Intravenous VAL-1221 Lafora Expanded Access Protocol

• Conditions: Lafora Disease

• Registration Number: NCT05930223
• Lead Sponsor: Parasail, LLC

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2023-06
• Study First Submitted: 2023-06-25
• Study First Submitted QC: 2023-06-25
• Last Update Submitted: 2023-06-25
• Study First Posted: 2023-07-05
• Last Update Posted: 2023-07-05

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified