ABBV-CLS-7262 for Vanishing White Matter Disease

Recruiting at 6 trial locations

Overseen By Call Center - English

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: AbbVie

Disqualifiers: Respiratory support, Uncontrolled seizures, Pregnancy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 201 weeks

50 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called ABBV-CLS-7262 in adults and children aged 6 years or older who have Vanishing White Matter disease. The study will last for almost two years and will involve frequent medical check-ups to see if the drug helps improve their condition.

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 changes in medication use for managing VWM disease symptoms within 4 weeks before screening are not allowed. It's best to discuss your current medications with the study team.

What makes the drug ABBV-CLS-7262 unique for treating Vanishing White Matter Disease?

There is no specific information available about ABBV-CLS-7262 for Vanishing White Matter Disease, but it may be unique if it targets specific pathways or mechanisms not addressed by existing treatments, similar to how fingolimod works by modulating immune responses and crossing the blood-brain barrier in multiple sclerosis. ^{1 2 3 4 5}

Research Team

ABBVIE INC.

Principal Investigator

AbbVie

Eligibility Criteria

Adults diagnosed with Vanishing White Matter disease, confirmed by a physician and MRI, who have a caregiver to assist them. Participants must be able to consent or have someone who can legally do so on their behalf. They should not have changed VWM medications in the last 4 weeks or received other investigational treatments recently. Adequate contraception is required for sexually active participants.

Inclusion Criteria

I am 18 years old or older.

Signed and dated informed consent provided by the subject, or from a legally authorized representative (LAR) if subject is incapable to consent themselves

I have been diagnosed with VWM disease by a doctor, confirmed through genetic testing and MRI results.

Exclusion Criteria

I am unable to attend all required study visits and procedures.

I haven't changed my VWM disease symptom management medication in the last 4 weeks.

My seizures have not been well-controlled in the past 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Fosigotifator and attend regular visits for medical assessments, blood tests, questionnaires, and evaluation for side effects

201 weeks

Regular visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ABBV-CLS-7262

Trial Overview The trial tests ABBV-CLS-7262's safety and how the body processes it over a period of 96 weeks in patients with Vanishing White Matter disease. It's an open-label study, meaning everyone knows they're getting the drug, without any comparison group.

Participant Groups

Group I: Fosigotifator - Cohort 4 Experimental Treatment 1 Intervention

Cohort 4: VWM children \>= 6 months and \<6 years.

Group II: Fosigotifator - Cohort 3 Experimental Treatment 1 Intervention

Cohort 3: VWM children \>= 6 y and \<12 years.

Group III: Fosigotifator - Cohort 2 Experimental Treatment 1 Intervention

Cohort 2: VWM children\>= 12 y and \<18 years.

Group IV: Fosigotifator - Cohort 1b Experimental Treatment 1 Intervention

Cohort 1b: VWM adults \>= 18 years.

Group V: Fosigotifator - Cohort 1 Experimental Treatment 1 Intervention

Cohort 1: VWM adults \>= 18 years.

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+

Findings from Research

Fingolimod (FTY720) promotes myelination in the central nervous system but, at higher concentrations, it can induce apoptosis in Schwann cells, which are crucial for peripheral nerve myelination.

The study found that FTY720P treatment led to a significant reduction in myelin formation and increased reactive oxygen species in Schwann cells, indicating that while it has therapeutic potential, it may also have detrimental effects on peripheral nerve health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod impedes Schwann cell-mediated myelination: implications for the treatment of immune neuropathies? Köhne, A., Stettner, M., Jangouk, P., et al.[2015]

Fingolimod effectively prevented the progression of experimental autoimmune optic neuritis (EAON) in mice, showing only minimal decline in visual acuity compared to a significant decline in the control group.

The treatment was effective even when started after the onset of optic neuritis symptoms, indicating its potential as a therapeutic option for managing this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Suppression of experimental autoimmune optic neuritis by the novel agent fingolimod. An, X., Kezuka, T., Usui, Y., et al.[2015]

In a study of 42 patients, including 32 with atypical variants of multiple sclerosis (MS) and 10 with acute disseminated encephalomyelitis (ADEM), it was found that levels of specific antibodies (AQP1-IgG, AQP4-IgG, and MOG-IgG) were significantly higher in patients compared to healthy controls, indicating a potential role in the disease process.

A correlation was observed between high levels of MOG-IgG and increased disability (measured by EDSS scores) in patients with Balo's concentric sclerosis (BCS), suggesting that MOG-IgG may be important in understanding the pathology of this atypical MS variant.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical and biochemical characteristics of atypical variants of multiple sclerosis]. Shchepareva, ME., Skalnaya, AA., Zakharova, MN., et al.[2020]

FTY720 (fingolimod) is an oral medication that shows promise in treating both relapsing-remitting and progressive forms of multiple sclerosis (MS) by affecting the immune system and potentially providing neuroprotection within the central nervous system (CNS).

Unlike other MS treatments, FTY720 can cross the blood-brain barrier, allowing it to directly influence brain cells involved in immune regulation and repair, which may enhance its therapeutic effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Central nervous system-directed effects of FTY720 (fingolimod). Miron, VE., Schubart, A., Antel, JP.[2015]

In a study involving 88 healthy volunteers, fingolimod doses of 0.5 mg and 1.25 mg did not significantly alter cerebral blood flow, platelet function, or macular thickness compared to placebo, indicating a safety profile consistent with previous reports.

The changes observed in blood flow and other parameters were within normal variability, suggesting that fingolimod does not have adverse effects on these measures in healthy individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effect of fingolimod (FTY720) on cerebral blood flow, platelet function and macular thickness in healthy volunteers. Ocwieja, M., Meiser, K., David, OJ., et al.[2021]

References

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

Fingolimod impedes Schwann cell-mediated myelination: implications for the treatment of immune neuropathies? [2015]

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

Suppression of experimental autoimmune optic neuritis by the novel agent fingolimod. [2015]

3. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[Clinical and biochemical characteristics of atypical variants of multiple sclerosis]. [2020]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Central nervous system-directed effects of FTY720 (fingolimod). [2015]

5. England pubmed.ncbi.nlm.nih.gov

Effect of fingolimod (FTY720) on cerebral blood flow, platelet function and macular thickness in healthy volunteers. [2021]