Parkinson's DiseasePhase 1NCT02438215
Study of IRX4204 for Treatment of Early Parkinson's Disease
A Phase 1 study testing IRX4204 in people with Parkinson's Disease, run by Io Therapeutics at 1 site in United States.
Trial summary
This is a single site, open-label study designed to examine dopamine transporter density using [123I]β-CIT SPECT imaging before and following treatment with IRX4204 for a 30-day period in early Parkinson's disease patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- IRX4204
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2014to May 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Participant is 40-80 years of age, inclusive.
- Participant has a clinical diagnosis of PD based on the UK Brain Bank Criteria.
- Participant has Hoehn and Yahr stage < 3.
- Participant may be treated with PD symptomatic therapy on a stable dose for at least 30 days prior to the Screening Visit. Dose levels of PD symptomatic therapies will remain stable through the patient's participation in the study, unless a change of dose level is indicated because of adverse events.
- Participant must be willing and able to provide informed consent.
- Females must be of either non-child bearing potential based on:
- post-menopausal for at least 2 years, or
- surgically sterilized If of child bearing potential, must be neither pregnant or breastfeeding at Screening, and must be willing to avoid pregnancy by using medically accepted contraception (use of an intrauterine device or use of a double barrier method when engaging in sexual intercourse with a male partner) for 4 weeks prior to and 4 weeks following the last dose of study medication.
Who cannot join
- Has any form of parkinsonism other than idiopathic PD
- Are currently experiencing motor fluctuations (end of dose wearing off or dyskinesias) reflective of later stage PD
- Has evidence of dementia or significant cognitive dysfunction
- Has clinically significant abnormal laboratory value and/or clinically significant unstable medical or psychiatric illness.
- The subject has any disorder that may interfere with drug absorption, distribution, metabolism or excretion.
- The subject has evidence of clinically significant gastrointestinal, cardiovascular, hepatic, renal, hematological, neoplastic, endocrine, alternative neurological, immunodeficiency, pulmonary, or other disorder or disease.
- Pregnancy or breastfeeding
Trial locations and contacts
1 site in 1 country.
United States1 site
Molecular NeuroImaging, [MNI]
New Haven, Connecticut, 06510
About this study
Official title: An Open-Label, Single Site Study Using [123I]β-CIT Single Photon Emission Tomography (SPECT) to Evaluate Dopamine Transporter Binding Following Treatment With IRX4204 in Early Parkinson's Disease Subjects
This is a single site, open-label study designed to examine dopamine transporter density using [123I]β-CIT SPECT imaging before and following treatment with IRX4204 for a 30-day period in early Parkinson's disease patients. In addition, clinical evaluations will be performed to evaluate the effect of IRX4204 treatment on the motor and cognitive symptoms of PD.
Read the detailed description
Fifteen patients with early PD were enrolled in this open label study, in 3 cohorts of 5 patients each, treated with IRX4204 at 5 mg/day, 10mg/day, or 20 mg/day. Patients were administered IRX4204 orally once daily. Baseline assessments were performed for total motor score, and Unified Parkinson's Disease Rating Scale (UPDRS). Follow-up assessments of these clinical outcome measures were performed at 14 and 29 days of treatment. [123]β-CIT SPECT imaging for assessment of dopamine active transporter (DAT) expression was performed at baseline, and on day 30 of IRX4204 treatment. Patients had clinical hematology and chemistry laboratory tests, and recording of adverse events, performed at baseline and at follow up visits.
- Conditions
- Parkinson's Disease
- Being tested
- IRX4204
- Keywords
- PD, Parkinson's Disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IRX4204Drug
Also called NRX194204
IRX4204 is a potent and highly selective orally available and brain penetrant RXR nuclear receptor agonist small compound administered as gel capsules.
| Study group | Role | What they receive |
|---|---|---|
| IRX4204 | Experimental | IRX4204 20 mg QD for Days 1-30 Receives: Drug: IRX4204 |
What is being measured
The primary outcome is the main question the study is designed to answer.
striatal binding ratio (SBR)
The percent change from baseline to end of dosing period (Day 30) of the striatal binding ratio (SBR)
Measured: 30 days
Secondary outcomes (2)
Total Motor and UPDRS scores
The change in motor and UPDRS scores to end of dosing period (Day 30)
Measured: 30 days
Safety including hematology and chemistry laboratories, vital signs, and adverse events
Clinically significant changes in hematology and chemistry laboratories, vital signs, and frequency of adverse events
Measured: 30 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2015 (estimated)
- Study startAug 2014
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Io Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ken Marek, MD, Principal investigator, Molecular NeuroImaging, [MNI]
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02438215
- Sponsor's protocol ID
- IRX4204-001
- Status last verified
- May 2015
- First submitted
- 7 Aug 2014
- Last updated
- 12 May 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Single Site Study Using [123I]β-CIT Single Photon Emission Tomography (SPECT) to Evaluate Dopamine Transporter Binding Following Treatment With IRX4204 in Early Parkinson's Disease Subjects
- ClinicalTrials.gov ID
- NCT02438215
- Acronym / sponsor ID
- IRX4204-001
- Conditions
- Parkinson's Disease
- Keywords
- PD, Parkinson's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Io TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Aug 2014
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 8 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single site, open-label study designed to examine dopamine transporter density using [123I]β-CIT SPECT imaging before and following treatment with IRX4204 for a 30-day period in early Parkinson's disease patients. In addition, clinical evaluations will be performed to evaluate the effect of IRX4204 treatment on the motor and cognitive symptoms of PD.
Detailed description
Fifteen patients with early PD were enrolled in this open label study, in 3 cohorts of 5 patients each, treated with IRX4204 at 5 mg/day, 10mg/day, or 20 mg/day. Patients were administered IRX4204 orally once daily. Baseline assessments were performed for total motor score, and Unified Parkinson's Disease Rating Scale (UPDRS). Follow-up assessments of these clinical outcome measures were performed at 14 and 29 days of treatment. [123]β-CIT SPECT imaging for assessment of dopamine active transporter (DAT) expression was performed at baseline, and on day 30 of IRX4204 treatment. Patients had clinical hematology and chemistry laboratory tests, and recording of adverse events, performed at baseline and at follow up visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IRX4204 | Experimental | Drug: IRX4204IRX4204 20 mg QD for Days 1-30 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Participant is 40-80 years of age, inclusive.
- Participant has a clinical diagnosis of PD based on the UK Brain Bank Criteria.
- Participant has Hoehn and Yahr stage < 3.
- Participant may be treated with PD symptomatic therapy on a stable dose for at least 30 days prior to the Screening Visit. Dose levels of PD symptomatic therapies will remain stable through the patient's participation in the study, unless a change of dose level is indicated because of adverse events.
- Participant must be willing and able to provide informed consent.
Key exclusion criteria (7 listed)
- Has any form of parkinsonism other than idiopathic PD
- Are currently experiencing motor fluctuations (end of dose wearing off or dyskinesias) reflective of later stage PD
- Has evidence of dementia or significant cognitive dysfunction
- Has clinically significant abnormal laboratory value and/or clinically significant unstable medical or psychiatric illness.
- The subject has any disorder that may interfere with drug absorption, distribution, metabolism or excretion.
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 |
|---|---|---|
| IRX4204Also: NRX194204IRX4204 is a potent and highly selective orally available and brain penetrant RXR nuclear receptor agonist small compound administered as gel capsules. | Drug | IRX4204 |
Primary outcome
striatal binding ratio (SBR)
Time frame: 30 days
The percent change from baseline to end of dosing period (Day 30) of the striatal binding ratio (SBR)
2 secondary outcomes
Total Motor and UPDRS scores
Time frame: 30 days
Safety including hematology and chemistry laboratories, vital signs, and adverse events
Time frame: 30 Days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2015.
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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.
Ken Marek, MD principal investigator
Molecular NeuroImaging, [MNI]
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IRX4204 in people with Parkinson's Disease, run by Io Therapeutics at 1 site 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 8 inclusion and 7 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 12 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
striatal binding ratio (SBR) (time frame: 30 days). 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 12 May 2015.