Ignite Creation Date:
2024-05-06 @ 7:55 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06181526
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-12-26
First Post:
2023-12-06
Brief Title:
Safety and Preliminary Efficacy of Aleeto in Amyotrophic Lateral Sclerosis
Sponsor:
Beijing Tiantan Hospital
Organization:
Beijing Tiantan Hospital
Study Overview
Official Title:
The Study of Safety and Preliminary Efficacy of Aleeto in Patients With Amyotrophic Lateral Sclerosis
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SPECIALS
Brief Summary:
This study is a single-center randomized double-blind placebo parallel-controlled dose-escalation clinical study The aim of this study was to evaluate the safety tolerability and preliminary effect of Aleeto in adult patients with ALS and to provide an appropriate dose for the future clinical trial
Detailed Description:
Amyotrophic lateral sclerosis ALS is a fatal neurodegenerative disease which is characterized by progressive loss of number and function of upper and lower motor neurons located in the brain and spinal cord which leads to paralysis ALS mainly begins in the limbs and face where about one-third of patients have difficulty in speaking chewing or swallowing then progressively affecting the pharyngeal muscles attacking the sphincter in the advanced stages of the disease and eventually affecting the trunk and respiratory function as the disease progresses
Aleeto derived from cellular exosomes is a group of specific microenvironment protein polymers secreted by stem cells under stressed conditions which has the advantages of selective assembly targeted delivery efficient repair of damaged tissues high safety stable chemical properties and easy preservation Whats more Aleeto has a strong nerve repair function
This study is a single-center randomized double-blind placebo parallel-controlled dose-escalation clinical study aiming to evaluate the safety tolerability and preliminary effect of Aleeto in adult ALS patients and provide an appropriate dose for the future clinical trial
In this study we plan to enroll 24 patients and will be randomly assigned to 4 groups The initial dose group consists of four subjects out of which three subjects will receive intravenous administration and intrathecal administration of 05μgkg Aleeto while one subject will receive a placebo Fourteen subjects will be randomly assigned to the 1μgkg and 2μgkg dose groups to receive intrathecal administration combined with intravenous administration with one subject in each dose group randomly receiving placebo treatment Six subjects will be randomly assigned to the 2μgkg dose group to receive intravenous administration only Data are collected in face-to-face interviews at baseline and Day 14 30 37 60 67 90 120 follow-up visits All patients will be examined before and after treatment and the results will be compared
The primary outcomes are the incidence of adverse events AEs and serious adverse events SAEs and the incidence of abnormal changes in laboratory examination indicators vital signs neurological physical examination and electrocardiogram within 120 days after the treatment The secondary outcomes included changes in immunological indicators pharmacokinetics ALS Functional Rating Scale-Revised ALSFRS-R as well as the incidence of invasive mechanical ventilation and mortality rate
The statistical analysis was performed by bilateral test and the test level α was 005
Baseline Equilibrium Analysis Analysis of variance or Kruskal-Wallis H test is used to compare continuous dataThe Chi-Squared test or Fishers exact test is used to compare the categorical data
Primary Efficacy Analysis The comparability among different dosage groups in terms of incidence of adverse events abnormal liver functions hypersensitivity reactions liver failure dyspnea etc severe adverse events and other abnormal indicators will be analyzed using Chi-squared tests or Fishers exact test Changes in laboratory examination indicators and vital signs before and after treatment will be analyzed using paired t-tests or Wilcoxon signed rank sum test The intergroup differences in laboratory examination indicators and changes in vital signs before and after treatment will be analyzed using t-test or Mann-Whitney U test
Secondary Efficacy Analyses The improvement of immunological indicators lymphocyte subgroup analysis pharmacokinetic indicators and ALSFRS-R before and after treatment will be analyzed using paired t-tests or Wilcoxon signed rank sum test The intergroup comparison of immunological indicators lymphocyte subgroup analysis pharmacokinetic indicators and ALSFRS-R before and after treatment will be analyzed using t-tests or Mann-Whitney U tests The intergroup significance of the incidence of invasive mechanical ventilation and mortality rate is determined using Chi-squared tests or Fishers exact test
Exploratory Analysis Comparison of measurement data The improvement of ALSAQ-40 EQ-5D-5L modified Norris scale forced lung capacity muscle strength electromyographic indicators gait function biomarker levels FSS imaging indicators HAMA and HAMD scales will be analyzed using paired t-tests or Wilcoxon signed rank sum test The above exploratory analysis indicators and the difference in survival time between two groups will be analyzed using t-test or Mann-Whitney U test Analysis of variance or Kruskal Wallis H-test will be used for comparing the differences ａｍｏｎｇ different dosａｇｅ groups and Bonferroni correction method will be used for multiple comparisons Mixed linear models will be used for analyzing the differences in the trend of changes between different dose groups over a period of 120 days the data which corresponds to skewed distribution will be transformed LSmeans will be used for analyzing the differences of indicators between different groups at different times as well as the differences within the same group at different times
Comparison of enumeration data Chi-square test or Fishers exact test for enumeration data
Aleeto derived from cellular exosomes is a group of specific microenvironment protein polymers secreted by stem cells under stressed conditions which has the advantages of selective assembly targeted delivery efficient repair of damaged tissues high safety stable chemical properties and easy preservation Whats more Aleeto has a strong nerve repair function
This study is a single-center randomized double-blind placebo parallel-controlled dose-escalation clinical study aiming to evaluate the safety tolerability and preliminary effect of Aleeto in adult ALS patients and provide an appropriate dose for the future clinical trial
In this study we plan to enroll 24 patients and will be randomly assigned to 4 groups The initial dose group consists of four subjects out of which three subjects will receive intravenous administration and intrathecal administration of 05μgkg Aleeto while one subject will receive a placebo Fourteen subjects will be randomly assigned to the 1μgkg and 2μgkg dose groups to receive intrathecal administration combined with intravenous administration with one subject in each dose group randomly receiving placebo treatment Six subjects will be randomly assigned to the 2μgkg dose group to receive intravenous administration only Data are collected in face-to-face interviews at baseline and Day 14 30 37 60 67 90 120 follow-up visits All patients will be examined before and after treatment and the results will be compared
The primary outcomes are the incidence of adverse events AEs and serious adverse events SAEs and the incidence of abnormal changes in laboratory examination indicators vital signs neurological physical examination and electrocardiogram within 120 days after the treatment The secondary outcomes included changes in immunological indicators pharmacokinetics ALS Functional Rating Scale-Revised ALSFRS-R as well as the incidence of invasive mechanical ventilation and mortality rate
The statistical analysis was performed by bilateral test and the test level α was 005
Baseline Equilibrium Analysis Analysis of variance or Kruskal-Wallis H test is used to compare continuous dataThe Chi-Squared test or Fishers exact test is used to compare the categorical data
Primary Efficacy Analysis The comparability among different dosage groups in terms of incidence of adverse events abnormal liver functions hypersensitivity reactions liver failure dyspnea etc severe adverse events and other abnormal indicators will be analyzed using Chi-squared tests or Fishers exact test Changes in laboratory examination indicators and vital signs before and after treatment will be analyzed using paired t-tests or Wilcoxon signed rank sum test The intergroup differences in laboratory examination indicators and changes in vital signs before and after treatment will be analyzed using t-test or Mann-Whitney U test
Secondary Efficacy Analyses The improvement of immunological indicators lymphocyte subgroup analysis pharmacokinetic indicators and ALSFRS-R before and after treatment will be analyzed using paired t-tests or Wilcoxon signed rank sum test The intergroup comparison of immunological indicators lymphocyte subgroup analysis pharmacokinetic indicators and ALSFRS-R before and after treatment will be analyzed using t-tests or Mann-Whitney U tests The intergroup significance of the incidence of invasive mechanical ventilation and mortality rate is determined using Chi-squared tests or Fishers exact test
Exploratory Analysis Comparison of measurement data The improvement of ALSAQ-40 EQ-5D-5L modified Norris scale forced lung capacity muscle strength electromyographic indicators gait function biomarker levels FSS imaging indicators HAMA and HAMD scales will be analyzed using paired t-tests or Wilcoxon signed rank sum test The above exploratory analysis indicators and the difference in survival time between two groups will be analyzed using t-test or Mann-Whitney U test Analysis of variance or Kruskal Wallis H-test will be used for comparing the differences ａｍｏｎｇ different dosａｇｅ groups and Bonferroni correction method will be used for multiple comparisons Mixed linear models will be used for analyzing the differences in the trend of changes between different dose groups over a period of 120 days the data which corresponds to skewed distribution will be transformed LSmeans will be used for analyzing the differences of indicators between different groups at different times as well as the differences within the same group at different times
Comparison of enumeration data Chi-square test or Fishers exact test for enumeration data
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None