Lafora Disease
Conditions
Keywords
Glycogen Storage Diseases, Polyglucosan Disorders, Progressive Myoclonic Epilepsy
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.
Interventions
DRUGVAL-1221
VAL-1221 20mg/kg intravenous (IV) infusion every other week
Sponsors
Parasail, LLC
Eligibility
Sex/Gender
ALL
Age
12 Years to 28 Years
Inclusion criteria
* 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
Exclusion criteria
* Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the participant unsafe for inclusion or could interfere with the participant participating in or completing the protocol
Contacts
Primary ContactDustin Armstrong, PhD
Outcome results
None listed