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

310 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called ABBV-CLS-7262 to see if it can help people with ALS. ALS is a serious disease with few treatments, so new options are needed. The drug aims to improve symptoms or slow down the disease.

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.

What data supports the effectiveness of the drug ABBV-CLS-7262 for ALS?

There is no direct data on ABBV-CLS-7262 for ALS, but similar drugs like AMX0035 have shown therapeutic effects in clinical trials and have been approved for ALS treatment, suggesting potential for new drugs in this area. ^{1 2 3 4 5}

Research Team

Merit Cudkowicz, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Clinical trials for neuroprotection in ALS. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. [2022]

3. England 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. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Edaravone efficacy in amyotrophic lateral sclerosis with reduced forced vital capacity: Post-hoc analysis of Study 19 (MCI186-19) [clinical trial NCT01492686]. [2022]

5. Switzerland pubmed.ncbi.nlm.nih.gov

New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. [2022]