2 sachets daily (one morning dose and one evening dose) starting on Day 1, for the duration of the study (if twice a day dosing is poorly tolerated, dosing interruptions and reductions are further discussed in section 6.3) AMX0035 will be supplied by Amylyx as a carton box containing approximately 1 month supply of single use sachets. Each AMX0035 sachet contains active ingredients in a powder formulation with 3 g PB and 1 g taurursodiol. AMX0035 powder is mixed with water and taken orally (or via feeding tube). Receives: Drug: AMX0035 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the Incidence of Treatment-Emergent Adverse Events during treatment with AMX0035
Incidence of all adverse events (AE)s; AEs leading to treatment discontinuation or study withdrawal, and all serious adverse events (SAE)s in participants treated with AMX0035
Measured: 108 weeks
Secondary outcomes (1)
To assess the impact of long-term treatment with AMX0035 on survival
- Overall survival of all-cause mortality
- Ventilation free survival (defined as death, tracheostomy for respiratory distress or permanent non-invasive ventilation [>22 hours per day for 7 consecutive days])
Measured: 108 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2022
- Study start29 Dec 2022
- Primary completion16 Oct 2024
- Study completion30 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 352 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lahar Mehta, MD, Study director, Head, Global Clinical Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05619783
- Sponsor's protocol ID
- A35-011
- Status last verified
- Jan 2025
- First submitted
- 27 Oct 2022
- Last updated
- 13 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants With Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)
- ClinicalTrials.gov ID
- NCT05619783
- Acronym / sponsor ID
- A35-011
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 352 participants (actual)
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Dec 2022 (actual)
- Primary completion
- 16 Oct 2024 (actual)
- Study completion
- 30 Oct 2024 (actual)
- First posted
- 17 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
All participants will receive open-label treatment with AMX0035, starting on Day 1 with twice a day oral dosing (once in the morning and once in the evening) for the duration of the study. After the Baseline Visit (Day 1), enrolled participants will complete visits approximately every 12 weeks (± 2 weeks), until Week 108 or the end of treatment (EOT) visit, followed by a safety follow-up approximately 28 days after the last dose. A survival follow-up assessment will be completed every 12 weeks following the EOT visit until time of death or end of study (EOS).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: AMX0035All participants will be treated with oral (or feeding tube) AMX0035 (a fixed-dose combination of Sodium Phenylbutyrate (PB) and taurursodiol). All participants will take 2 sachets daily (one morning dose and one evening dose) starting on Day 1, for the duration of the study (if twice a day dosing is poorly tolerated, dosing interruptions and reductions are further discussed in section 6.3) AMX0035 will be supplied by Amylyx as a carton box containing approximately 1 month supply of single use sachets. Each AMX0035 sachet contains active ingredients in a powder formulation with 3 g PB and 1 g taurursodiol. AMX0035 powder is mixed with water and taken orally (or via feeding tube). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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;
- Capable of providing informed consent;
- Capable and willing to follow trial procedures including visits to the trial clinic, remote visits, and survival status reporting requirements;
- 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;
- Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of AMX0035;
Key exclusion criteria (12 listed)
- History of known allergy to phenyl butyrate or bile salts;
- 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);
- Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 normal (obtained within 12 weeks from first dose);
- Pregnant women or women currently breastfeeding;
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMX0035Also: RELYVRIOCombination of 3 g phenylbutyrate and 1 g taurursodiol | Drug | Active |
Primary outcome
To assess the Incidence of Treatment-Emergent Adverse Events during treatment with AMX0035
Time frame: 108 weeks
Incidence of all adverse events (AE)s; AEs leading to treatment discontinuation or study withdrawal, and all serious adverse events (SAE)s in participants treated with AMX0035
1 secondary outcome
To assess the impact of long-term treatment with AMX0035 on survival
Time frame: 108 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lahar Mehta, MD study director
Head, Global Clinical Development
46 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 46 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in France, Italy, Germany, Spain, United Kingdom and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the Incidence of Treatment-Emergent Adverse Events during treatment with AMX0035 (time frame: 108 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2025.