drugset / Trial / NCT05930223
Intravenous VAL-1221 Lafora Expanded Access Protocol
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.
Timeline
- Start
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- Primary completion
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- Completion
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Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CLERVONAFUSP ALFA | Protein / enzyme biologic | 20 mg/kg | Intravenous |