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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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.

Tracking Information
First Submitted Date  ICMJE May 6, 2015
First Posted Date  ICMJE May 15, 2015
Last Update Posted Date October 4, 2016
Study Start Date  ICMJE August 2015
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 12, 2015)
Number of patients with adverse events [ Time Frame: 28 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02445794 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 12, 2015)
  • Pharmacokinetics - Area under the concentration-time curve after a single dose [ Time Frame: 24 hours ]
  • Pharmacokinetics - Maximum observed plasma concentration after a single dose [ Time Frame: 24 hours ]
  • Pharmacokinetics - Time to reach maximum plasma concentration after a single dose [ Time Frame: 24 hours ]
  • Pharmacokinetics - Area under the concentration-time curve during a dosing interval at steady state [ Time Frame: 72 hours ]
  • Pharmacokinetics - Maximum observed plasma concentration at steady state [ Time Frame: 72 hours ]
  • Pharmacokinetics - Time to reach maximum plasma concentration at steady state [ Time Frame: 72 hours ]
  • Pharmacokinetics - Time to reach minimum plasma concentration at steady state [ Time Frame: 72 hours ]
  • Pharmacokinetics - Minimum observed plasma concentration at steady state [ Time Frame: 72 hours ]
  • Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity [ Time Frame: 72 hours ]
  • Pharmacokinetics - Area under the concentration-time curve from time 0 to the last measurable time point [ Time Frame: 72 hours ]
  • Pharmacokinetics - Apparent total plasma clearance after oral administration [ Time Frame: 72 hours ]
  • Pharmacokinetics - Elimination rate constant [ Time Frame: 72 hours ]
  • Pharmacokinetics - Terminal half-life estimation [ Time Frame: 72 hours ]
  • Pharmacokinetics - Apparent volume of distribuation after oral dosing at steady state [ Time Frame: 72 hours ]
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A First in Human Study of RT001 in Patients With Friedreich's Ataxia
Official Title  ICMJE A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia
Brief Summary The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE
  • 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.
Study Arms  ICMJE
  • Experimental: RT001, oral, 1.8 g/day
    RT001, oral, 1.8 g QD for 28 days or matching comparator
    Interventions:
    • Drug: RT001
    • Drug: RT001 comparator
  • Experimental: RT001, oral, 9 g/day
    RT001, oral, 4.5 g BID for 28 days or matching comparator
    Interventions:
    • Drug: RT001
    • Drug: RT001 comparator
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 20, 2016)
19
Original Estimated Enrollment  ICMJE
 (submitted: May 12, 2015)
18
Actual Study Completion Date  ICMJE July 2016
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 50 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02445794
Other Study ID Numbers  ICMJE RT001-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Retrotope, Inc.
Study Sponsor  ICMJE Retrotope, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Curtis Scribner, MD Retrotope, Inc.
Principal Investigator: Theresa Zesiewicz, MD USF Ataxia Research Center
PRS Account Retrotope, Inc.
Verification Date October 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP