Fosigotifator for Cree Leukoencephalopathy

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Genevieve Bernard

Disqualifiers: Ineligible for other fosigotifator trials

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new medication, fosigotifator, to determine if it can help people with Cree Leukoencephalopathy (CLE), a rare brain disease that disrupts nerve communication and causes severe neurological issues. The study aims to discover whether fosigotifator can slow or stop damage to the brain's white matter, potentially improving symptoms and quality of life. It is open to individuals with a confirmed CLE diagnosis who are either pre-symptomatic or in the early stages and have no other treatment options. As an unphased trial, participants can contribute to groundbreaking research that could lead to new treatment options.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research has shown that fosigotifator (also known as ABBV-CLS-7262) is under evaluation for safety in treating Vanishing White Matter (VWM) disease, which shares the same genetic cause as Cree Leukoencephalopathy (CLE). Current studies, including one involving multiple research centers, are assessing how well patients tolerate fosigotifator.

Participants in these studies have shown varying levels of tolerance to the medication. While specific side effects have not been detailed, the testing of fosigotifator in humans suggests researchers consider it potentially safe. One study involves both doctors and patients knowing the treatment being administered, which helps researchers gather more information on the drug's long-term effects.

For those considering joining a trial, it may be reassuring to know that fosigotifator is already being tested in humans for similar conditions. However, discussing any concerns or questions with the trial team is crucial to understanding the potential risks and benefits for your situation. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Fosigotifator is unique because it offers a new approach to treating Cree Leukoencephalopathy, a rare genetic disorder. Unlike existing treatments that primarily focus on managing symptoms, Fosigotifator targets the underlying metabolic dysfunction caused by the disease. Researchers are excited about its potential to directly address the root cause of the condition, which could lead to more effective management and improved quality of life for patients. This innovative mechanism of action sets it apart from current options and provides hope for a more comprehensive treatment strategy.

What evidence suggests that fosigotifator might be an effective treatment for Cree Leukoencephalopathy?

Research has shown that Fosigotifator (FGT) is under investigation as a potential treatment for Vanishing White Matter (VWM) disease, which resembles Cree Leukoencephalopathy (CLE). Both VWM and CLE involve the breakdown of the brain's white matter, leading to serious neurological issues. Fosigotifator targets the eIF2B5 gene, linked to both VWM and CLE. This treatment aims to slow or stop white matter damage, potentially reducing symptoms and improving quality of life. Although research is ongoing, early studies are examining its safety and effectiveness for similar conditions. The hope is that FGT can offer new treatment options for those affected by these rare diseases. Participants in this trial will receive Fosigotifator in a single patient trial to further evaluate its potential benefits for Cree Leukoencephalopathy. ^{1 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for patients at least 1 month old, weighing over 5kg, with a confirmed diagnosis of Cree Leukoencephalopathy (CLE). They should be in the early stages of the disease and not eligible for other fosigotifator trials. Legal guardians must consent.

Inclusion Criteria

I am in the early stages of my illness, with few or no symptoms.

My legal guardians have agreed to the treatment plan.

My diagnosis of CLE is confirmed through genetic testing.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patient receives fosigotifator (FGT) to evaluate its potential benefits and risks in slowing or halting white matter degeneration

2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Fosigotifator

Trial Overview

The trial tests an investigational drug called fosigotifator on a patient with CLE to see if it can slow down or stop brain deterioration by targeting the EIF2B5 gene mutation responsible for the disease.

How Is the Trial Designed?

Group I: Single patient trial Experimental Treatment 1 Intervention

Fosigotifator

Who Is Running the Clinical Trial?

Genevieve Bernard

Lead Sponsor

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07300397

Single Patient Investigational Treatment for Cree ...

Fosigotifator (FGT, ABBV-CLS-7262) has been developed and its safety and efficacy are currently being studied in a multi-center Phase 1b/2 clinical trial for ...

2.

withpower.com

withpower.com/trial/early-phase-1-leukoencephalopathies-10-2024-ea0e5

Fosigotifator for Cree Leukoencephalopathy

Research has shown that Fosigotifator (FGT) is under investigation for its safety and efficacy in treating Vanishing White Matter (VWM) disease, which resembles ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05757141

Study Details | NCT05757141 | An Open-Label Exploratory ...

Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.

4.

app.trialscreen.org

app.trialscreen.org/trials/early-phase-1-research-single-patient-treatment-cree-leukoencephalopathy-trial-nct07272525

Research Study for Single-Patient Treatment of Cree ...

Fosigotifator (FGT, ABBV-CLS-7262) has been developed and its safety and efficacy are currently being studied in a multi-center Phase 1b/2 ...

5.

trial.medpath.com

trial.medpath.com/drug/86892eab963fd37d/abbv-cls-7262

ABBV-CLS-7262 - MedPath

Fosigotifator (ABBV-CLS-7262): A Comprehensive Monograph on an eIF2B Activator in Clinical Development for Neurodegenerative and Psychiatric Disorders.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12615013/

Consensus-Based Expert Recommendations for Diagnosis ...

We listed all known pathogenic and likely pathogenic variants in EIF2B1-5 genes reported since the 2010 last variant update up to October 2024, ...

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06594016

NCT06594016 | Expanded Access to Fosigotifator

Also called a data safety and monitoring board, or DSMB. Early Phase 1 ... Cree Leukoencephalopathy. Additional Relevant MeSH Terms. Brain Diseases.

8.

ctv.veeva.com

ctv.veeva.com/study/phase-1b-study-to-investigate-abbv-cls-7262s-safety-tolerability-pharmacokinetics-in-vanishing-w

An Open-Label Exploratory Study of Fosigotifator in ...

This is a 201-week, open-label, multiple cohort study enrolling adults, pediatric and infant participants with Vanishing White Matter disease.