A Safety and Tolerability Study of GB301

Phase 1

• Conditions: Alzheimer Disease
• Interventions: Other: Saline; Biological: Regulatory T cells

• Registration Number: NCT03865017
• Lead Sponsor: VTBIO Co. LTD

Brief Summary

The primary objective of this study is to evaluate the safety and tolerability of multiple intravenous (IV) infusions at a single dose strength of GB301 in subjects with mild to moderate AD.

Detailed Description

Not available

Study Timeline

• Status Verified: 2019-02
• Study First Submitted: 2019-02-15
• Study First Submitted QC: 2019-03-04
• Study First Posted: 2019-03-06
• Last Update Submitted: 2019-08-02
• Last Update Posted: 2019-08-05
• Study Start: 2019-12-01
• Primary Completion: 2020-12-01
• Study Completion: 2021-12-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Saline	Saline+cell suspension solution infusion
Regulatory T cells	Regulatory T cells	Biologicals: Autologous Regulatory T cells (1.7\*10\^5 cells/kg, i.v) other name: GB301

Primary Outcome Measures

Name	Time	Method
Number of subjects with abnormal clinical chemistry parameters	Between Baseline and 12 weeks	Blood samples will be collected for the assessment of following clinical chemistry parameters: Albumin, Total bilirubin, Total protein, Calcium, alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), blood urea nitrogen (BUN), Creatinine, Glucose, Sodium, Potassium, Chloride, Bicarbonate, LDH, FSH, Uricacid
Columbia Suicide Severity Rating Scale (C-SSRS)	Between Baseline and 12 weeks	The C-SSRS captures the occurrence, severity, and frequency of suicide-related thoughts and behaviors during the assessment period. Some questions are yes/no and some are on a scale of 1 (low severity) to 5 (high severity). Completed suicide and non-fatal suicide events are yes/no questions and results presented are the number of participants with these events.
Number of subjects With Clinically Significant Abnormalities in 12-lead Electrocardiogram	Between Baseline and 12 weeks	The number of subjects with normal and abnormal ECG findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator.; ECG measures PR interval (ms), QRS interval, QT interval(ms), QTc interval (ms), and heart rate(bpm) for each treatment group at each time point.
Number of subjects with abnormal Coagulation parameters	Between Baseline and 12 weeks	Blood samples will be collected for the assessment of following coagulation parameters: Prothrombin Time(PT), International normalized ratio(INR), partial thromboplastin time (PTT)
Number of subjects with abnormal vital signs	Between Baseline and 12 weeks	Vital signs, including height (㎡, only assessed at Screening), weight(kg), systolic and diastolic blood pressure(mm Hg), heart rate(bpm), and body temperature(℃), will be measured after the subject has been in a sitting position for 5 minutes.
Number of subjects with adverse events (AEs)	Between Baseline and 12 weeks	An AE is defined as any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to the medicinal product.
Number of subjects with abnormal Hematology parameters	Between Baseline and 12 weeks	Blood samples will be collected for the assessment of following hematology parameters: red blood cell (RBC) count, Hemoglobin, Hematocrit, mean corpuscular volume (MCV),mean corpuscular hemoglobin (MCH), mean corpuscular hemoglobin concentration (MCHC), Platelet, white blood cell (WBC) count, Neutrophil, Lymphocyte, Monocyte, Eosinophil, Basophil
Number of subjects with abnormal Urinalysis parameters	Between Baseline and 12 weeks	Samples will be collected to measure specific gravity, potential of hydrogen (pH), glucose, protein, blood and ketones.
Number of subjects with abnormal physical examination	Between Baseline and 12 weeks	A full physical examination will include assessments of the general apperance, skin, head, neck, eyes, ears, nose, throat, respiratory, cardiovascular, abdomen, extremities, musculoskeletal, neurological, lymph nodes etc.

Secondary Outcome Measures

Name	Time	Method
Change from baseline in MMSE score	baseline, 29, 57, and 85 days	The MMSE is a brief, screening instrument often used in clinical studies to assess dementia severity. The MMSE assesses several aspects of memory and cognitive functioning including orientation, attention, concentration, comprehension, recall, and praxis. The total possible score is 30, with high scores indicating less impairment.
Change from Baseline in Alzheimer´s Disease Assessment Scale- Cognitive Subscale (ADAS-Cog-13) Score	baseline, 29, 57, and 85 days	13-item ADAS-Cog assesses a range of cognitive abilities including memory, comprehension, orientation in time and place, and spontaneous speech. Most items are evaluated by tests, but some are dependent on clinician ratings on a 5-point scale. The ADAS-Cog-13 is the ADAS-Cog-11 with 2 additional items: delayed word recall and total digit cancellation. Scores for the ADAS-Cog-13 range from 0 to 85 with higher scores indicating greater dysfunction.

Trial Locations

Locations (1)

Bht Lifescience Australia Pty Ltd; 🇦🇺 Brisbane, Queensland, Australia