Study of IRX4204 for Treatment of Early Parkinson's Disease

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.

Study Overview

• Status: Completed
• Conditions: Parkinson's Disease
• Intervention / Treatment: Drug: IRX4204

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 Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Molecular NeuroImaging, [MNI]

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Participant is 40-80 years of age, inclusive.
2. Participant has a clinical diagnosis of PD based on the UK Brain Bank Criteria.
3. Participant has Hoehn and Yahr stage < 3.
4. 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.
5. Participant must be willing and able to provide informed consent.

6. 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.

Exclusion Criteria:

1. Has any form of parkinsonism other than idiopathic PD
2. Are currently experiencing motor fluctuations (end of dose wearing off or dyskinesias) reflective of later stage PD
3. Has evidence of dementia or significant cognitive dysfunction
4. Has clinically significant abnormal laboratory value and/or clinically significant unstable medical or psychiatric illness.
5. The subject has any disorder that may interfere with drug absorption, distribution, metabolism or excretion.
6. The subject has evidence of clinically significant gastrointestinal, cardiovascular, hepatic, renal, hematological, neoplastic, endocrine, alternative neurological, immunodeficiency, pulmonary, or other disorder or disease.
7. Pregnancy or breastfeeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IRX4204; IRX4204 20 mg QD for Days 1-30	Drug: IRX4204; IRX4204 is a potent and highly selective orally available and brain penetrant RXR nuclear receptor agonist small compound administered as gel capsules.; Other Names: NRX194204

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
striatal binding ratio (SBR)	The percent change from baseline to end of dosing period (Day 30) of the striatal binding ratio (SBR)	30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total Motor and UPDRS scores	The change in motor and UPDRS scores to end of dosing period (Day 30)	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	30 Days

Collaborators and Investigators

• Sponsor: Io Therapeutics
• Investigators: Principal Investigator: Ken Marek, MD, Molecular NeuroImaging, [MNI]

Study record dates

Study Major Dates

• Study Start: August 1, 2014
• Primary Completion (Actual): May 1, 2015
• Study Completion (Actual): May 1, 2015

Study Registration Dates

• First Submitted: August 7, 2014
• First Submitted That Met QC Criteria: May 5, 2015
• First Posted (Estimate): May 8, 2015

Study Record Updates

• Last Update Posted (Estimate): May 12, 2015
• Last Update Submitted That Met QC Criteria: May 7, 2015
• Last Verified: May 1, 2015

More Information

Terms related to this study

• Keywords: Parkinson's Disease; PD
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: IRX4204-001