ALSPhase 1NCT04948645
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis
A Phase 1 study testing Fosigotifator in people with ALS and Amyotrophic Lateral Sclerosis, run by AbbVie at 14 sites in 2 countries.
Trial summary
Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- ALS, Amyotrophic Lateral Sclerosis
- What is being tested?
- Fosigotifator
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 22 Sep 2021to 15 Jul 2025
- Phase
- Phase 1
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must have an identified, reliable caregiver.
- Confirmed diagnosis of Familial Amyotrophic Lateral Sclerosis (ALS) or Sporadic ALS.
- First ALS symptoms occurred <= 36 months before screening.
- Able to swallow solids.
- No known active COVID-19 infection at screening.
- Slow vital capacity (SVC) >= 50% predicted value (for sex, age, ethnic origin, and height) at screening.
- If taking concomitant standard-of-care medications approved for the treatment of ALS (or their components), subjects must be on a stable dose of the medication(s) for >30 days prior to Baseline in order to enter the study. For edaravone, a stable dose is defined by having completed 2 treatment cycles prior to Baseline.
Who cannot join
- History of dementia/severe cognitive problems at screening.
- History of clinically significant medical conditions (other than ALS) or any other reason, including any physical, psychological, or psychiatric condition that, in the opinion of the Investigator, would compromise the safety or interfere with the subject's participation in the study, or would make the subject an unsuitable candidate to receive study drug, or would put the subject at risk by participating in the study.
- History of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease that would preclude administration of Fosigotifator.
- Documented active or suspected malignancy or history of any malignancy within the last 5 years except for successfully treated non-melanoma skin cancer or localized carcinoma in situ of the cervix.
- If female, is known to be pregnant, breastfeeding, considering becoming pregnant, or donating/banking eggs during the study or within 30 days or >5 half-lives (whichever is longer) after the last dose of study drug.
- If male, plans to donate sperm or father a child during the study or within 30 days after the last dose of study drug.
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- Known to have received any investigational product within 30 days or 5 half-lives of the drug (whichever is longer) prior to the first dose of study drug or is currently enrolled in another clinical study.
- History of Fosigotifator use prior to participation in this study.
- Recent (within 6 months prior to Screening) history of drug or alcohol abuse.
- Previous participation in a stem cell clinical study for treatment of ALS.
- Current or anticipated use of diaphragmatic pacing during the study period.
- Tracheostomy or use of non-invasive ventilatory support >= 22 hours a day.
Trial locations and contacts
14 sites in 2 countries.
14 sites in 2 countries
United States7 sites
Johns Hopkins ALS Clinical Trials Unit
Baltimore, Maryland, 21205
Healey & AMG Center for ALS Research
Boston, Massachusetts, 02114
UC Irvine Health ALS and Neuromuscular Center
Irvine, California, 92868
Mayo Clinic
Jacksonville, Florida, 32224
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Mayo Clinic
Rochester, Minnesota, 55905
Forbes Norris MDA/ALS Research and Treatment Center
San Francisco, California, 94109
Canada7 sites
University of Calgary - Heritage Medical Research Clinic
Calgary, Alberta, T2V1P9
University of Alberta
Edmonton, Alberta, T6G 2G3
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E38 0C7
London Health Sciences Centre
London, Ontario, N6A585
Centre Hospitalier de l'Universite de Montreal (CHUM)
Montreal, Quebec, H2L 4M1
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
Sunnybrook Research Institute
Toronto, Ontario, M4N 3M5
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, and Pharmacokinetics Following Multiple Doses of Fosigotifator in Subjects With Amyotrophic Lateral Sclerosis Followed by an Active Treatment Extension
Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive Fosigotifator.
- Conditions
- ALS, Amyotrophic Lateral Sclerosis
- Being tested
- Fosigotifator, Placebo
- Keywords
- Fosigotifator, ABBV-CLS-7262
- 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.
FosigotifatorDrug
Also called ABBV-CLS-7262
Oral
PlaceboDrug
Oral
| Study group | Role | What they receive |
|---|---|---|
| Fosigotifator Low Dose | Active comparator | Not described Receives: Drug: Fosigotifator |
| Fosigotifator Medium Dose | Active comparator | Not described Receives: Drug: Fosigotifator |
| Fosigotifator High Dose | Active comparator | Not described Receives: Drug: Fosigotifator |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Measured: Baseline Up to Approximately Day 28
Pharmacokinetics
Maximum Plasma Concentration [Cmax]
Measured: Baseline Up to Approximately Day 28
Pharmacokinetics
Area Under the Curve [AUC]
Measured: Baseline Up to Approximately Day 28
Secondary outcomes (2)
CSF Pharmacokinetics
Concentration at steady state in CSF
Measured: Baseline Up to Approximately Day 28
Safety and Tolerability
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Measured: Baseline Up to Approximately Week 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2021
- Study start22 Sep 2021
- Primary completion15 Jul 2025
- Study completion15 Jul 2025
Why it stopped: Company Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSStudies of the same condition, open and under way.
- More trials run by AbbVieThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- AbbVie
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Calico Life Sciences LLC
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04948645
- Sponsor's protocol ID
- M20-405
- Status last verified
- Jul 2025
- First submitted
- 23 Jun 2021
- Last updated
- 1 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, and Pharmacokinetics Following Multiple Doses of Fosigotifator in Subjects With Amyotrophic Lateral Sclerosis Followed by an Active Treatment Extension
- ClinicalTrials.gov ID
- NCT04948645
- Acronym / sponsor ID
- M20-405
- Conditions
- ALS, Amyotrophic Lateral Sclerosis
- Keywords
- Fosigotifator, ABBV-CLS-7262
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company Decision
- Enrolment
- 31 participants (actual)
- Lead sponsor
- AbbVieIndustry (pharma, biotech, devices)
- Collaborators
- Calico Life Sciences LLC
- Study start
- 22 Sep 2021 (actual)
- Primary completion
- 15 Jul 2025 (actual)
- Study completion
- 15 Jul 2025 (actual)
- First posted
- 2 Jul 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.
Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive Fosigotifator.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Fosigotifator Low Dose | Active comparator | Drug: Fosigotifator |
| Fosigotifator Medium Dose | Active comparator | Drug: Fosigotifator |
| Fosigotifator High Dose | Active comparator | Drug: Fosigotifator |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Must have an identified, reliable caregiver.
- Confirmed diagnosis of Familial Amyotrophic Lateral Sclerosis (ALS) or Sporadic ALS.
- First ALS symptoms occurred <= 36 months before screening.
- Able to swallow solids.
- No known active COVID-19 infection at screening.
Key exclusion criteria (12 listed)
- History of dementia/severe cognitive problems at screening.
- History of clinically significant medical conditions (other than ALS) or any other reason, including any physical, psychological, or psychiatric condition that, in the opinion of the Investigator, would compromise the safety or interfere with the subject's participation in the study, or would make the subject an unsuitable candidate to receive study drug, or would put the subject at risk by participating in the study.
- History of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease that would preclude administration of Fosigotifator.
- Documented active or suspected malignancy or history of any malignancy within the last 5 years except for successfully treated non-melanoma skin cancer or localized carcinoma in situ of the cervix.
- If female, is known to be pregnant, breastfeeding, considering becoming pregnant, or donating/banking eggs during the study or within 30 days or >5 half-lives (whichever is longer) after the last dose of study drug.
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 |
|---|---|---|
| FosigotifatorAlso: ABBV-CLS-7262Oral | Drug | Fosigotifator High Dose, Fosigotifator Low Dose, Fosigotifator Medium Dose |
| PlaceboOral | Drug | Placebo |
Primary outcome
Safety and Tolerability
Time frame: Baseline Up to Approximately Day 28
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Pharmacokinetics
Time frame: Baseline Up to Approximately Day 28
Maximum Plasma Concentration [Cmax]
Pharmacokinetics
Time frame: Baseline Up to Approximately Day 28
Area Under the Curve [AUC]
2 secondary outcomes
CSF Pharmacokinetics
Time frame: Baseline Up to Approximately Day 28
Safety and Tolerability
Time frame: Baseline Up to Approximately Week 156
Results and safety
The study was stopped early (reason given: Company Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2025.
- 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.
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fosigotifator in people with ALS and Amyotrophic Lateral Sclerosis, run by AbbVie at 14 sites in 2 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 7 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: stopped early, as of the sponsor's last update on 1 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada. 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 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.
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 was stopped early (reason given: Company Decision). 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?
Safety and Tolerability (time frame: Baseline Up to Approximately Day 28). The study lists 3 primary outcomes in all. 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 1 Aug 2025.