A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

Recruiting

• Conditions: ALSP; 10029299

• Registration Number: NL-OMON52367
• Lead Sponsor: Vigil Neuroscience, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 9 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 4

Inclusion Criteria

General and Administrative
1. Male or female subjects aged >=18 years on the day the informed consent form
(ICF) is signed.
2. Subjects who are able, in the opinion of the investigator, to understand the
nature of the study and to comply with the protocol requirements,
including scheduled visits, blood sampling, and other study procedures,
or who have a caregiver or legal guardian who can
understand and assist the subject in complying with the protocol
requirements.
3. Subjects who are willing and able to refrain from use of any prohibited
medication/treatments that are not permitted by the protocol
throughout the study period.
4. Subjects who receive approval of sponsor medical personnel as to final
suitability for the study.

Inclusion Criteria - Subjects With Definitive ALSP
5. Subjects who have documentation of a gene mutation in the CSF1R gene (prior
to enrollment).
6. Subjects who fulfill both of the following criteria (a and b):
a. More than two findings of clinical signs or symptoms in any of 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: specially, bilateral cerebral white
matter lesions with or without thinning of the corpus callosum (Konno et al,
2018;
Appendix 3, Section 10.3).
NOTE: Subjects with other causes of leukoencephalopathy, including vascular
dementia, multiple sclerosis, or leukodystrophy (e.g.,
adrenoleukodystrophy, Krabbe disease, metachromatic
leukodystrophy), will be excluded.
7. Subjects who, in the investigator*s opinion, have demonstrated clinical
progression of their ALSP within the past year.
8. Subjects who have a score of >=12 on the MoCA.
9. Subjects who are ambulatory with or without aids (cane, crutches, etc) or,
if restricted to a wheelchair, are still able to wheel self, transfer in
and out of wheelchair, and walk up to 5 meters with or without aid.
NOTE: 7 subjects that are non-ambulatory for reasons other than progression of
ALSP may be enrolled.
10. 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.

Inclusion Criteria - Subjects With Prodromal ALSP
11. Subjects who have documentation of a gene mutation in CSF1R gene (prior to
enrollment).
12. MRI findings consistent with ALSP: specifically, bilateral cerebral white
matter lesions with or without thinning of the corpus callosum (Konno et al,
2018;
Appendix 3, Section 10.3). 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 6a as *more than two*).
13. Subjects who meet the criteria for prodromal ALSP and who, at later study
visits,
meet the criteria for definitive ALSP should have a designated caregiver for
subsequent visits who spends at least 4 hours per week with them unless
otherwise approved by the sponsor and/or medical monitor. The careg

Exclusion Criteria

Medical Conditions
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 with any concurrent diagnosis that may confound neuropsychological
testing (e.g., hearing impairment, visual impairment).
3. Subjects with any concurrent diagnosis that may confound ambulation
measurements (e.g., amputee).
4. Subjects with contraindications for undergoing a lumbar puncture, such as
bleeding disorders, increased intracranial pressure, or
abnormal spinal anatomy.
NOTE: Only for subjects who participate in the optional CSF Biomarker
Sub-study.
5. Subjects who are unable to undergo MRI. (e.g., implants not compatible
for MRI, claustrophobia, inability to remain still that will prevent
acquisition of a good quality scan)
6. Female subjects who are pregnant, planning pregnancy in the next 12 months,
or breastfeeding.
7. Subjects who are at significant risk of suicidal or violent behavior, in the
opinion of the investigator. If a subject answers *yes* to the
Question 4 or 5 on the C SSRS, a risk assessment should be done by a
qualified healthcare professional to assess whether it is
safe for the subject to participate in the study.
8. Subjects with a current history of major medical illness, such as renal
failure, congestive heart failure, or advanced pulmonary
disease, that could put the subject at additional risk if participating
in the study.
9. Subjects with a history of cancer that required active treatment in the last
5 years, with the exception of in situ cervical cancer and
basal cell carcinoma of the skin.
10. 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.
11. Subjects who have previously undergone HSCT or plan to undergo HSCT
within 12 months prior to Screening/Baseline visit.

Prior/Current Clinical Study Experience
12. Subjects who are concurrently enrolled in an investigational drug or device
study or who received an investigational product within 30 days of 5 half-lives
before signing the ICF.
Note: Subjects who are receiving VGL101 in a clinical study may enroll after
conclusion of their participation in the VGL101 clinical study

Other Exclusion Criteria
13. Subjects who are involved, directly or indirectly, in the conduct or
administration of this study as an investigator, sub-investigator,
study coordinator, or other study staff member.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified