ABBV-CLS-7262 for ALS

Recruiting at 15 trial locations

Overseen By Calico Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: AbbVie

Must be taking: Standard ALS medications

Disqualifiers: Dementia, Malignancy, Pregnancy, Drug abuse, others

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

31 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new drug called Fosigotifator (also known as ABBV-CLS-7262) to determine its potential in treating Amyotrophic Lateral Sclerosis (ALS), a condition that affects the nervous system and weakens muscles. Participants will begin with a 4-week period where some receive Fosigotifator at different doses, while others receive a placebo. This is followed by a longer phase where all participants receive the drug. The study seeks individuals who have experienced ALS symptoms within the last three years and can still swallow solids. The goal is to assess the safety of Fosigotifator and its ability to improve ALS symptoms. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

If you are taking standard ALS medications, you must be on a stable dose for more than 30 days before starting the trial. This means you can continue your current medications if they are stable.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that fosigotifator, the drug under study for ALS (Amyotrophic Lateral Sclerosis), is safe and well-tolerated. Previous studies found no major safety differences among low, medium, and high doses, indicating that participants did not experience serious safety issues regardless of the dosage. Some side effects appeared after treatment began, but they were not severe. This suggests that fosigotifator has a good safety record based on current data. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Researchers are excited about Fosigotifator for ALS because it offers a new approach that could potentially target the disease differently from existing treatments like Riluzole and Edaravone. While most current therapies focus on slowing disease progression with limited mechanisms, Fosigotifator aims to tackle the disease at a cellular level, possibly enhancing energy production and reducing nerve cell damage. This novel mechanism of action makes Fosigotifator a promising candidate in the fight against ALS, sparking hope for more effective management of the condition.

What evidence suggests that Fosigotifator might be an effective treatment for ALS?

Research has shown that fosigotifator activates a protein called eIF2B, which helps nerve cells produce proteins normally, even under stress. This action might protect nerve cells in people with Amyotrophic Lateral Sclerosis (ALS). However, in the HEALEY ALS Platform Trial, fosigotifator did not slow ALS progression as expected. While the drug's mechanism is promising, it has not yet proven effective in treating ALS based on current evidence. More studies, including trials testing different doses of fosigotifator, are needed to determine its potential benefits for people with ALS. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for people with ALS who've had symptoms start within the last 3 years and can swallow solids. They need a reliable caregiver, no recent drug abuse, or severe cognitive issues. Participants should not be using certain ALS treatments like diaphragmatic pacing or have been in stem cell studies for ALS.

Inclusion Criteria

I can swallow solid foods.

My first ALS symptoms appeared less than 3 years ago.

I do not have an active COVID-19 infection.

Exclusion Criteria

You are currently using or planning to use a diaphragmatic pacing device during the study.

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 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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo a 4-week, randomized, double-blind, placebo-controlled study to assess safety, tolerability, and pharmacokinetics of Fosigotifator

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Active Treatment Extension

Participants receive Fosigotifator for up to 152 weeks in an open-label extension to further assess long-term safety and tolerability

152 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ABBV-CLS-7262
• Placebo

Trial Overview ABBV-CLS-7262, a new potential treatment for ALS, is being tested against a placebo over up to 156 weeks. Initially, there's a 4-week double-blind phase where participants won't know if they're getting the real drug or placebo, followed by an active treatment extension where all get ABBV-CLS-7262.

How Is the Trial Designed?

Group I: Fosigotifator Low Dose Active Control 1 Intervention

Group II: Fosigotifator High Dose Active Control 1 Intervention

Group III: Fosigotifator Medium Dose Active Control 1 Intervention

Group IV: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Top Products

Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

Calico Life Sciences LLC

Industry Sponsor

Trials

11

Recruited

960+

Published Research Related to This Trial

The ALSUMMIT trial is a phase III study involving 115 participants to evaluate the long-term efficacy and safety of intrathecal autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) in patients with ALS, comparing a single-cycle treatment with additional booster injections against a control group.

Initial results from a previous study indicated that a single cycle of BM-MSC treatment was safe and provided benefits for up to 6 months, but the ALSUMMIT trial aims to determine if the addition of booster injections can enhance long-term outcomes beyond this period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Lenzumestrocel (Neuronata-R® inj.) in patients with amyotrophic lateral sclerosis (ALSUMMIT study): study protocol for a multicentre, randomized, double-blind, parallel-group, sham procedure-controlled, phase III trial. Nam, JY., Lee, TY., Kim, K., et al.[2023]

A review of 53 new drugs evaluated in clinical trials for ALS from 2020 to 2022 identified five promising candidates, including AMX0035, which has recently been approved by the FDA, marking it as the third drug approved for ALS treatment after riluzole and edaravone.

The review categorized these drugs into eight mechanistic groups, highlighting a diverse range of approaches being explored to combat ALS, with 13 drugs currently in phase 3 trials, indicating ongoing efforts to find more effective treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. Jiang, J., Wang, Y., Deng, M.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04948645

NCT04948645 | A Phase 1 Study to Investigate the Safety ... 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 ...

2. abbvieclinicaltrials.com abbvieclinicaltrials.com/study/?id=M20-405

A Phase 1 Study to Investigate the Safety and ... Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study.

3. massgeneral.org massgeneral.org/neurology/als/news/healeyamg-announces-platform-trial-update-fosigotifator

Sean M. Healey & AMG Center Announces Update in ALS ... We remain deeply committed to fully understanding the effects of fosigotifator in ALS, and will further evaluate the data before determining ...

4. calicolabs.com calicolabs.com/press/calico-statement-on-fosigotifator-in-healey-als-platform-trial/

Calico Provides Update on Fosigotifator in HEALEY ALS ... By activating eIF2B, fosigotifator could attenuate the ISR pathway and restore normal protein production in stressed nerve cells, potentially ...

5. trial.medpath.com trial.medpath.com/drug/report/86892eab963fd37d

ABBV-CLS-7262 | Advanced Drug Monograph - MedPath The largest and most advanced study, Regimen F of the HEALEY ALS Platform Trial, failed to meet its primary endpoint of slowing disease ...

6. en.wikipedia.org en.wikipedia.org/wiki/Fosigotifator

Fosigotifator Fosigotifator is an experimental small-molecule developed by AbbVie, which is running clinical trials to determine if the drug can treat amyotrophic lateral ...