Amyotrophic Lateral SclerosisPhase 3PhoenixNCT05021536
Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
A Phase 3 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 69 sites in 12 countries.
Trial summary
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS 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?
- Yes, some participants may receive a placebo
- How many people
- 664 participants
- Where
- 69 sitesin 12 countries
- Study dates
- 28 Oct 2021to 1 Jan 2026
- 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
- Male or female, at least 18 years of age
- Diagnosis of ALS (definite or clinically probable)
- Time since onset of first symptom of ALS should be <24 months prior to randomization;
- If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone;
- Capable of providing informed consent
- Capable and willing to follow trial procedures including visits to the trial clinic and visit requirements;
- Women of child bearing potential (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 study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug
- Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug
Who cannot join
- Presence of tracheostomy or permanent assisted ventilation(PAV)
- Slow Vital Capacity (SVC) less than 55%
- 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 eGFR <60 mL/min/1.73m^2 (obtained within 12 weeks from first dose)
- Pregnant women (confirmed by a pregnancy test within 7 days of first dose) or women currently breastfeeding
Show all 16Show fewer
- 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)
- 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
- Previous treatment for ALS with cellular therapies or gene therapies
- Currently enrolled in another trial involving use of an investigational therapy
- Previous treatment with PB or taurursodiol within 30 days from Screening
- Implantation of Diaphragm Pacing System (DPS)
- Currently or previously treated within the last 30 days or planned exposure to any prohibited medications listed in Section 6.8 of the protocol
Trial locations and contacts
69 sites in 12 countries.
69 sites in 12 countries
United States28 sites
Emory University
Atlanta, Georgia, 30322
Augusta University Neuroscience Center
Augusta, Georgia, 30912
University of Colorado
Aurora, Colorado, 80045
Austin Neuromuscular Center
Austin, Texas, 78756
Johns Hopkins University School of Medicine Outpatient Center
Baltimore, Maryland, 21287
Healey & AMG Center for ALS Research at Massachusetts General Hospital
Boston, Massachusetts, 02114
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514
Northwestern University
Chicago, Illinois, 60611
The Ohio State University
Columbus, Ohio, 43210
Texas Neurology
Dallas, Texas, 72506
University of Florida
Gainesville, Florida, 32068
Virginia Commonwealth University
Henrico, Virginia, 23233
Somnos Clinical Research
Lincoln, Nebraska, 68510
University of Southern California
Los Angeles, California, 90033
Hennepin Healthcare Research Institute
Minneapolis, Minnesota, 55415
Rutgers University
New Brunswick, New Jersey, 08901
Columbia University
New York, New York, 10032
University of California Irvine
Orange, California, 92868
University of Pennsylvania
Philadelphia, Pennsylvania, 19107
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania, 19140
Barrow Neurological Institute
Phoenix, Arizona, 85013
California Pacific Medical Center Research Institute
San Francisco, California, 94109
Swedish Neuroscience Institute
Seattle, Washington, 98122
University of Washington
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida
Tampa, Florida, 33612
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27109
University of Massachusetts
Worcester, Massachusetts, 01655
France9 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
CHU Nice
Nice
Hôpital de la Salpêtrière
Paris
Le Centre Hospitalier Régional Universitaire de Tours
Tours
Germany7 sites
Charité - Universitätsmedizin Berlin
Berlin
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
Italy7 sites
Università degli Studi di Bari Aldo Moro
Bari
Centro Clinico NEMO
Milan
University of Milan Medical School
Milan
Azienda Ospedaliero Universitaria Di Modena
Modena
Università degli Studi della Campania Luigi Vanvitelli
Naples
University of Padua
Padova
University of Torino
Turin
Spain5 sites
Hospital del Mar
Barcelona
Hospital Universitari de Bellvitge-IDIBELL
Barcelona
Hospital San Rafael
Madrid
Biodonostia Health Research Institute; Hospital Universitario Donostia
San Sebastián
Hospital Universitario y Politécnico La Fe
Valencia
United Kingdom5 sites
King's College London
London
UCL Queen Square Institute of Neurology
London
University of Plymouth
Plymouth
Salford Royal Hospital Barnes
Salford
Sheffield Institute for Translational Neuroscience (SITraN)
Sheffield
About this study
Official title: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS)
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
Read the detailed description
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R and survival over 48 week. The trial will also assess the effects of AMX0035 on slow vital capacity, quality of life and plasma biomarkers of ALS.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Placebo, 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.
PlaceboOther
Matching Placebo Comparator
AMX0035Drug
Proprietary formulation of taurursodiol and sodium phenylbutyrate
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating Receives: Other: Placebo |
| AMX0035 | Experimental | Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating Receives: Drug: AMX0035 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change
Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function.
Measured: 48 weeks
Secondary outcomes (5)
Participant Quality of Life (QOL)
QOL will be measured using the 40-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) patient-reported outcome (PRO)
Measured: 48 weeks
Assess Long-Term Survival
Long-Term Survival will be obtained by monitoring of all-cause mortality
Measured: 3 years from LPI
Rate of Decline in Slow Vital Capacity (SVC)
Respiratory muscle function will be assessed according to slow vital capacity (SVC). SVC is measured in an upright position for at least three trials per assessment. SVC volumes will be standardized to the percentage of predicted normal value based on age, sex, and height.
Measured: 48 weeks
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change
Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function.
Measured: 24 weeks
Number of Participants With Adverse Events
Comparison Between Groups of Number of Participants With Adverse Events Until Planned Completion
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2021
- Study start28 Oct 2021
- Primary completion19 Jan 2024
- Study completion1 Jan 2026 (estimated)
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 664 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amylyx Study Director, Study director, Amylyx Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05021536
- Sponsor's protocol ID
- A35-004
- Acronym
- Phoenix
- Status last verified
- Aug 2024
- First submitted
- 20 Aug 2021
- Last updated
- 14 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT05021536
- Acronym / sponsor ID
- Phoenix / A35-004
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 664 participants (actual)
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Oct 2021 (actual)
- Primary completion
- 19 Jan 2024 (actual)
- Study completion
- 1 Jan 2026 (estimated)
- First posted
- 25 Aug 2021 (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 Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
Detailed description
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R and survival over 48 week. The trial will also assess the effects of AMX0035 on slow vital capacity, quality of life and plasma biomarkers of ALS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: PlaceboPlacebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating |
| AMX0035 | Experimental | Drug: AMX0035Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female, at least 18 years of age
- Diagnosis of ALS (definite or clinically probable)
- Time since onset of first symptom of ALS should be <24 months prior to randomization;
- If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone;
- Capable of providing informed consent
Key exclusion criteria (16 listed)
- Presence of tracheostomy or permanent assisted ventilation(PAV)
- Slow Vital Capacity (SVC) less than 55%
- 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 eGFR <60 mL/min/1.73m^2 (obtained within 12 weeks from first dose)
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 |
|---|---|---|
| PlaceboMatching Placebo Comparator | Other | Placebo |
| AMX0035Proprietary formulation of taurursodiol and sodium phenylbutyrate | Drug | AMX0035 |
Primary outcome
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change
Time frame: 48 weeks
Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function.
5 secondary outcomes
Participant Quality of Life (QOL)
Time frame: 48 weeks
Assess Long-Term Survival
Time frame: 3 years from LPI
Rate of Decline in Slow Vital Capacity (SVC)
Time frame: 48 weeks
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change
Time frame: 24 weeks
Number of Participants With Adverse Events
Time frame: 48 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2024.
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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.
Amylyx Study Director study director
Amylyx Pharmaceuticals
69 sites in 12 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 69 sites in 12 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 8 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 14 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 69 sites in United States, France, Germany, Italy, Spain and 7 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
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?
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change (time frame: 48 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 14 Aug 2024.