Lafora DiseaseLEAPNCT05930223
Intravenous VAL-1221 Lafora Expanded Access Protocol
A study testing VAL-1221 in people with Lafora Disease, run by Parasail, LLC.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 28 years
- Conditions the study is for
- Lafora Disease
- What is being tested?
- VAL-1221
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 28 years
- All sexes
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Intravenous VAL-1221 Lafora Expanded Access Protocol (LEAP)
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
- Being tested
- VAL-1221
- Keywords
- Glycogen Storage Diseases, Polyglucosan Disorders, Progressive Myoclonic Epilepsy
- Medical subject headings
- Lafora Disease, Glycogen Storage Disease, Myoclonic Epilepsies, Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAL-1221Drug
VAL-1221 20mg/kg intravenous (IV) infusion every other week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lafora DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Parasail, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05930223
- Sponsor's protocol ID
- VAL1221-LEAP-01
- Acronym
- LEAP
- Status last verified
- Jun 2023
- First submitted
- 25 Jun 2023
- Last updated
- 5 Jul 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous VAL-1221 Lafora Expanded Access Protocol (LEAP)
- ClinicalTrials.gov ID
- NCT05930223
- Acronym / sponsor ID
- LEAP / VAL1221-LEAP-01
- Conditions
- Lafora Disease
- Keywords
- Glycogen Storage Diseases, Polyglucosan Disorders, Progressive Myoclonic Epilepsy
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Parasail, LLCIndustry (pharma, biotech, devices)
- First posted
- 5 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 12 to 28 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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:
Key exclusion criteria (1 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VAL-1221VAL-1221 20mg/kg intravenous (IV) infusion every other week | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2023.
Related trials for Lafora Disease
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Lafora Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A study testing VAL-1221 in people with Lafora Disease, run by Parasail, LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Dustin Armstrong, PhD ((617) 462-3084, dustin.armstrong@parasailllc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jul 2023.