Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Key Inclusion Criteria for both Definitive ALSP patients and Prodromal ALSP patients:
1. Subjects who have documentation of a gene mutation in the CSF1R gene
Key Inclusion Criteria for Definitive ALSP patients, only:
1. Subjects who fulfill both of the following criteria (a and b):
a. More than two findings of clinical signs or symptoms in the following categories:
i. Cognitive impairment or psychiatric problem ii. Pyramidal signs on neurological
examination iii. Extrapyramidal signs, such as rigidity, tremor, abnormal gait, or
bradykinesia iv. Epilepsy b. MRI findings consistent with ALSP: specifically,
bilateral cerebral white matter lesions with or without thinning of the corpus
callosum NOTE: Subjects with other causes of leukoencephalopathy, including vascular
dementia, multiple sclerosis, or leukodystrophy (e.g., adrenoleukodystrophy, Krabbe
disease, metachromatic leukodystrophy), will be excluded.
2. Subjects who, in the investigator's opinion, have demonstrated clinical progression of
their ALSP within the past year.
3. Subjects who meet the criteria for definitive ALSP must have a designated caregiver
who spends at least 4 hours per week with them. The caregiver must be able and willing
to assist the subject in complying with the study requirements, be able to provide
information during study visits, and be willing to sign a caregiver ICF.
Key Inclusion Criteria for Prodromal ALSP patients, only:
1. MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions
with or without thinning of the corpus callosum. Prodromal subjects may have none or up to
and including 2 ALSP-related clinical signs or symptoms (i.e., they do not meet the
clinical criteria outlined in the Definitive ALSP as "more than two").
Key Exclusion Criteria (for all study participants):
1. Subjects with any neurological or psychiatric diseases that can produce cognitive,
motor, or behavioral impairment similar to ALSP, including, but not limited to,
Alzheimer's disease, frontotemporal dementia, ALS, stroke, Huntington disease,
multiple sclerosis, Parkinson's disease, and Down syndrome, or with active
alcohol/drug abuse
2. Subjects who are unable to undergo MRI
3. Subjects 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 subject's participation in the
study.
4. Subjects who have previously undergone HSCT or plan to undergo HSCT within 12 months
of the Screening/Baseline visit.
Note: Other protocol defined Inclusion/Exclusion Criteria may apply.
Eligibility last updated 1/30/2024. Questions regarding updates should be directed to the study team contact.