Amyotrophic Lateral SclerosisPhase 2/3NCT04516096
A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2/3 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 5 sites in United States.
Trial summary
The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for 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?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 5 sitesUnited States
- Study dates
- 22 Nov 2020to 31 Jan 2023
- Phase
- Phase 2/3
- 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
- Any patient who has completed follow-up in an Amylyx sponsored trial of AMX0035 for the treatment of ALS will be eligible to enroll into this Protocol
- Capable of providing informed consent
- Capable and willing to follow trial procedures
- Capable and willing of travelling to the site at regular intervals for interim site visits and to return and collect study drug or able to attend telemedicine remote site visits if such are currently in use at the site
- Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the protocol.
- 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 nursing, be pregnant or 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 sperm for donation for the duration of the study and 3 months after last dose of study drug
Who cannot join
- Ongoing severe adverse events that in the opinion of the Site Investigator are contraindication to the study drug, including severe renal or liver insufficiency or Class III/IV heart failure (per New York Heart Association)
- Ongoing serious adverse event that was assessed as related to study drug per the Site Investigator
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the patient to provide informed consent, according to Site Investigator judgment;
- 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, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the patient if they were to participate in the study, according to Site Investigator judgment;
- Treatment, current or within 90 days from screening with any cell therapies or gene therapies;
- Implantation of Diaphragm Pacing System (DPS);
- Current or planned exposure to any prohibited medications listed in Section
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Michigan Medical Center
Ann Arbor, Michigan, 48109
Massachusetts General Hospital
Boston, Massachusetts, 02114
Forbes Norris MDA/ALS Research Center - California Pacific Medical Center
San Francisco, California, 94114
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655
About this study
Official title: AMX0035 for Amyotrophic Lateral Sclerosis - Compassionate Use Protocol
The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.
Read the detailed description
In this open-label, compassionate extended use study, patients who have completed their participation in an Amylyx sponsored protocol for ALS may be offered compassionate use extension of treatment with AMX0035 under this protocol. Patient currently treated with AMX0035 will continue to receive treatment at their current dose/regimen. Patients who have interrupted AMX0035 for more than 28 days will resume AMX0035 treatment with 1 sachet (oral [or feeding tube]) once a day in the morning to be escalated to twice a day (morning and evening) after approximately 2 weeks on the basis of GI tolerance. Enrolled patients will complete regular outpatient visit approximately every 16 weeks (± 2 weeks). Completion of the ALS functional rating scale (ALSFRS-R) will be optional.
- 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
Tauroursodeoxycholic Acid and Sodium Phenylbutyrate orally given twice a day
| Study group | Role | What they receive |
|---|---|---|
| AMX-0035 long term treatment extension | Experimental | AMX0035 administered twice daily p.o. Receives: Drug: AMX0035 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment emergent Adverse Events
Rate of treatment emergent adverse events during AMX0035 therapy
Measured: Through study completion an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2020
- Study start22 Nov 2020
- Primary completion30 Dec 2022
- Study completion31 Jan 2023
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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label long term extension of treatment
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Machelle Manual, PhD, Study director, Amylyx Pharmaceuticals Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04516096
- Sponsor's protocol ID
- A35-003
- Status last verified
- Oct 2022
- First submitted
- 9 Aug 2020
- Last updated
- 7 Apr 2023
- 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 7 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AMX0035 for Amyotrophic Lateral Sclerosis - Compassionate Use Protocol
- ClinicalTrials.gov ID
- NCT04516096
- Acronym / sponsor ID
- A35-003
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Nov 2020 (actual)
- Primary completion
- 30 Dec 2022 (actual)
- Study completion
- 31 Jan 2023 (actual)
- First posted
- 18 Aug 2020 (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 protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.
Detailed description
In this open-label, compassionate extended use study, patients who have completed their participation in an Amylyx sponsored protocol for ALS may be offered compassionate use extension of treatment with AMX0035 under this protocol. Patient currently treated with AMX0035 will continue to receive treatment at their current dose/regimen. Patients who have interrupted AMX0035 for more than 28 days will resume AMX0035 treatment with 1 sachet (oral [or feeding tube]) once a day in the morning to be escalated to twice a day (morning and evening) after approximately 2 weeks on the basis of GI tolerance. Enrolled patients will complete regular outpatient visit approximately every 16 weeks (± 2 weeks). Completion of the ALS functional rating scale (ALSFRS-R) will be optional.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label long term extension of treatment
| Arm | Type | Receives |
|---|---|---|
| AMX-0035 long term treatment extension | Experimental | Drug: AMX0035AMX0035 administered twice daily p.o. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Any patient who has completed follow-up in an Amylyx sponsored trial of AMX0035 for the treatment of ALS will be eligible to enroll into this Protocol
- Capable of providing informed consent
- Capable and willing to follow trial procedures
- Capable and willing of travelling to the site at regular intervals for interim site visits and to return and collect study drug or able to attend telemedicine remote site visits if such are currently in use at the site
- Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the protocol.
Key exclusion criteria (8 listed)
- Ongoing severe adverse events that in the opinion of the Site Investigator are contraindication to the study drug, including severe renal or liver insufficiency or Class III/IV heart failure (per New York Heart Association)
- Ongoing serious adverse event that was assessed as related to study drug per the Site Investigator
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the patient to provide informed consent, according to Site Investigator judgment;
- 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, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the patient if they were to participate in the study, according to Site Investigator judgment;
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 |
|---|---|---|
| AMX0035Tauroursodeoxycholic Acid and Sodium Phenylbutyrate orally given twice a day | Drug | AMX-0035 long term treatment extension |
Primary outcome
Treatment emergent Adverse Events
Time frame: Through study completion an average of 1 year
Rate of treatment emergent adverse events during AMX0035 therapy
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 7 Apr 2023.
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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.
Machelle Manual, PhD study director
Amylyx Pharmaceuticals Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis, run by Amylyx Pharmaceuticals Inc. at 5 sites in United States.
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 8 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 7 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Treatment emergent Adverse Events (time frame: Through study completion an average of 1 year). 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 7 Apr 2023.