Prospective, Retrospective, Multicenter, Observational Study of Disease Progression in Adults with Inherited Forms of Spastic Paraplegia
- Conditions
- Spastic paraplegia / adrenomyeloneuropathy10029299
- Registration Number
- NL-OMON54146
- Lead Sponsor
- SwanBio Therapeutics, Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 20
1. Male adults aged >=18 years
2. Diagnosed with ALD based on elevated VLCFA assay and pedigree analysis
3. Clinical evidence of spinal cord involvement with EDSS score between 1 and
6.5. The number of patients with normal pyramidal function on the Functional
System Score (FSS) of the EDSS scale, or EDSS >=5.5 will be limited to 10 (ten)
for each criterion.
4. The participant provided written informed consent prior to any study
procedures being performed
1. Diagnosed with cerebral inflammatory disease. Cerebral inflammatory disease
is diagnosed with the presence of inflammatory (Gadolinium-enhancing) lesion(s)
on a brain MRI.
Note: Absence of cerebral inflammatory disease will be confirmed at Visit 1
with review of a MRI scan or report performed within 12 months prior to the
Baseline Visit.
2. In AMN participants, pathological changes identified on a brain MRI except
for the abnormalities that can be observed in AMN participants.
3. Any conditions that make it impossible to perform MRI studies (including
allergy to Gadolinium or contrast agents).
4. Unstable, clinically significant neurologic (other than the disease being
studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic,
renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or
endocrine disease (other than adrenal insufficiency) or other abnormality,
which may impact the ability to participate in the study or that may
potentially confound the study results. It is the responsibility of the
Investigator to assess the clinical significance; however, consultation with
the Medical Monitor may be warranted.
5. Participant who, in the opinion of the Investigator, has any other medical
or psychological condition or social circumstances which would impair their
ability to participate reliably in the assessments, or who may increase the
risk to themselves or others by participating.
6. The participant is employed by SwanBio Therapeutics, Contract Research
Organization (CRO), or trial site (permanent, temporary contract worker, or
designee responsible for the conduct of the study) or is an immediate family
(e.g. spouse, parent, child, sibling) member of a SwanBio Therapeutics, CRO, or
trial site employee.
Study & Design
- Study Type
- Observational non invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method