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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05542576
Recruitment Status : Recruiting
First Posted : September 15, 2022
Last Update Posted : October 26, 2022
Sponsor:
Information provided by (Responsible Party):
Amydis Inc.

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).

Condition or disease Intervention/treatment Phase
Parkinson Disease Amyotrophic Lateral Sclerosis Drug: AMDX2011P Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Dose escalating via Cohorts total 1-4 cohorts
Masking: None (Open Label)
Primary Purpose: Diagnostic
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
Actual Study Start Date : August 24, 2022
Estimated Primary Completion Date : November 30, 2022
Estimated Study Completion Date : February 28, 2023


Arm Intervention/treatment
Experimental: AMDX2011P 25mg
25mg (1ml) single bolus injection intravenous for diagnostic review
Drug: AMDX2011P
AMDX2011P single bolus injection intravenous for diagnostic review

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

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

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




Primary Outcome Measures :
  1. AMDX-2011P adverse events profile [ Time Frame: 1 week ]
    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 :
  1. Detection of a-syn and TDP-43 deposits in retina [ Time Frame: 8 hours ]
    The presence of hyperfluorescent spots in retinal images

  2. Concentration of AMDX-2011P [ Time Frame: 8 hours ]
    Peak Plasma Concentration (Cmax)

  3. Pharmacokinetic Analysis of AMDX-2011P [ Time Frame: 8 hours ]
    Area under the plasma concentration versus time curve (AUC)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05542576


Contacts
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Contact: Masoud Mokhtarani, MD 310-229-5710 masoud@amydis.com

Locations
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United States, California
Eye Research Foundation Recruiting
Newport Beach, California, United States, 92663
Contact: David Wirta, MD    949-650-1863    info@drwirta.com   
California Eye Specialists Recruiting
Pasadena, California, United States, 91107
Contact: Payam Amini, MD    626-793-4168      
Sponsors and Collaborators
Amydis Inc.
Investigators
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Study Chair: Masoud Mokhtarani, MD Amydis Inc.
Additional Information:
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Responsible Party: Amydis Inc.
ClinicalTrials.gov Identifier: NCT05542576    
Other Study ID Numbers: AMDX-2011P
First Posted: September 15, 2022    Key Record Dates
Last Update Posted: October 26, 2022
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: There is no plan to share individual participant data at this time.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amydis Inc.:
Parkinsons
ALS
Additional relevant MeSH terms:
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Parkinson Disease
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Synucleinopathies
Neuromuscular Diseases
Spinal Cord Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases