Parkinson DiseasePhase 1/2PROBENCT05542576
Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
A Phase 1/2 study testing AMDX2011P in people with Parkinson Disease and Amyotrophic Lateral Sclerosis, run by Amydis Inc. at 2 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Parkinson Disease, Amyotrophic Lateral Sclerosis
- What is being tested?
- AMDX2011P
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Aug 2022to 15 Mar 2023
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Eye Research Foundation
Newport Beach, California, 92663
Brittany NIcholl
Pasadena, California, 91107
About this study
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
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).
Read the detailed description
The primary objective of this study is to evaluate the safety and tolerability of three different doses of AMDX-2011P (25mg, 50mg, or 100mg) given as a single intravenous dose in patients with neurodegenerative diseases (Parkinson's disease and ALS). The first cohort of participants taking part in the study will receive the lowest dose of AMDX-2011P (25mg). If no major side effects occur, the dose will be increased for the next group of participants to 50mg and then to 100mg.
Secondary objective of this study is to characterize the pharmacokinetic (PK) profile of AMDX-2011P and AMDX-2011.
Exploratory evaluations of the biological activity of AMDX-2011P in the retina will be performed by imaging.
- Conditions
- Parkinson Disease, Amyotrophic Lateral Sclerosis
- Being tested
- AMDX2011P
- Keywords
- Parkinsons, ALS
- Medical subject headings
- Parkinson Disease, Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMDX2011PDrug
AMDX2011P single bolus injection intravenous for diagnostic review
| Study group | Role | What they receive |
|---|---|---|
| AMDX2011P 25mg | Experimental | 25mg (1ml) single bolus injection intravenous for diagnostic review Receives: Drug: AMDX2011P |
| AMDX2011P 50mg | Experimental | AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review Receives: Drug: AMDX2011P |
| AMDX2011P 100mg | Experimental | AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review Receives: Drug: AMDX2011P |
What is being measured
The primary outcome is the main question the study is designed to answer.
AMDX-2011P Adverse Events Profile
Incidence of Treatment Emergent Adverse Events (TEAEs) at for each cohort (dose level).
Measured: 1 week
Secondary outcomes (2)
Concentration of AMDX-2011P
Peak Plasma Concentration (Cmax)
Measured: 8 hours
Pharmacokinetic Analysis of AMDX-2011P
Area under the plasma concentration versus time curve (AUC)
Measured: 8 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2022
- Study start24 Aug 2022
- Primary completion15 Mar 2023
- Study completion15 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Dose escalating via Cohorts total 1-3 cohorts
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amydis Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Masoud Mokhtarani, MD, Study chair, Amydis Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05542576
- Sponsor's protocol ID
- AMDX-2011P-101
- Acronym
- PROBE
- Status last verified
- Aug 2025
- First submitted
- 7 Sep 2022
- Last updated
- 20 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share individual participant data at this time.