ABBV-CLS-7262 for ALS

Overseen By Healey Center for ALS at Massachusetts General Hospital

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Merit E. Cudkowicz, MD

Must not be taking: Cytochrome P450 inhibitors

Disqualifiers: ECG abnormalities, Lab abnormalities, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if the drug ABBV-CLS-7262 (also known as Fosigotifator) is safe and effective for treating ALS, a condition affecting nerve cells in the brain and spinal cord, leading to loss of muscle control. The study includes two groups testing different doses of the drug and a group receiving a placebo (a substance with no active drug). It is part of a larger ongoing effort to explore various treatments for ALS. Individuals diagnosed with ALS who experience symptoms affecting daily life may be suitable candidates for this trial. As a Phase 2, Phase 3 trial, this research evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering hope for effective ALS treatment.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that certain drugs affecting liver enzymes might not be allowed. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that ABBV-CLS-7262 is likely to be safe for humans?

Research shows that ABBV-CLS-7262, also known as fosigotifator, is under evaluation for safety in treating ALS, a disease that weakens muscles. Although detailed safety information remains limited, studies suggest that researchers are considering fosigotifator for long-term use in ALS patients, with ongoing checks for side effects. The trial is in an early but promising stage, indicating that some information on patient tolerance has been gathered. As it progresses to later testing phases, confidence in its safety typically increases compared to earlier phases. Discussing participation in trials with a healthcare provider is crucial to understanding personal risks and benefits. ^{1 2 3 4 5}

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

Researchers are excited about ABBV-CLS-7262 for ALS because it offers a novel approach to tackling this challenging condition. Unlike current treatments such as riluzole and edaravone, which have limited effects on disease progression, ABBV-CLS-7262 targets specific cellular pathways believed to be involved in ALS. This new mechanism of action could potentially slow down or even alter the course of the disease more effectively. By introducing innovative strategies to combat ALS, ABBV-CLS-7262 holds promise for making a significant impact on patients' lives.

What evidence suggests that this trial's treatments could be effective for ALS?

Research shows that ABBV-CLS-7262, also known as fosigotifator, is under investigation as a potential treatment for ALS (amyotrophic lateral sclerosis) in this trial. This drug activates a protein called eIF2B, which aids cells in responding to stress. Participants may receive one of two different doses of ABBV-CLS-7262 or a matching placebo. Early studies suggest that healthy volunteers tolerated this drug well. Although direct evidence of its effectiveness for ALS remains limited, its mechanism offers a promising approach to tackling the disease. Further research is necessary to fully understand its potential in treating ALS. ^{2 3 4 5 6}

Who Is on the Research Team?

Merit Cudkowicz, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for people with ALS, also known as Lou Gehrig's disease. Participants must meet the general criteria set by the master protocol (NCT04297683). They can't join if they have significant heart rhythm problems, abnormal lab test results, or are taking certain drugs that affect enzyme activity in the body.

Inclusion Criteria

No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria

I am not taking any drugs that affect my body's drug processing enzymes.

You have any important heart rhythm problems.

The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Baseline Assessment

Participants complete a baseline assessment before randomization

1 week

1 visit (in-person)

Treatment

Participants receive either active ABBV-CLS-7262 or matching placebo

24 weeks

Regular visits (frequency not specified)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ABBV-CLS-7262

Trial Overview The HEALEY ALS Platform Trial is testing ABBV-CLS-7262 at two different doses to see if it's safe and effective for treating ALS. Some participants will receive a placebo instead to compare outcomes.

How Is the Trial Designed?

Group I: ABBV-CLS-7262 Dose 2 Experimental Treatment 1 Intervention

Group II: ABBV-CLS-7262 Dose 1 Experimental Treatment 1 Intervention

Group III: Matching Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Merit E. Cudkowicz, MD

Lead Sponsor

Trials

8

Recruited

2,900+

Calico Life Sciences LLC

Industry Sponsor

Trials

11

Recruited

960+

Published Research Related to This Trial

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]

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]

Citations

1. 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.

2. 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.

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

Sean M. Healey & AMG Center Announces Update in ALS ... ... ALS Platform Trial evaluating fosigotifator (ABBV-CLS-7262) in people living with amyotrophic lateral sclerosis (ALS). Fosigotifator, an ...

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

ABBV-CLS-7262 | Advanced Drug Monograph - MedPath Comprehensive analysis about ABBV-CLS-7262, including its mechanisms, clinical uses, and therapeutic considerations.

5. bioworld.com bioworld.com/articles/707776-eif2b-activator-abbv-cls-7262-shows-promise-in-vanishing-white-matter-disease

eIF2B activator ABBV-CLS-7262 shows promise in ... The compound is currently in phase I trials for VWM and was well tolerated following dosing for up to 14 days in healthy volunteers.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05740813

HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262 Regimen F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS. Detailed Description. The HEALEY ALS Platform ...