A Study of AMDX-2011P in Participants With CAA

Phase 2

Recruiting

• Conditions: Cerebral Amyloid Angiopathy
• Interventions: Drug: AMDX-2011P

• Registration Number: NCT05709314
• Lead Sponsor: Amydis Inc.

Brief Summary

The purpose of this study is to assess safety, tolerability, plasma pharmacokinetics and biologic activity of a single intravenous dose of AMDX-2011P in participants with cerebral amyloid angiopathy (CAA).

Detailed Description

This open-label, masked endpoint assessment study will evaluate the safety, tolerability, plasma pharmacokinetics (PK) and biological activity of an intravenous (IV) dose of AMDX-2011P in participants with CAA. Assessments of retinal images will be conducted by central masked assessors.

Participants will be admitted to the study site where eye examination and retinal imaging will be conducted before administration of the study drug. AMDX-2011P will be administered through a single IV bolus injection followed by safety assessments, retinal imaging and PK blood collection.

Study Timeline

• Study First Submitted: 2023-01-24
• Study First Submitted QC: 2023-01-24
• Study First Posted: 2023-02-02
• Status Verified: 2024-07
• Study Start: 2024-07-09
• Last Update Submitted: 2024-07-29
• Last Update Posted: 2024-07-30
• Primary Completion: 2024-11
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

1. Diagnosis of hereditary CAA or probable and definite symptomatic or asymptomatic sporadic CAA diagnosed through genetic testing or according to the modified Boston neuroradiological criteria, who had undergone at least one brain magnetic resonance imaging (MRI)prior to entry into study.
2. Abnormality consistent with CAA on historical MRI.
3. In general good health

Exclusion Criteria

1. Presence of any underlying physical or psychological medical condition that, in the opinion of the investigator, would make it unlikely that the participant will complete the study per protocol.
2. Clinically significant laboratory abnormalities assessed by the investigator.
3. Active malignancy and/or history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia.
4. Prolonged QTcF (>450 ms for males and >470 ms for females),cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the investigator.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AMDX-2011P 100 mg	AMDX-2011P	AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review

Primary Outcome Measures

Name	Time	Method
AMDX-2011P Adverse Events Profile	8 days	Incidence, nature and severity of AEs/SAEs

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic Analysis of AMDX-2011P	2 hours	Area under the plasma versus time curve (AUC)
Biological Activity	8 days	Detection of amyloid deposits in the retina after intravenous AMDX-2011P administration using a conventional retinal fundus fluorescence imaging device.
Concentration of AMDX-2011P	2 hours	Plasma PK Concentration (CMax)AMDX-2011P to AMDX-2011

Trial Locations

Locations (3)

Associated Retina Consultants; 🇺🇸 Phoenix, Arizona, United States

Eye Research Foundation; 🇺🇸 Newport Beach, California, United States

Global Research Management; 🇺🇸 Glendale, California, United States