MVX-220 for Angelman Syndrome

(ASCEND-AS Trial)

Recruiting at 1 trial location

Overseen By MavriX Bio, LLC

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: MavriX Bio, LLC

Must be taking: Antiepileptic medications

Must not be taking: High dose immunosuppressants

Disqualifiers: Autoimmune conditions, Thrombotic microangiopathy, others

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called MVX-220 for individuals with Angelman syndrome, a genetic disorder affecting the nervous system. Researchers aim to determine if this treatment is safe and effective for those with specific genetic variations of the syndrome, such as a missing or defective UBE3A gene. Participants may qualify if they have Angelman syndrome, can walk independently, and have stable epilepsy medications for at least a month. The study includes both children and adults, with separate groups for each. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Do I have to stop taking my current medications for the trial?

The trial requires that participants be on stable antiepileptic medications, meaning you should not change these medications within one month before the screening visit. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that MVX-220 is likely to be safe for humans?

Research shows that MVX-220, a new gene therapy for Angelman syndrome, undergoes careful study to ensure safety. In earlier studies, both children and adults received this treatment. Although detailed safety information from these studies remains limited, the FDA Fast Track status for MVX-220 suggests promising evidence regarding its safety. This status often indicates that early studies have shown it to be generally safe, with manageable side effects. However, it is important to remember that this research continues, and while researchers remain hopeful, more results are needed to confirm its safety and effectiveness. ^{1 2 3 4 5}

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

Researchers are excited about MVX-220 for Angelman Syndrome because it offers a new approach by delivering the treatment directly into the cerebrospinal fluid via a single dose injection into the cisterna magna. This method aims to target the central nervous system more directly than traditional treatments like behavioral therapies and symptomatic medications, which can be less specific. Additionally, MVX-220 may introduce a novel mechanism of action that could potentially address the underlying causes of Angelman Syndrome, rather than just managing symptoms. This direct and potentially more effective approach fuels optimism about providing a significant breakthrough for patients.

What evidence suggests that MVX-220 might be an effective treatment for Angelman syndrome?

Research has shown that MVX-220 is a promising new treatment for Angelman Syndrome, a condition with no current treatments that alter the disease's course. MVX-220 uses a harmless virus to deliver a working copy of the UBE3A gene, which is missing or malfunctioning in people with Angelman Syndrome. This treatment aims to restore the UBE3A gene's function, crucial for normal brain development and function. Although research in humans continues, the FDA has granted MVX-220 fast track status, highlighting its potential to meet this critical medical need. Early results suggest that this treatment could improve Angelman Syndrome symptoms. Participants in this trial will receive a single dose of MVX-220 via intra cisterna magna injection, with different cohorts for adults, children, and an optional mixed cohort. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for children and adults with Angelman syndrome aged 4-8 or 18-50. Participants must have a specific genetic cause of the syndrome, be able to walk on their own, and be on stable epilepsy medication. Consent from a parent or guardian is required for minors.

Inclusion Criteria

I can walk on my own without help.

My parent or guardian has agreed to the study.

Key

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of MVX-220 gene therapy administered by intra-cisterna magna injection

1 day

1 visit (in-person)

Initial Follow-up

Participants are monitored for safety and efficacy for an initial 2-year period post-treatment

104 weeks

Regular visits (frequency not specified)

Extended Follow-up

Participants transition to a less frequent monitoring schedule for an additional 3 years

156 weeks

What Are the Treatments Tested in This Trial?

Interventions

• MVX-220

Trial Overview The study tests MVX-220 gene therapy's safety and effectiveness in treating Angelman syndrome caused by UBE3A gene deletion, uniparental disomy, or imprinting center defects.

How Is the Trial Designed?

Group I: Cohort 3: Optional cohort, adults and children ages 4-50 Experimental Treatment 1 Intervention

MVX-220, single dose intra cisterna magna injection

Group II: Cohort 2: Children ages 4-8 Experimental Treatment 1 Intervention

MVX-220, single dose intra cisterna magna injection

Group III: Cohort 1: Adults ages 18-50 Experimental Treatment 1 Intervention

MVX-220, single dose intra cisterna magna injection

Who Is Running the Clinical Trial?

MavriX Bio, LLC

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07181837

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in ... The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, ...

2. angelmansyndromenews.com angelmansyndromenews.com/news/mvx-220-angelman-syndrome-earns-fda-fast-track-status/

MVX-220 for Angelman syndrome earns FDA fast track status The Phase 1/2 ASCEND-AS trial (NCT07181837) will evaluate the safety and efficacy of MVX-220 in 12 children and adults with Angelman syndrome.

3. cureangelman.org cureangelman.org/articles/mavrix-mvx-220-fast-track-angelman

MavriX Bio MVX-220 Update ... granted Fast Track designation for MVX-220, an investigational adeno-associated virus (AAV) gene therapy for the treatment of Angelman syndrome.

4. prnewswire.com prnewswire.com/news-releases/mavrix-bio-announces-first-patient-dosed-in-ascend-as-trial-of-mvx-220-investigational-gene-therapy-for-angelman-syndrome-302607143.html

MavriX Bio Announces First Patient Dosed in ASCEND-AS ... Angelman syndrome affects approximately 1 in 12,000–20,000 individuals worldwide and currently has no approved disease-modifying treatments.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07181837?cond=angelman&rank=8

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in ... The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, ...

6. cureangelman.org cureangelman.org/current-pipeline/gemma-aav-gene-therapy

MVX-220 MVX-220. MavriX Bio is exploring an investigational Adeno-Associated Virus Gene Therapy (AAV-GT) approach to treating Angelman syndrome using a vector developed ...

7. firstwordpharma.com firstwordpharma.com/story/6094777

MavriX Bio Receives FDA Fast Track Designation for MVX ... The first-in-human study ASCEND-AS will evaluate the safety, tolerability, and efficacy of MVX-220 in adult and pediatric individuals living ...