Los requisitos de elegibilidad de los participantes incluyen la edad, el sexo, el tipo y el estadio de la enfermedad, y los problemas de salud o tratamientos previos. Las pautas difieren de un estudio a otro e identifican quiénes pueden o no pueden participar. No hay garantía de que cada persona elegible que desee participar en un ensayo se inscribirá. Comunícate con el equipo del estudio para analizar la elegibilidad del estudio y la posible participación.
Key Inclusion Criteria:
- Participants who have documentation of a gene mutation in the CSF1R gene
- Participants fulfill both (Parts A and B) of the following criteria:
1. The participant has more than 2 findings of clinical signs or symptoms in the following categories:
1. Cognitive impairment or psychiatric problem;
2. Pyramidal signs on neurological examination;
3. Extrapyramidal signs, such as rigidity;
4. Epilepsy.
2. MRI findings consistent with ALSP (Konno, 2018; Appendix 5), specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum, on the Screening MRI using local reads.
Note: Subjects who have other causes of leukoencephalopathy, including vascular dementia, multiple sclerosis, or leukodystrophy (eg, adrenoleukodystrophy, Krabbe disease, metachromatic leukodystrophy) will be excluded.
Inclusion Criteria - Long-Term Extension (Weeks 52-148):
Subjects who meet the following criteria are eligible for the LTE:
- The subject completed the 52-week Core Study and is a suitable candidate for continued treatment with VGL101 in the LTE;
- The subject is capable of providing written informed consent for the LTE, including signing and dating the ICF, or has a study partner or legal guardian who can provide written informed consent with subject assent;
- The subject has a study partner (i.e., caregiver, family member, friend, etc) who, in the investigator’s judgment, has frequent and sufficient contact with the subject so as to be able to provide accurate information about the subject’s health and cognitive and functional abilities. The study partner must be willing to sign a study partner ICF.
Key Exclusion Criteria:
- The participant has any neurological disease that poses a risk to the participant or can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but
not limited to, brain tumor, hydrocephalus, Alzheimer's disease, frontotemporal dementia (FTD), ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's
disease, and Down syndrome.
- Participant with any condition or situation that, in the opinion of the investigator or sponsor medical personnel, may place the subject at significant risk, confound the
study results, or interfere significantly with the participant's participation in the study.
Exclusion Criteria - Long-Term Extension (Weeks 52-148):
Subjects will be excluded from the LTE if any of the following apply:
- The subject prematurely discontinued treatment with VGL101 in the Core Study;
- The subject was, in the opinion of the investigator, noncompliant with the study procedures in the Core Study;
- The subject had a clinically significant medical, surgical, laboratory, or other abnormality that, in the opinion of the investigator, would compromise the subject’s safety in the LTE.
Note: Other protocol defined Inclusion/Exclusion Criteria may apply.
Eligibility last updated 12/7/23. Questions regarding updates should be directed to the study team contact.