Proof-of-concept Study of New Imaging Diagnostic in Patients With Suspected Alzheimer's Disease

NCT05453539 | Terminated Phase 1 DMC

• 2 other identifiers: ADx-001-101-POC2021-003399-14
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 2

Brief Summary

This study is a Phase 1, first-in-human, multi-center study to establish safety of ADx-001 in healthy volunteers, and safety and proof of concept in patients with confirmed amyloid plaques in the brain (confirmed by amyloid positron emission tomography (PET)). ADx-001 is a novel, intravenously delivered, Gd- containing molecularly targeted liposomal product that is being developed for use in contrast-enabled MR imaging of amyloid plaques.

Conditions

Alzheimer Disease

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

  Day 0-28

Secondary Outcomes (2)

• The number of brain regions identified the same way in ADx-001 enhanced MRI and in amyloid-PET

  Screening - day 4

• The amount of contrast enhancement in the amyloid positive brain regions in different dose levels

  Screening - day 4

Study Arms (5)

Healthy volunteers dose level 1

EXPERIMENTAL

0.53 mL ADx-001 / kg

Drug: DSPE-DOTA-Gd Liposomal Injection

Patients dose level 1

EXPERIMENTAL

0.53 mL ADx-001 / kg

Drug: DSPE-DOTA-Gd Liposomal Injection

Patients dose level 2

EXPERIMENTAL

1 mL ADx-001 / kg

Drug: DSPE-DOTA-Gd Liposomal Injection

Patients dose level 3

EXPERIMENTAL

2 mL ADx-001 / kg

Drug: DSPE-DOTA-Gd Liposomal Injection

Patients dose level 4

EXPERIMENTAL

4 mL ADx-001 / kg

Drug: DSPE-DOTA-Gd Liposomal Injection

Interventions

DSPE-DOTA-Gd Liposomal Injection DRUG

Single dose, intravenous infusion

Also known as: ADx-001

Healthy volunteers dose level 1; Patients dose level 1; Patients dose level 2; Patients dose level 3; Patients dose level 4

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• For healthy volunteers
• years old (inclusive)
• Women of child-bearing potential must have a negative serum pregnancy test and agree to use highly effective contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of the study and for 3 months after completion of dosing; men enrolled on this protocol must agree to use adequate contraception prior to study entry, for the duration of the study and for 3 months after completion of dosing; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
• Signed informed consent prior to any study specific tests or procedures
• Healthy, based on medical history, physical examination, electrocardiogram (ECG), and laboratory tests
• For patients with amyloid plaques in PET:
• years old inclusive
• Proven amyloid deposits in brain (amyloid PET)
• Women of child-bearing potential must have a negative serum pregnancy test and agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of the study and for 3 months after completion of dosing. Men enrolled on this protocol must agree to use adequate contraception prior to study entry and for 3 months after completion of dosing; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
• Willing and able to provide written informed consent
• The ability to provide consent determined and documented by a neurologist or psychiatrist.

You may not qualify if:

• For healthy volunteers
• Disturbances in fat metabolism \[hyperlipemia, hyperlipoproteinemia or hypercholesterolemia (total cholesterol \>260 mg/dL)\]
• History of allergic reactions attributed to any of the components of ADx-001, liposomes, other gadolinium contrast agents, or any other component of the formulation
• Pathological liver function:
• Bilirubin \> upper limit of normal, and/or
• AST (SGOT), ALT (SGPT) and Alkaline phosphatase \> upper limit of normal
• Calculated creatinine clearance \< 60 mL/min/1.73 m2 (CKD-EPI formula)
• Heart rate at rest \<50 or \>90 bpm
• Systolic blood pressure \<100 or \>140 mmHg
• Diastolic blood pressure \<50 or \>90 mmHg
• Clinically relevant ECG findings, e.g., PQ \>220 ms, QTc \>450 ms, QRS \>120 ms, branch bundle block, any sign of coronary heart disease
• Unwillingness or inability to comply with procedures required in this protocol
• Subjects who are currently receiving any other investigational agent, or who have received an investigational agent within the last 10 half-lives of the investigational agent
• Any known disposition for allergic, anaphylactoid, hypersensitivity or idiosyncratic reactions, e.g. any history of clinical signs of hypersensitivity reaction to any agent (including, but not limited to, any allergen, food, drug, chemical, or contrast agent)
• Use of systemic or topically active medication or herbal remedies, prescription or non-prescription, from screening to the first drug administration. Only use of contraceptives and occasional use of paracetamol, aspirin, or ibuprofen is permissible.

Sponsors & Collaborators

• Alzeca Biosciences, Inc.: lead
• pharmtrace klinische Entwicklung GmbH: collaborator

Study Sites (2)

CRS Clinical Research Services Berlin GmbH

Berlin, 13353, Germany

Location

LMU Klinikum

Munich, 81377, Germany

Location

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• Sophia Stoecklein

  LMU Klinikum

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2022
• First Posted: July 12, 2022
• Study Start: November 9, 2022
• Primary Completion: November 8, 2024
• Study Completion: November 8, 2024
• Last Updated: August 13, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

DE (2)