Repeat Intracerebroventricular Injections of RB-ADSC in Subjects Previously Treated in RBI Protocol RB-ADSC-02

Phase 1Not Yet RecruitingNCT07373067

Regeneration Biomedical, Inc.9 enrolled

Overview

This is a Phase 1b Extension Trial to allow repeat intracerebroventricular injections of RB-ADSCs in subjects previously treated in and successfully completed RBI Protocol RB-ADSC-02. In the previous Phase 1 clinical trial, RB-ADSC-02, subjects with mild to moderate Alzheimer's disease (AD) received a single intraventricular injection of RB-ADSC. RB-ADSC will be delivered intracerebroventricularly every 2 months via the previously implanted Ommaya reservoir for up to 6 injections in total. Participants will be followed for 2 months after the last administration. The primary objective is safety and tolerability of repeated dosing of RB-ADSC. Adverse events (AEs) and serious adverse events (SAEs) will be assessed by the incidence and severity of dose-limiting toxicity (DLT) and other AEs, incidence and severity of cytokine response syndrome, vital sign measurements, clinical laboratory tests and physical examination. Preliminary efficacy of repeated dosing will be evaluated with clinical assessments (MMSE, FAST, ADAS-Cog), volumetric MRI (NeuroQuant), CSF biomarkers (phosphor-Tau, total-Tau,AB-42), and diagnostic imaging comparison (Amyloid-PET).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Eligibility

Sex: ALLMin age: 45 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Successful completion of the Phase 1 single dose administration study of RB-ADSC, Protocol RB-ADSC-02. 2. Written informed consent to participate in this Phase 1b Extension Study either provided by the participant or a legal representative 3. The participant must have a relative/caregiver who is alert for possible side effects. 4. The caregiver must separately meet the specified inclusion/exclusion criteria for caregivers for this Phase 1b Extension Study. Exclusion Criteria: 1. Participant experienced treatment related SAE or DLT in Protocol RB-ADSC-02 2. Participants who, in the opinion of the Investigator, are unsuitable for this Phase 1 Extension Study

Outcomes

Primary Outcomes

The safety of repeated RB-ADSC treatment in study participants with AD

Safety will be determined by incidence, type and severity of adverse events (AE) and serious adverse events (SAE) graded according to CTCAE v5.0 and CRS revised grading system and defined by clinical relevant findings at every visit and 2 month following last dose in physical examination, vital signs and laboratory data

Time frame: up to 12 months

Secondary Outcomes

Change from Baseline in cerebrospinal fluid (CSF) biomarker pTau

CSF Biomarkers will be measured with the ADmark® phospho-tau/total-tau/ Abeta-42 assay to determine the levels of Phosphorylated-Tau protein in the cerebrospinal fluid (CSF).