Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy

NCT05542576 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: AMDX-2011P-101
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with neurodegenerative diseases (Parkinson's disease and ALS).

Conditions

Parkinson Disease; Amyotrophic Lateral Sclerosis

Keywords

Parkinsons; ALS

Outcome Measures

Primary Outcomes (1)

• AMDX-2011P Adverse Events Profile

  Incidence of Treatment Emergent Adverse Events (TEAEs) at for each cohort (dose level).

  1 week

Secondary Outcomes (2)

• Concentration of AMDX-2011P

  8 hours

• Pharmacokinetic Analysis of AMDX-2011P

  8 hours

Study Arms (3)

AMDX2011P 25mg

EXPERIMENTAL

25mg (1ml) single bolus injection intravenous for diagnostic review

Drug: AMDX2011P

AMDX2011P 50mg

EXPERIMENTAL

AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review

Drug: AMDX2011P

AMDX2011P 100mg

EXPERIMENTAL

AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review

Drug: AMDX2011P

Interventions

AMDX2011P DRUG

AMDX2011P single bolus injection intravenous for diagnostic review

AMDX2011P 100mg; AMDX2011P 25mg; AMDX2011P 50mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• For Subjects with Parkinson's Disease
• Clinically established Parkinson's disease based on Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's disease (Table 8) and a modified Hoehn \& Yahr scale of 1-3 (Table 9).
• No suspected atypical parkinsonian syndromes due to drugs, metabolic disorders, encephalitis, or degenerative diseases.
• For Subjects with ALS
• Confirmed diagnosis of ALS with both upper and lower motor neuron involvement.
• For All Subjects
• Ability to undergo retinal imaging.
• Subject or legally authorized representative must provide signed informed consent (or signed assent form) prior to study entry and have the ability and willingness to attend and comply with the necessary study procedures and visits at the study site. For subjects unable to physically sign the informed consent, a guardian or trusted care giver can sign on their behalf in presence of an independent witness.
• Contraception use by study subjects of childbearing potential (male and female) and female partners of childrearing potential male subjects should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

You may not qualify if:

• Presence of any underlying physical or psychological medical condition that would make it unlikely that the subject will complete the study per protocol.
• Clinically significant laboratory abnormalities assessed by the investigator.
• 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.
• 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.
• Presence of any ocular condition that would significantly hinder the ability to detect and quantify hyper-fluorescent puncta (e.g., eyes with significant hyper-autofluorescence that would mask the ability to detect, quantify, and discern post-injection hyper-fluorescent signal from pre-injection hyper-autofluorescence signal).
• Use of any new prescription therapies or vaccines within 7 days prior to the study drug administration.
• Drugs with potential phototoxicity per Package Insert are prohibited within 48 hours or 5 half-lives, whichever is longer, prior to first study drug until End-of-study (EOS) visit, except for those required for treatment of underlying disease.
• Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer.
• Females who are pregnant or breastfeeding.

Sponsors & Collaborators

• Amydis Inc.: lead

Study Sites (2)

Eye Research Foundation

Newport Beach, California, 92663, United States

Location

Brittany NIcholl

Pasadena, California, 91107, United States

Location

MeSH Terms

Conditions

Parkinson Disease; Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Matthew Lehman
• Organization: Amydis, Inc.

matt.lehman@amydis.com

6463317899

Study Officials

• Masoud Mokhtarani, MD

  Amydis Inc.

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

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

Model Details: Dose escalating via Cohorts total 1-3 cohorts

Study Record Dates

• First Submitted: September 7, 2022
• First Posted: September 15, 2022
• Study Start: August 24, 2022
• Primary Completion: March 15, 2023
• Study Completion: March 15, 2023
• Last Updated: August 20, 2025
• Results First Posted: August 20, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)