Los Angeles, CANCT07181837Now EnrollingIRB Ready

Angelman Syndrome Clinical Trial in Los Angeles, CA

Access cutting-edge angelman syndrome treatment through this phase 1/2 clinical trial at a research site in Los Angeles. Study-provided care at no cost to qualified participants.

Sponsored by MavriX Bio, LLC

Quick Self-Assessment

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Expert Care in Los Angeles

Access angelman syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related angelman syndrome treatment provided free

Apply for This Los Angeles Location

Check if you qualify for this angelman syndrome clinical trial in Los Angeles, CA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Los Angeles

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Los Angeles site if eligible
  4. 4Begin participation

About This Angelman Syndrome Study in Los Angeles

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, uniparental disomy, or imprinting center defect genotypes.

Sponsor: MavriX Bio, LLC

Who Can Participate

Inclusion Criteria

The participant's parent/legal guardian must provide written informed consent.
Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
Uniparental disomy
Imprinting center defect
The participant must be 18 to 50 years of age, inclusive (for adult participants), or 4 to 8 years of age, inclusive (for pediatric participants), at Screening.
The participant must have the ability to ambulate independently.
The participant must be on stable antiepileptic medications (with no changes within 1 month prior to the Screening visit, except for weight associated dose adjustments). Key

Exclusion Criteria

Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
Laboratory abnormalities including but not limited to:
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
Total and/or fractionated bilirubin (direct and/or indirect) \> ULN
Gamma-glutamyl transferase (GGT) \> ULN
Estimated glomerular filtration rate (eGFR) below the lower limit of normal (LLN) for age
Hemoglobin \< 8 g/dL
White blood cell (WBC) count outside the normal range for age
Platelet count \< LLN
Partial thromboplastin time (PTT) outside the reference range
PT/International normalized ratio (INR) outside the reference range
Any known history and/or family history of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) or multisystem inflammatory syndrome (MIS).
Any known history and/or family history of disordered complement function and/or complement gene mutation(s).
History of systemic lupus erythematous, Still's disease, rheumatoid arthritis, and/or other severe autoimmune conditions per judgment of the Investigator.
Any known history of thrombotic microangiopathy (TMA)/microangiopathic hemolytic anemia, or hypercoagulable conditions including, but not limited to, disseminated intravascular coagulation (DIC), deep venous thrombosis, and pulmonary embolism.
Current therapy with high dose immunosuppressants.
Prior or current treatment with an investigational drug within 6 months or 5-half-lives of the hospital admission whichever is longer.
Prior treatment with an antisense oligonucleotide within 1 year of hospital admission.
A history of gene therapy administration.
Any contraindication to ICM administration procedure, including contraindications to imaging, contrast use, anesthesia, or any condition that would increase the risk of adverse outcomes from the ICM procedure.
Any contraindication to glucocorticoid use

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Los Angeles?

Yes, this clinical trial (NCT07181837) has an active research site in Los Angeles, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Angelman Syndrome Treatment Options in Los Angeles, CA

If you're searching for angelman syndrome treatment options in Los Angeles, CA, this clinical trial (NCT07181837) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Los Angeles research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced angelman syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all angelman syndrome clinical trials near you to find additional studies recruiting in your area.

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