patients with mid- to late stage Lafora Desease MedDRA version: 20.0 Level: PT Classification code 10054030 Term: Lafora's myoclonic epilepsy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10054030 Term: Lafora's myoclonic epilepsy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10054030 Term: Lafora's myoclonic epilepsy System Organ Class:
Conditions
Interventions
Product Name: VAL-1221
Product Code: [VAL-1221]
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Clervonafusp alfa
CAS Number: 2145123-44-8
Current Sponsor code: VAL-1221
Concentration
Sponsors
AZIENDA USL DI BOLOGNA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented genetic diagnosis of LD based on pathogenic variants in both alleles of either the EPM2A or the NHLRC1 gene 2. Between 12 and 28 years of age 3. Mid- or late-stage in the LD evolution (LD progression scale 2-3) 4. Able and willing to comply with the study protocol including: a. Caregiver/trial partner committed to facilitating patient's involvement in the study who is reliable, competent, and at least 18 years of age. b. Adequate cognitive capacity to participate in neuropsychological testing adjusted to their level of functioning Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Any known genetic abnormality confounding the phenotype 2.Subjects a. with complete absence of speech OR b. unable to perform any activities of daily living OR c. completely bedridden 3.Current participation in an interventional or therapeutic study 4. Pregnancy 5.Receiving an investigational drug within 90 days of the Baseline Visit 6.Prior or current treatment with gene or stem cell therapy 7.Any other diseases which may significantly interfere with the assessment of LD 8.Have any additional conditions which, in the opinion of the Investigator, would make the subject unsafe for inclusion or could interfere with the subject participating in or completing the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective for this study is to evaluate the safety of VAL-1221;Secondary Objective: Exploratory efficacy objective The secondary objective is the identification and/or evaluation of biomarkers that are predictive of response to VAL-1221, are associated with susceptibility to developing adverse events, can provide evidence of VAL-1221 activity or can increase the knowledge and understanding of disease biology and drug safety;Primary end point(s): - Nature, incidence, seriousness and severity of adverse events - Incidence of treatment discontinuation due to adverse events - Nature, incidence, seriousness, severity, and timing of infusion-related reactions - Mean change in vital signs from baseline over time and incidence of abnormal vital sign measurements - Changes in ECG assessments from baseline over time and incidence of abnormal ECG assessments - Change from baseline and incidence of laboratory abnormalities (including hematology, clinical chemistry, coagulation, and urinalysis parameters);Timepoint(s) of evaluation of this end point: Screening visit, V1, V2, V3, V4 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Epilepsy - Change in tonic-clonic seizures frequency comparing the trial period with the 6 months preceding the baseline - Change in frequency of epileptiform discharges on EEG Cognitive functions - Non-worsening or improvement of Montreal Cognitive Assessment (MoCA) - Non-worsening or improvement of Leiter Performance Scale 3 (Leiter-3) - Non-worsening or improvement of the Visual Motor Integration test (VMI) - Non-worsening or improvement of children’s color trail test - Non-worsening or improvement of NEPSY-II inhibition subtest - Non-worsening or improvement of verbal fluency tests - Non-worsening or improvement of Weiss working memory subtest - Non-worsening or improvement of Weiss vocabulary subtest Motor functions - Non-worsening or improvement of 4-Stage Balance Test - Non-worsening or improvement of Timed Up and Go (TUG) - Non-worsening or improvement of 6-minute walk test (6MWT) - Non-worsening or improvement of 10-Meter Walk Test (10MWT) - Non-worsening or improvement of 9-hole peg test (9HPT) - Non-worsening or improvement of Scale for Assessment and Rating of Ataxia (SARA) - Non-worsening or improvement of Unified Myoclonus Rating Scale (UMRS) Global assessment and disease burden - Non-worsening or improvement of Lafora Disease Performance Scale (LDPS) - Non-worsening or improvement of Lafora Epilepsy Severity Scale (LESS) - Non-worsening or improvement of Barthel Index - Non-worsening or improvement of Vineland-II - Change of Clinical Global Impression– Improvement (CGI-I) - Change of Patient Global Impression – Change (PGI-C) - Change of Caregiver Global Impression – Change (CaGI-C) - Non-worsening or improvement of Burden Scale for Family Caregivers (short form); - Blood and CSF biomarkers - Brain MRI measures - Brain FDG-PET measures;Timepoint(s) of evaluation of this end point: Screening visit, V1, V2, V3, V4; Screening visit, V3 | — |
Countries
Italy
Contacts
Public ContactIRCCS Ricerca AUSL di Bologna
IRCCS Istituto delle Scienze Neurologiche di Bologna, Azienda USL di Bologna
Outcome results
None listed