Intravenous VAL-1221 Lafora Expanded Access Protocol

NCT05930223 | Unknown

• 1 other identifier: VAL1221-LEAP-01
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Only qualified physicians treating a Lafora Disease patient may request VAL-1221 Expanded Access treatment under the Parasail Lafora Expanded Access Protocol (LEAP). According to FDA guidance, this protocol can enroll up to 10 patients. However, individual patient inclusion will ultimately be dependent on protocol eligibility, site geography, treatment requirements, and available supply of the investigational therapy. Inquiring physicians can submit a request by contacting the Central Contact personnel listed below in Contacts/Locations.

Conditions

Lafora Disease

Keywords

Glycogen Storage Diseases; Polyglucosan Disorders; Progressive Myoclonic Epilepsy

Interventions

VAL-1221 DRUG

VAL-1221 20mg/kg intravenous (IV) infusion every other week

Eligibility Criteria

• Age: 12 Years - 28 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Documented genetic diagnosis of Lafora Disease (LD) based on likely pathogenic or pathogenic variants in both alleles of either the EPM2A or the EPM2B gene
• Mid-stage in evolution of LD between 12 and 28 years of age
• Able and willing to comply with the protocol, including travel to Protocol Center, procedures, measurements and visits, including:
• Adequately supportive psychosocial circumstances, in the opinion of the Investigator
• Caregiver/trial partner committed to facilitate patient's involvement in the study who is reliable, competent, at least 18 years of age

Sponsors & Collaborators

• Parasail, LLC: lead

MeSH Terms

Conditions

Lafora Disease; Glycogen Storage Disease; Myoclonic Epilepsies, Progressive

Condition Hierarchy (Ancestors)

Epilepsies, Myoclonic; Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Dustin Armstrong, PhD

CONTACT

(617) 462-3084; dustin.armstrong@parasailllc.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2023
• First Posted: July 5, 2023
• Last Updated: July 5, 2023

Record last verified: 2023-06