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
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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.
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05542576
- Acronym / sponsor ID
- PROBE / AMDX-2011P-101
- Conditions
- Parkinson Disease, Amyotrophic Lateral Sclerosis
- Keywords
- Parkinsons, ALS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Amydis Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Aug 2022 (actual)
- Primary completion
- 15 Mar 2023 (actual)
- Study completion
- 15 Mar 2023 (actual)
- First posted
- 15 Sep 2022 (actual)
- Results
- Posted 20 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Dose escalating via Cohorts total 1-3 cohorts
| Arm | Type | Receives |
|---|---|---|
| AMDX2011P 25mg | Experimental | Drug: AMDX2011P25mg (1ml) single bolus injection intravenous for diagnostic review |
| AMDX2011P 50mg | Experimental | Drug: AMDX2011PAMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review |
| AMDX2011P 100mg | Experimental | Drug: AMDX2011PAMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMDX2011PAMDX2011P single bolus injection intravenous for diagnostic review | Drug | AMDX2011P 100mg, AMDX2011P 25mg, AMDX2011P 50mg |
Primary outcome
AMDX-2011P Adverse Events Profile
Time frame: 1 week
Incidence of Treatment Emergent Adverse Events (TEAEs) at for each cohort (dose level).
2 secondary outcomes
Concentration of AMDX-2011P
Time frame: 8 hours
Pharmacokinetic Analysis of AMDX-2011P
Time frame: 8 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. There is no plan to share individual participant data at this time.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Masoud Mokhtarani, MD study chair
Amydis Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AMDX-2011P Adverse Events Profile (time frame: 1 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2025.