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AMDX-2011P Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy (PROBE)

Primary Purpose

Parkinson Disease, Amyotrophic Lateral Sclerosis

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AMDX2011P
Sponsored by
Amydis Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Parkinson Disease focused on measuring Parkinsons, ALS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

For Subjects with Parkinson's Disease

  1. 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).
  2. No suspected atypical parkinsonian syndromes due to drugs, metabolic disorders, encephalitis, or degenerative diseases.

    For Subjects with ALS

  3. Confirmed diagnosis of ALS with both upper and lower motor neuron involvement.

    For All Subjects

  4. Ability to undergo retinal imaging.
  5. 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.
  6. 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.

Exclusion Criteria:

  1. Presence of any underlying physical or psychological medical condition that would make it unlikely that the subject 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.
  5. 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).
  6. Use of any new prescription therapies or vaccines within 7 days prior to the study drug administration.
  7. 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.
  8. Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer.
  9. Females who are pregnant or breastfeeding.

Sites / Locations

  • Eye Research FoundationRecruiting
  • Brittany NIchollRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

AMDX2011P 25mg

AMDX2011P 50mg

AMDX2011P 100mg

AMDX2011P 200mg

Arm Description

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

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

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

AMDX2011P 200mg (6-8ml) single bolus injection intravenous for diagnostic review

Outcomes

Primary Outcome Measures

AMDX-2011P adverse events profile
Rate and nature of adverse events after a single intravenous (IV) dose of AMDX-2011P in subjects with neurodegenerative diseases (Parkinson's disease and ALS).

Secondary Outcome Measures

Detection of a-syn and TDP-43 deposits in retina
The presence of hyperfluorescent spots in retinal images
Concentration of AMDX-2011P
Peak Plasma Concentration (Cmax)
Pharmacokinetic Analysis of AMDX-2011P
Area under the plasma concentration versus time curve (AUC)

Full Information

First Posted
September 7, 2022
Last Updated
January 31, 2023
Sponsor
Amydis Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05542576
Brief Title
AMDX-2011P Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
Acronym
PROBE
Official Title
Prospective Randomized Open, Blinded Endpoint (PROBE) Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 24, 2022 (Actual)
Primary Completion Date
September 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amydis Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).
Detailed Description
This study will also evaluate the ability of AMDX-2011P to identify α-syn in the retina of patients with Parkinson's disease and to identify the protein TDP-43 in the retina of patients with ALS. This study will help to evaluate the ability to detect these protein deposits for the purpose of diagnosing PD and ALS. To determine the safest dose, participants will receive different amounts of the investigational retinal tracer. The first group of participants taking part in the study will receive a low dose of AMDX-2011P. If no major side effects occur, the dose will be increased for the next group of participants. Participants will receive a 1 time intravenous injection. This study plans to enroll approximately 24-36 subjects with active disease (PD or ALS). Participants will be age 18 and older.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson Disease, Amyotrophic Lateral Sclerosis
Keywords
Parkinsons, ALS

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Dose escalating via Cohorts total 1-4 cohorts
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
AMDX2011P 25mg
Arm Type
Experimental
Arm Description
25mg (1ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX2011P 50mg
Arm Type
Experimental
Arm Description
AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX2011P 100mg
Arm Type
Experimental
Arm Description
AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX2011P 200mg
Arm Type
Experimental
Arm Description
AMDX2011P 200mg (6-8ml) single bolus injection intravenous for diagnostic review
Intervention Type
Drug
Intervention Name(s)
AMDX2011P
Intervention Description
AMDX2011P single bolus injection intravenous for diagnostic review
Primary Outcome Measure Information:
Title
AMDX-2011P adverse events profile
Description
Rate and nature of adverse events after a single intravenous (IV) dose of AMDX-2011P in subjects with neurodegenerative diseases (Parkinson's disease and ALS).
Time Frame
1 week
Secondary Outcome Measure Information:
Title
Detection of a-syn and TDP-43 deposits in retina
Description
The presence of hyperfluorescent spots in retinal images
Time Frame
8 hours
Title
Concentration of AMDX-2011P
Description
Peak Plasma Concentration (Cmax)
Time Frame
8 hours
Title
Pharmacokinetic Analysis of AMDX-2011P
Description
Area under the plasma concentration versus time curve (AUC)
Time Frame
8 hours

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Masoud Mokhtarani, MD
Phone
310-229-5710
Email
masoud@amydis.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Masoud Mokhtarani, MD
Organizational Affiliation
Amydis Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Eye Research Foundation
City
Newport Beach
State/Province
California
ZIP/Postal Code
92663
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Linda Wirta
Phone
949-650-1863
Email
info@drwirta.com
Facility Name
Brittany NIcholl
City
Pasadena
State/Province
California
ZIP/Postal Code
91107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brittany Nicholl
Phone
626-305-9100
Email
bnicholl@azulvision.com

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
There is no plan to share individual participant data at this time.
Links:
URL
http://probeclinicaltrial.com
Description
IRB approved potential participant information website

Learn more about this trial

AMDX-2011P Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy

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