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

Regeneration Biomedical

Status and phase: Not yet enrolling; Phase 1

Conditions: Alzheimer Disease

Treatments: Biological: RB-ADSC

Study type: Interventional

Funder types: Industry

Identifiers

NCT07373067

RB-ADSC-02E

Details and patient eligibility

About

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. The primary objective of safety is performed 2 months after the last dose administration at the month 12 follow-up visit. The secondary objective endpoint evaluations of efficacy are performed at the month 6 and 12 visits.

Full description

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).

Enrollment: 9 estimated patients

Sex: All

Ages: 45+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 3 patient groups

RB-ADSC low dose

Experimental group

Description:

Participants will receive 2x10\^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses

Treatment:

Biological: RB-ADSC

RB-ADSC medium dose

Experimental group

Description:

Participants will receive 5x10\^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses

Treatment:

Biological: RB-ADSC

RB-ADSC high dose

Experimental group

Description:

Participants will receive 10x10\^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses

Treatment:

Biological: RB-ADSC