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A First in Human Study of RT001 in Patients With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT02445794
Recruitment Status : Completed
First Posted : May 15, 2015
Last Update Posted : October 4, 2016
Sponsor:
Information provided by (Responsible Party):
Retrotope, Inc.

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: RT001 Drug: RT001 comparator Phase 1 Phase 2

Detailed Description:
Study RT001-002 is a randomized, double-blind, controlled, ascending dose study to evaluate the safety, tolerability, pharmacokinetic, disease state, and exploratory endpoints in patients with Friedreich's ataxia after oral administration. The study includes 2 dose levels of RT001.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia
Study Start Date : August 2015
Actual Primary Completion Date : June 2016
Actual Study Completion Date : July 2016

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Arms and Interventions
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Arm Intervention/treatment
Experimental: RT001, oral, 1.8 g/day
RT001, oral, 1.8 g QD for 28 days or matching comparator
 Drug: RT001
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.

Drug: RT001 comparator
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.
Experimental: RT001, oral, 9 g/day
RT001, oral, 4.5 g BID for 28 days or matching comparator
 Drug: RT001
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.

Drug: RT001 comparator
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.


Outcome Measures
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Primary Outcome Measures :
  1. Number of patients with adverse events [ Time Frame: 28 days ]

Secondary Outcome Measures :
  1. Pharmacokinetics - Area under the concentration-time curve after a single dose [ Time Frame: 24 hours ]
  2. Pharmacokinetics - Maximum observed plasma concentration after a single dose [ Time Frame: 24 hours ]
  3. Pharmacokinetics - Time to reach maximum plasma concentration after a single dose [ Time Frame: 24 hours ]
  4. Pharmacokinetics - Area under the concentration-time curve during a dosing interval at steady state [ Time Frame: 72 hours ]
  5. Pharmacokinetics - Maximum observed plasma concentration at steady state [ Time Frame: 72 hours ]
  6. Pharmacokinetics - Time to reach maximum plasma concentration at steady state [ Time Frame: 72 hours ]
  7. Pharmacokinetics - Time to reach minimum plasma concentration at steady state [ Time Frame: 72 hours ]
  8. Pharmacokinetics - Minimum observed plasma concentration at steady state [ Time Frame: 72 hours ]
  9. Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity [ Time Frame: 72 hours ]
  10. Pharmacokinetics - Area under the concentration-time curve from time 0 to the last measurable time point [ Time Frame: 72 hours ]
  11. Pharmacokinetics - Apparent total plasma clearance after oral administration [ Time Frame: 72 hours ]
  12. Pharmacokinetics - Elimination rate constant [ Time Frame: 72 hours ]
  13. Pharmacokinetics - Terminal half-life estimation [ Time Frame: 72 hours ]
  14. Pharmacokinetics - Apparent volume of distribuation after oral dosing at steady state [ Time Frame: 72 hours ]
  15. Change from baseline in the Timed 25 Foot Walk (T25FW) [ Time Frame: 28 days ]
    The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk.

  16. Change from baseline in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score [ Time Frame: 28 days ]
    The FARS is neurological rating scale specifically developed and validated for Friedreich's Ataxia.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female 18 to 50 years of age
  2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
  3. Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
  4. FARS-Neurological score of 20-90 points
  5. Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
  6. Body Mass Index ≤ 29.9 kg/m2
  7. Agrees to dietary restrictions and agrees to receive calls from a dietary coach
  8. Signed the informed consent form prior to entry into the study
  9. Agrees to spend the required number of overnight clinic days
  10. Able to provide the necessary repeated blood samples

Exclusion Criteria:

  1. Received treatment with other experimental therapies within the last 30 days prior to the first dose
  2. Known point mutation in the FXN gene
  3. History of malignancies (other than basal cell carcinomas)
  4. Impaired renal function at screening
  5. Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values > 2 x upper limit of normal (ULN) at screening
  6. Known hepatitis B surface antigen (HBsAg)-positive, or known or suspected active hepatitis C infection, or is known to be human immunodeficiency virus (HIV) positive
  7. Female who is breastfeeding or has a positive pregnancy test
  8. Male participant or female participant of child bearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study
  9. Unwilling or unable to comply with the requirements of the protocol
  10. Clinically significant cardiac abnormalities at screening that, in the opinion of the Investigator, would make the patient unsuitable for enrollment
  11. Diabetes mellitus (Type 1 or 2)
  12. Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale
  13. History, within the last 2 years, of alcohol abuse, significant mental illness, or physical opioid dependence
  14. Cannot adhere to the dietary guidance required to be followed by the protocol
  15. Cannot take the medication due to impairment in swallowing capsules
Contacts and Locations
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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): NCT02445794


Locations
United States, California
Collaborative Neuroscience Network, LLC
Long Beach, California, United States, 90806
United States, Florida
University of South Florida
Tampa, Florida, United States, 33612
Sponsors and Collaborators
Retrotope, Inc.
Investigators
Study Director: Curtis Scribner, MD Retrotope, Inc.
Principal Investigator: Theresa Zesiewicz, MD USF Ataxia Research Center
More Information
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Responsible Party: Retrotope, Inc.
ClinicalTrials.gov Identifier: NCT02445794     History of Changes
Other Study ID Numbers: RT001-002
First Posted: May 15, 2015    Key Record Dates
Last Update Posted: October 4, 2016
Last Verified: October 2016

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
 Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases