The Study of Safety and Preliminary Efficacy of Aleeto in Patients With MultIple System Atrophy
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age30-75
SponsorBeijing Tiantan Hospital
About this trial
This is a single-center, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. The objective of the study is to evaluate the safety, tolerability and potential preliminary efficacy of Aleeto in the treatment of patients with multiple system atrophy (MSA).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
Head MRI at screening showing evidence of other CNS lesions consistent with a diagnosis of neurodegenerative disease other than MSA;
Patients with MMSE scores indicative of dementia prior to enrolment (≤17 points for illiterate individuals, ≤20 points for individuals with elementary school education, ≤24 points for individuals with junior high school education or higher) or those with a prior confirmed diagnosis of dementia;
Head MRI at screening showing other significant pathological findings including but not limited to: cerebral hemorrhage, acute phase of cerebral infarction, aneurysm, vascular malformation, infectious lesion, brain tumor or other space-occupying lesion (meningiomas or arachnoid cysts with a maximum diameter of <1 cm need not be excluded);
Presence of immune disorders that are inadequately controlled or require treatment with biological agents;
Trial design
Design model
Parallel
Treatments tested in this trial
Aleeto
Drug"Aleeto" is a nerve repair protein developed by Darwin Start (Beijing) Biopharmaceutical Co., Ltd. It is a group of specific microenvironmental protein polymers secreted under the emergency conditions of stem cells. It has the advantages of selective assembly, targeted delivery, efficient repair of damaged tissues, high safety, chemical stability, easy storage, etc., and has a powerful neural repair function. According to the groups, patients would be treated with Aleeto via intrathecal injection or intravenous injection.
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: Intervention groupExperimental treatment 1 intervention
Group B: Control groupNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)
Incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs) within 90±7 days after randomization;
Time frame
Day 90±7 after randomization2
The incidence of changes in clinical laboratory test parameters, changes in vital signs, neurological examination abnormalities and ECG abnormalities
Laboratory tests include: routine blood test, coagulation test, blood biochemical examination, urinalysis (note that abnormal changes in laboratory test results may need to be reviewed to further determine and assess their relevance to the study). Vital signs include: abnormal body temperature (≥38℃ or ≤35℃), blood pressure (systolic blood pressure ≥180mmHg or \<90mmHg), respiration (\>24 beats/min and other rhythm abnormalities), heart rate (\>100%), and blood pressure (\<10%).
Time frame
Day 90±7 after randomizationSecondary outcomes
1
Changes in the unified multiple system atrophy rating scale (UMSARS) part scores, sum of part 1 and 2 scores
The unified multiple system atrophy rating scale (UMSARS) is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability, UMSARS-II (14 items) assesses motor impairment based on a clinical examination, UMSARS-III records blood pressure and heart rate in the supine and standing positions, and UMSARS-IV (1 item) rates chore-based disability. Higher scores on the UMSARS indicate greater disability.
Time frame
Day 15, 45±3, 75±5 and 90±7 after randomization2
Changes in the composite autonomic symptom score (COMPASS) scores
The Composite Autonomic Symptom Score (COMPASS) is a revised version of the 169-item Autonomic Symptom Profile assessing 11 domains of autonomic function, to the 31-item COMPASS, now assessing six domains: orthostatic hypotension (maximum score 10), vasomotor (maximum score 6), secretomotor (maximum score 7), gastrointestinal (maximum score 28), bladder (maximum score 9), and pupillomotor functions (maximum score 15). Scores from each component are recorded and a weighted total score is calculated. Higher scores on this scale indicate more severe clinical symptoms related to autonomic dysfunction. It is a validated, easy-to-use self-assessment instrument designed for clinical autonomic research and practice.
Time frame
Day 15, 45±3, 75±5 and 90±7 after randomization3
Changes in the incidence of orthostatic hypotension
Changes in the incidence of orthostatic hypotension in patients on days 8, 38±3, 68±5, and 90±7 after randomization as measured by recumbent-upright blood pressure monitoring/24-hour ambulatory blood pressure monitoring/head-upright tilt test;
Time frame
Day 15, 45±3, 75±5 and 90±7 after randomization4
Variation of three-dimensional gait analysis parameters under multitasking
Changes in three-dimensional gait analysis parameters under multitasking in patients on day 90±7 after randomization;
Time frame
Day 90±7 after randomizationOther outcomes
1
Changes in postvoid residual urine volume (bladder ultrasound) in patients;
Changes in postvoid residual urine volume (bladder ultrasound) in patients on day 90±7 after randomization;
Time frame
Day 90±7 after randomization2
Changes in plasma biomarkers (NFL, α-syn, Tau protein, Aβ40/42/43, YKL-40, CoQ10, UA, Hcy, and other biomarkers) associated with neural repair, vascular endothelial injury, inflammatory factors and histology
Changes in NFL, α-syn, Tau protein, Aβ40/42/43, YKL-40, CoQ10, UA, Hcy, and other biomarkers related to neural repair, vascular endothelial injury, inflammatory factors, and histology in plasma on days 15, 30±3, 60±5, 90±7, 180±14, and 360±14 after randomization;
Time frame
Day 15, 30±3, 60±5, 90±7, 180±14, and 360±14 after randomization3
Changes in plasma biomarkers (NFL, α-syn, Tau protein, Aβ40/42/43, YKL-40, CoQ10, UA, Hcy, and other biomarkers) associated with neural repair, vascular endothelial injury, inflammatory factors and histology
Changes in NFL, α-syn, Tau protein, Aβ40/42/43, YKL-40, CoQ10, UA, Hcy, and other biomarkers related to neural repair, vascular endothelial injury, inflammatory factors, and histology in plasma on days 31±3 and 61±5 after randomization;
Time frame
Day 31±3 and 61±5 after randomization4
Changes in patients' 3T MRI imaging features (degree of cerebral atrophy in the putamen, other brain regions and whole brain; iron deposition; image parameters of diffusion tensor imaging; etc.)
3T MRI imaging features: degree of cerebral atrophy in the putamen, other brain regions and whole brain; iron deposition assessed by quantifying magnetic susceptibility values through the use of quantitative susceptibility mapping; image parameters of diffusion tensor imaging, including mean diffusivity and fractional anisotropy.
Time frame
Day 90±7 after randomizationSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov