ALSPhase 1/2NCT04987671
Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS
A Phase 1/2 study testing AMX0035 in people with ALS, run by Amylyx Pharmaceuticals Inc. at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ALS
- What is being tested?
- AMX0035
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Aug 2021to Sep 2023
- Phase
- Phase 1/2
- 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, >=18years of age;
- Diagnosis of sporadic ALS (definite, probable, laboratory probable, possible) made by physician experienced with management of ALS as defined by the World Federation of Neurology revised El Escorial criteria;
- If taking riluzole, must be on a stable dose for >30 days prior to Day 1 and anticipated to remain at that dose until the final study visit.
- If taking edaravone, must be on a stable regimen for > 30 days prior to Day 1 and infusion(s) can be scheduled to be performed at no less than 48 hours prior or after the planned pharmacokinetic and pharmacodynamic (PK/PD) sampling.
- Capable of providing informed consent and following trial procedures;
- Geographically accessible to the site;
Show all 17Show fewer
- Able to undergo the study procedures (including planned sampling on 3 occasions) and to adhere to the visit schedule, as determined by Investigator;
- 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 for at least 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
- Acceptable birth control methods for use in this study 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 child bearing potential (female)
Who cannot join
- Familial ALS
- Forced vital capacity < 50% (or alternatively SVC) or presence of tracheostomy or under PAV (PAV is defined as more than 22 hours daily of non-invasive mechanical ventilation for more than 7 days);
- Planned elective surgery (such as feeding tube or edaravone access port placement) during the duration of the study;
- History of known allergy to PB or bile salts;
- Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase (AST and/or ALT) > 3 times the upper limit of the normal;
- Renal insufficiency as defined by eGFR < 60 mL/min/1.73m2;
Show all 59Show fewer
- Ongoing Anemia with Hg concentration < 10.0 g/dL
- Pregnant women or women currently breastfeeding;
- Current biliary disease which may lead to biliary obstruction or impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;
- History of Class III/IV heart failure (per New York Heart Association - NYHA);
- Patient under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Site Investigator clinical judgement;
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment;
- Presence of an active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period.
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study, according to Site Investigator judgment.
- Clinically significant, as determined by the Investigator, 12-lead ECG abnormalities at screening.
- Treatment, current or within 90 days from start of study treatment, with any cell therapies or gene therapies;
- Treatment, current or within 90 days from start of study treatment, with experimental medication (for ALS or other indications)
- Current or anticipated need for a Diaphragm Pacing System (DPS);
- Anything that, in the opinion of the Site Investigator preclude the subject's full compliance with or completion of the study;
- Exposure to any disallowed medications listed below:
- HDAC Inhibitors including:
- Valproate
- Vorinostat (Zolinza)
- Romidepsin
- Chidamide
- Panobinostat
- Lithium
- Butyrate
- Suramin
- Probenecid
- Bile Acid Sequestrants including:
- Cholestyramine and Cholestyramine Light
- Questran and Questran Light
- Welchol
- Colestid and Colestid Flavored
- Prevalite
- Product that may interact with sterol absorption or excretion
- Ezetimibe
- Note on Antacids within Two Hours of AMX0035 Administration: Antacids containing aluminum hydroxide or smectite (aluminum oxide) may not be taken within two hours of administration of MX0035 as they inhibit absorption of taurursodiol. These include:
- Alamag
- Alumina and Magnesia
- Antacid, Antacid M and Antacid Suspension
- Gen-Alox
- Kudrox
- M.A.H.
- Maalox HRF and Maalox TC
- Magnalox
- Maldroxal
- Mylanta and Mylanta Ultimate
- Ri-Mox
- Rulox
- Clinically significant, in the opinion of the Investigator, infection or inflammation at time of screening or admission.
- Acute gastrointestinal symptoms (eg, nausea, vomiting, diarrhea) at time of screening or admission or a or clinical diagnosis of irritable bowel syndrome (IBS) per ROME criteria (Appendix 7).
- Any current or previous illicit use of Class A drugs such as opiates, cocaine, ecstasy, LSD, and amphetamines (Class B).
- An alcoholic intake greater than 14 units per week or unwillingness to stop alcohol consumption for the duration of the study. Note: 1 unit = 8 g ethanol (250 mL of beer or approximately 10 oz, 1 glass wine [100 mL or approximately 3 oz], 1 measure spirits [30 mL or approximately 1 oz]).
- Use of drugs with enzyme inducing properties such as St John's Wort within 3 weeks prior to the first administration of investigational product.
- History of plasma/blood donation in the last 2 months.
- Any condition, which compromises ability to give informed consent or to communicate with the Investigator as required for the completion of this study.
- Unwilling to conform to all lifestyle considerations and restrictions mandated by the protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Norman Fixel Institute for Neurological Diseases
Gainesville, Florida, 32608
About this study
Official title: A Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS
The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.
Read the detailed description
AMX0035 is a proprietary combination of two small molecules, phenylbutyrate (PB) and taurursodiol. The combination of PB and taurursodiol in AMX0035 is intended to block neuronal death and neurotoxic inflammation through simultaneous reduction of endoplasmic reticulum (ER) stress and mitochondrial stress. The pharmacokinetic of AMX0035 has been evaluated in healthy volunteers. This Phase IIa trial is intended to evaluate pharmacokinetic of PB and taurursodiol upon single dose multiple dose administration of AMX0035 in patients with ALS.
- Conditions
- ALS
- Being tested
- AMX0035
- Keywords
- Neurodegenerative Diseases, Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Nervous System Diseases, Neuromuscular Diseases
- Medical subject headings
- Neurodegenerative Diseases, Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Nervous System Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMX0035Drug
Fixed dose combination of Sodium Phenylbutyrate and taurursodiol.
| Study group | Role | What they receive |
|---|---|---|
| Treatment with AMX0035 | Experimental | Two sequential study period. In Period 1, subject receive AMX0035 daily for approximately 14 days. In Period 2, subjects receive AMX0035 twice a day, morning and evening, for up to 25 days. Receives: Drug: AMX0035 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood concentration of PB and taurursodiol
Maximum plasma concentration - Cmax of PB and taurursodiol
Measured: Between Day 1 and Day 40
Systemic exposure to PB and taurursodiol
Area under the plasma concentration versus time curve - AUC of PB and taurursodiol
Measured: Between Day 1 and Day 40
Secondary outcomes (3)
Effect of demographic characteristics on blood concentration of PB and taurursodiol
Maximum plasma concentration - Cmax of PB and taurursodiolin relation to gender, body weight and age.
Measured: Between Day 1 and Day 40
Effect of demographic characteristics on systemic exposure of PB and taurursodiol
Area under the plasma concentration versus time curve - AUC of PB and taurursodiol relation to gender, body weight and age.
Measured: Between Day 1 and Day 40
Effect of a fixed dose combination of sodium phenyl butyrate (PB) and taurursodiol on pharmacodynamic activity
Histone 3 and histone 4 acetylation levels in peripehral blood mononuclear cells (PBMC)
Measured: Between Day 1 and Day 40
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2021
- Study start5 Aug 2021
- Primary completion11 May 2022
- Study completionSep 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for ALSStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04987671
- Sponsor's protocol ID
- A35-005
- Status last verified
- Jul 2023
- First submitted
- 15 Jul 2021
- Last updated
- 24 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cuadrado-Tejedor M, Ricobaraza AL, Torrijo R, Franco R, Garcia-Osta A. Phenylbutyrate is a multifaceted drug that exerts neuroprotective effects and reverses the Alzheimer s disease-like phenotype of a commonly used mouse model. Curr Pharm Des. 2013;19(28):5076-84. doi: 10.2174/1381612811319280006.
PubMed 23448463Cudkowicz ME, Andres PL, Macdonald SA, Bedlack RS, Choudry R, Brown RH Jr, Zhang H, Schoenfeld DA, Shefner J, Matson S, Matson WR, Ferrante RJ; Northeast ALS and National VA ALS Research Consortiums. Phase 2 study of sodium phenylbutyrate in ALS. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106. doi: 10.1080/17482960802320487.
PubMed 18688762Nunes AF, Amaral JD, Lo AC, Fonseca MB, Viana RJ, Callaerts-Vegh Z, D'Hooge R, Rodrigues CM. TUDCA, a bile acid, attenuates amyloid precursor protein processing and amyloid-beta deposition in APP/PS1 mice. Mol Neurobiol. 2012 Jun;45(3):440-54. doi: 10.1007/s12035-012-8256-y. Epub 2012 Mar 23.
PubMed 22438081Dionisio PA, Amaral JD, Ribeiro MF, Lo AC, D'Hooge R, Rodrigues CM. Amyloid-beta pathology is attenuated by tauroursodeoxycholic acid treatment in APP/PS1 mice after disease onset. Neurobiol Aging. 2015 Jan;36(1):228-40. doi: 10.1016/j.neurobiolaging.2014.08.034. Epub 2014 Sep 28.
PubMed 25443293Lo AC, Callaerts-Vegh Z, Nunes AF, Rodrigues CM, D'Hooge R. Tauroursodeoxycholic acid (TUDCA) supplementation prevents cognitive impairment and amyloid deposition in APP/PS1 mice. Neurobiol Dis. 2013 Feb;50:21-9. doi: 10.1016/j.nbd.2012.09.003. Epub 2012 Sep 10.
PubMed 22974733Ricobaraza A, Cuadrado-Tejedor M, Marco S, Perez-Otano I, Garcia-Osta A. Phenylbutyrate rescues dendritic spine loss associated with memory deficits in a mouse model of Alzheimer disease. Hippocampus. 2012 May;22(5):1040-50. doi: 10.1002/hipo.20883. Epub 2010 Nov 10.
PubMed 21069780Ricobaraza A, Cuadrado-Tejedor M, Perez-Mediavilla A, Frechilla D, Del Rio J, Garcia-Osta A. Phenylbutyrate ameliorates cognitive deficit and reduces tau pathology in an Alzheimer's disease mouse model. Neuropsychopharmacology. 2009 Jun;34(7):1721-32. doi: 10.1038/npp.2008.229. Epub 2009 Jan 14.
PubMed 19145227Rodrigues CM, Sola S, Sharpe JC, Moura JJ, Steer CJ. Tauroursodeoxycholic acid prevents Bax-induced membrane perturbation and cytochrome C release in isolated mitochondria. Biochemistry. 2003 Mar 18;42(10):3070-80. doi: 10.1021/bi026979d.
PubMed 12627974Wiley JC, Pettan-Brewer C, Ladiges WC. Phenylbutyric acid reduces amyloid plaques and rescues cognitive behavior in AD transgenic mice. Aging Cell. 2011 Jun;10(3):418-28. doi: 10.1111/j.1474-9726.2011.00680.x. Epub 2011 Mar 22.
PubMed 21272191Wright JM, Zeitlin PL, Cebotaru L, Guggino SE, Guggino WB. Gene expression profile analysis of 4-phenylbutyrate treatment of IB3-1 bronchial epithelial cell line demonstrates a major influence on heat-shock proteins. Physiol Genomics. 2004 Jan 15;16(2):204-11. doi: 10.1152/physiolgenomics.00160.2003.
PubMed 14583596Zhou W, Bercury K, Cummiskey J, Luong N, Lebin J, Freed CR. Phenylbutyrate up-regulates the DJ-1 protein and protects neurons in cell culture and in animal models of Parkinson disease. J Biol Chem. 2011 Apr 29;286(17):14941-51. doi: 10.1074/jbc.M110.211029. Epub 2011 Mar 3.
PubMed 21372141
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS
- ClinicalTrials.gov ID
- NCT04987671
- Acronym / sponsor ID
- A35-005
- Conditions
- ALS
- Keywords
- Neurodegenerative Diseases, Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Nervous System Diseases, Neuromuscular Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Aug 2021 (actual)
- Primary completion
- 11 May 2022 (actual)
- Study completion
- Sep 2023 (estimated)
- First posted
- 3 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 purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.
Detailed description
AMX0035 is a proprietary combination of two small molecules, phenylbutyrate (PB) and taurursodiol. The combination of PB and taurursodiol in AMX0035 is intended to block neuronal death and neurotoxic inflammation through simultaneous reduction of endoplasmic reticulum (ER) stress and mitochondrial stress. The pharmacokinetic of AMX0035 has been evaluated in healthy volunteers. This Phase IIa trial is intended to evaluate pharmacokinetic of PB and taurursodiol upon single dose multiple dose administration of AMX0035 in patients with ALS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with AMX0035 | Experimental | Drug: AMX0035Two sequential study period. In Period 1, subject receive AMX0035 daily for approximately 14 days. In Period 2, subjects receive AMX0035 twice a day, morning and evening, for up to 25 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male or female, >=18years of age;
- Diagnosis of sporadic ALS (definite, probable, laboratory probable, possible) made by physician experienced with management of ALS as defined by the World Federation of Neurology revised El Escorial criteria;
- If taking riluzole, must be on a stable dose for >30 days prior to Day 1 and anticipated to remain at that dose until the final study visit.
- If taking edaravone, must be on a stable regimen for > 30 days prior to Day 1 and infusion(s) can be scheduled to be performed at no less than 48 hours prior or after the planned pharmacokinetic and pharmacodynamic (PK/PD) sampling.
- Capable of providing informed consent and following trial procedures;
Key exclusion criteria (59 listed)
- Familial ALS
- Forced vital capacity < 50% (or alternatively SVC) or presence of tracheostomy or under PAV (PAV is defined as more than 22 hours daily of non-invasive mechanical ventilation for more than 7 days);
- Planned elective surgery (such as feeding tube or edaravone access port placement) during the duration of the study;
- History of known allergy to PB or bile salts;
- Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase (AST and/or ALT) > 3 times the upper limit of the normal;
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 |
|---|---|---|
| AMX0035Fixed dose combination of Sodium Phenylbutyrate and taurursodiol. | Drug | Treatment with AMX0035 |
Primary outcome
Blood concentration of PB and taurursodiol
Time frame: Between Day 1 and Day 40
Maximum plasma concentration - Cmax of PB and taurursodiol
Systemic exposure to PB and taurursodiol
Time frame: Between Day 1 and Day 40
Area under the plasma concentration versus time curve - AUC of PB and taurursodiol
3 secondary outcomes
Effect of demographic characteristics on blood concentration of PB and taurursodiol
Time frame: Between Day 1 and Day 40
Effect of demographic characteristics on systemic exposure of PB and taurursodiol
Time frame: Between Day 1 and Day 40
Effect of a fixed dose combination of sodium phenyl butyrate (PB) and taurursodiol on pharmacodynamic activity
Time frame: Between Day 1 and Day 40
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2023.
- Background: PubMed 23448463Cuadrado-Tejedor M, Ricobaraza AL, Torrijo R, Franco R, Garcia-Osta A. Phenylbutyrate is a multifaceted drug that exerts neuroprotective effects and reverses the Alzheimer s disease-like phenotype of a commonly used mouse model. Curr Pharm Des. 2013;19(28):5076-84. doi: 10.2174/1381612811319280006.
- Background: PubMed 18688762Cudkowicz ME, Andres PL, Macdonald SA, Bedlack RS, Choudry R, Brown RH Jr, Zhang H, Schoenfeld DA, Shefner J, Matson S, Matson WR, Ferrante RJ; Northeast ALS and National VA ALS Research Consortiums. Phase 2 study of sodium phenylbutyrate in ALS. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106. doi: 10.1080/17482960802320487.
- Background: PubMed 22438081Nunes AF, Amaral JD, Lo AC, Fonseca MB, Viana RJ, Callaerts-Vegh Z, D'Hooge R, Rodrigues CM. TUDCA, a bile acid, attenuates amyloid precursor protein processing and amyloid-beta deposition in APP/PS1 mice. Mol Neurobiol. 2012 Jun;45(3):440-54. doi: 10.1007/s12035-012-8256-y. Epub 2012 Mar 23.
- Background: PubMed 25443293Dionisio PA, Amaral JD, Ribeiro MF, Lo AC, D'Hooge R, Rodrigues CM. Amyloid-beta pathology is attenuated by tauroursodeoxycholic acid treatment in APP/PS1 mice after disease onset. Neurobiol Aging. 2015 Jan;36(1):228-40. doi: 10.1016/j.neurobiolaging.2014.08.034. Epub 2014 Sep 28.
- Background: PubMed 22974733Lo AC, Callaerts-Vegh Z, Nunes AF, Rodrigues CM, D'Hooge R. Tauroursodeoxycholic acid (TUDCA) supplementation prevents cognitive impairment and amyloid deposition in APP/PS1 mice. Neurobiol Dis. 2013 Feb;50:21-9. doi: 10.1016/j.nbd.2012.09.003. Epub 2012 Sep 10.
- Background: PubMed 21069780Ricobaraza A, Cuadrado-Tejedor M, Marco S, Perez-Otano I, Garcia-Osta A. Phenylbutyrate rescues dendritic spine loss associated with memory deficits in a mouse model of Alzheimer disease. Hippocampus. 2012 May;22(5):1040-50. doi: 10.1002/hipo.20883. Epub 2010 Nov 10.
- Background: PubMed 19145227Ricobaraza A, Cuadrado-Tejedor M, Perez-Mediavilla A, Frechilla D, Del Rio J, Garcia-Osta A. Phenylbutyrate ameliorates cognitive deficit and reduces tau pathology in an Alzheimer's disease mouse model. Neuropsychopharmacology. 2009 Jun;34(7):1721-32. doi: 10.1038/npp.2008.229. Epub 2009 Jan 14.
- Background: PubMed 12627974Rodrigues CM, Sola S, Sharpe JC, Moura JJ, Steer CJ. Tauroursodeoxycholic acid prevents Bax-induced membrane perturbation and cytochrome C release in isolated mitochondria. Biochemistry. 2003 Mar 18;42(10):3070-80. doi: 10.1021/bi026979d.
- Background: PubMed 21272191Wiley JC, Pettan-Brewer C, Ladiges WC. Phenylbutyric acid reduces amyloid plaques and rescues cognitive behavior in AD transgenic mice. Aging Cell. 2011 Jun;10(3):418-28. doi: 10.1111/j.1474-9726.2011.00680.x. Epub 2011 Mar 22.
- Background: PubMed 14583596Wright JM, Zeitlin PL, Cebotaru L, Guggino SE, Guggino WB. Gene expression profile analysis of 4-phenylbutyrate treatment of IB3-1 bronchial epithelial cell line demonstrates a major influence on heat-shock proteins. Physiol Genomics. 2004 Jan 15;16(2):204-11. doi: 10.1152/physiolgenomics.00160.2003.
- Background: PubMed 21372141Zhou W, Bercury K, Cummiskey J, Luong N, Lebin J, Freed CR. Phenylbutyrate up-regulates the DJ-1 protein and protects neurons in cell culture and in animal models of Parkinson disease. J Biol Chem. 2011 Apr 29;286(17):14941-51. doi: 10.1074/jbc.M110.211029. Epub 2011 Mar 3.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AMX0035 in people with ALS, run by Amylyx Pharmaceuticals Inc. at 1 site 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 17 inclusion and 59 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 24 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Blood concentration of PB and taurursodiol (time frame: Between Day 1 and Day 40). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04987671, which also lists 11 linked publications.
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 24 Jul 2023.