跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NL-OMON52367
NL-OMON52367招募中不适用

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

Vigil Neuroscience, Inc.0 个研究点目标入组 4 人开始时间: 待定最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit

试验速览

阶段
不适用
状态
招募中
入组人数
4
研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型
Observational

入排标准

年龄范围
18 至 99（—）

入选标准

  • •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,
  • •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,
  • 另有 7 项未显示

排除标准

  • •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
  • •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.

研究者

相似试验

已完成
不适用
A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay SyndromeVenous malformation and Klippel-Trenaunay Syndrome
JPRN-UMIN000047330ARTham Therapeutics Inc.34
已完成
不适用
An exploratory study to biotype adult patients with unipolar depression using digital technologiesDepressionUnipolar depression10027946
NL-OMON48415Centre for Human Drug Research60
已完成
不适用
The natural history study of ambulatory patients with Duchenne muscular dystrophyDuchenne muscular dystrophy
JPRN-UMIN000030562ational Center of Neurology and Psychiatry (NCNP)33
尚未招募
不适用
A CLINICAL STUDY AMONG PATIENTS OF PRIMARY CUTANEOUS AMYLOIDOSIS REGARDING DISEASE SYMPTOMS AND ITS CAUSES USING SKIN BIOPSY RESULTS AND DERMOSCOPY
CTRI/2022/11/047739I
已完成
不适用
In those undertaking Periacetabular Osteotomy (PAO), does the use of i-Factor bone graft affect bone healing?Developmental Dysplasia of the Hip (DDH)Periacetabular Osteotomy of the Hip (PAO)Musculoskeletal - Other muscular and skeletal disordersSurgery - Surgical techniques
ACTRN12621000625875a Trobe Sport and Exercise Medicine Research Centre, La Trobe University295