Friedreich's AtaxiaPhase 1/2NCT02445794
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
A Phase 1/2 study testing Low dose cohort and High dose cohort in people with Friedreich's Ataxia, run by Biojiva LLC at 2 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- Low dose cohort and High dose cohort
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2015to Jul 2016
- 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 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female 18 to 50 years of age
- Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
- Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
- FARS-Neurological score of 20-90 points
- Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
- Body Mass Index ≤ 29.9 kg/m2
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- Agrees to dietary restrictions and agrees to receive calls from a dietary coach
- Signed the informed consent form prior to entry into the study
- Agrees to spend the required number of overnight clinic days
- Able to provide the necessary repeated blood samples
Who cannot join
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Known point mutation in the FXN gene
- History of malignancies (other than basal cell carcinomas)
- Impaired renal function at screening
- Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values > 2 x upper limit of normal (ULN) at screening
- 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
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- Female who is breastfeeding or has a positive pregnancy test
- 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
- Unwilling or unable to comply with the requirements of the protocol
- Clinically significant cardiac abnormalities at screening that, in the opinion of the Investigator, would make the patient unsuitable for enrollment
- Diabetes mellitus (Type 1 or 2)
- Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale
- History, within the last 2 years, of alcohol abuse, significant mental illness, or physical opioid dependence
- Cannot adhere to the dietary guidance required to be followed by the protocol
- Cannot take the medication due to impairment in swallowing capsules
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Collaborative Neuroscience Network, LLC
Long Beach, California, 90806
University of South Florida
Tampa, Florida, 33612
About this study
Official title: A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
Read the 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.
- Conditions
- Friedreich's Ataxia
- Being tested
- Low dose cohort, High dose cohort
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low dose cohortDrug
Also called RT001 1.8 g/d (2 capsule per day), RT001 comparator 1.8 g/d (2 capsule per day)
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.
High dose cohortDrug
Also called RT001 9.0 g/d (9 capsule per day), RT001 comparator 9.0 g/d (9 capsule per day)
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.
| Study group | Role | What they receive |
|---|---|---|
| RT001, oral, 1.8 g/day | Experimental | RT001, oral, 1.8 g QD for 28 days or matching comparator Receives: Drug: Low dose cohort; Drug: High dose cohort |
| RT001, oral, 9 g/day | Experimental | RT001, oral, 4.5 g BID for 28 days or matching comparator Receives: Drug: Low dose cohort; Drug: High dose cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Adverse Events
Measured: 28 days
Secondary outcomes (8)
Pharmacokinetics - Area Under the Concentration-time Curve After a Single Dose
AUC 0-24 hours post-dose (Hours -1.0 to -0.5 (pre-breakfast, pre-dose), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 2: 24 hours following dosing on Day 1 (± 30 min; pre-breakfast, pre-dose) was measured for the low and high dose cohorts after a single dose of RT001
Measured: 24 hours
Pharmacokinetics - Maximum Observed Plasma Concentration After a Single Dose
Plasma levels were measured for the following time points: Day 1: Hours -1.0 to -0.5 (pre-breakfast, pre-dose), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 2: 24 hours following dosing on Day 1 (± 30 min; pre-breakfast, pre-dose) PK curves were constructed from these data and CMax measured on the curves for the low and high dose cohorts
Measured: 24 hours
Pharmacokinetics - Time to Reach Maximum Plasma Concentration After a Single Dose
TMax measured for the low and high dose cohorts
Measured: 24 hours
Pharmacokinetics - Maximum Observed Plasma Concentration After Final Dose on Day 28
After 28 days of dosing, the final dose of RT001 was administered, and PK samples were obtained at the following timepoints (all timepoints refer to final dose on Day 28):
Day 28: Hours -1.0 to -0.5 (pre-breakfast, pre-dose on Day 28), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 29: Hours 24 (± 30 min; pre-breakfast) and 32 (± 30 min) hours following final dose on Day 28 Day 30: 48 hours following final dose on Day 28 (± 30 min; pre-breakfast) Day 31: 72 hours following final dose on Day 28 (± 30 min; pre-breakfast) PK curves were constructed, and CMax at 28 days was determined from these curves
Measured: Day 28-Day 31 (3 days)
Pharmacokinetics - Terminal Half-life Estimation After Final Dose on Day 28
After 28 days of dosing, the final dose of RT001 was administered, and PK samples were obtained at the following timepoints (all timepoints refer to final dose on Day 28):
Day 28: Hours -1.0 to -0.5 (pre-breakfast, pre-dose on Day 28), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 29: Hours 24 (± 30 min; pre-breakfast) and 32 (± 30 min) hours following final dose on Day 28 Day 30: 48 hours following final dose on Day 28 (± 30 min; pre-breakfast) Day 31: 72 hours following final dose on Day 28 (± 30 min; pre-breakfast) PK curves were constructed, and T1/2 at 28 days was determined from these curves
Measured: Day 28-Day 31 (3 days)
Change From Baseline at 28 Days in the Timed 25 Foot Walk (T25FW)
The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk.
T25FW was measured at baseline and at 28 days. These data were compared.
Measured: 28 days
Change From Baseline at 28 Days in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score (Minimum Score 0, Maximum Score 125, Lower is Better)
The FARS-neurological rating scale specifically developed and validated for Friedreich's Ataxia. The FARS-Neurological included evaluations of the neurological signs that specifically reflect neural substrates affected in patients with FA. Based on a neurological examination bulbar (11 points), upper limb coordination (36 points), lower limb coordination (16 points), peripheral nervous system (26 points), and upright stability (36 points) functions were assessed for individual sub-scores (11, 36, 16, 26, and 36) with a maximum score of 125 (Friedreich's Ataxia Study Group, Subramony et al., 2005, Lynch et al., 2006). FARS-Neurologic examinations were conducted by a qualified physician or health professional trained in the use of the FARS format. A lower score is better. The minimum score is 0, the maximum score is 125.
Measured: 28 days
Change From Baseline at 28 Days in Peak Workload for the Treated Population vs. the Comparator Population
Peak workload was measured using cardiopulmonary exercise testing at baseline and after 28 days of treatment. The results of treatment were compared to baseline examination.
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2015 (estimated)
- Study startAug 2015
- Primary completionJun 2016
- Study completionJul 2016
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 Friedreich's AtaxiaStudies 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biojiva LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Curtis Scribner, MD, Study director, Biojiva LLCTheresa Zesiewicz, MD, Principal investigator, USF Ataxia Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02445794
- Sponsor's protocol ID
- RT001-002
- Status last verified
- Sep 2020
- First submitted
- 6 May 2015
- Last updated
- 27 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT02445794
- Acronym / sponsor ID
- RT001-002
- Conditions
- Friedreich's Ataxia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Biojiva LLCIndustry (pharma, biotech, devices)
- Study start
- Aug 2015
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 15 May 2015 (estimated)
- Results
- Posted 27 Nov 2020
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 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RT001, oral, 1.8 g/day | Experimental | Drug: Low dose cohort, Drug: High dose cohortRT001, oral, 1.8 g QD for 28 days or matching comparator |
| RT001, oral, 9 g/day | Experimental | Drug: Low dose cohort, Drug: High dose cohortRT001, oral, 4.5 g BID for 28 days or matching comparator |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Male or female 18 to 50 years of age
- Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
- Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
- FARS-Neurological score of 20-90 points
- Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
Key exclusion criteria (15 listed)
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Known point mutation in the FXN gene
- History of malignancies (other than basal cell carcinomas)
- Impaired renal function at screening
- Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values > 2 x upper limit of normal (ULN) at screening
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 |
|---|---|---|
| Low dose cohortAlso: RT001 1.8 g/d (2 capsule per day), RT001 comparator 1.8 g/d (2 capsule per day)RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001. | Drug | RT001, oral, 1.8 g/day, RT001, oral, 9 g/day |
| High dose cohortAlso: RT001 9.0 g/d (9 capsule per day), RT001 comparator 9.0 g/d (9 capsule per day)RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester. | Drug | RT001, oral, 1.8 g/day, RT001, oral, 9 g/day |
Primary outcome
Number of Patients With Adverse Events
Time frame: 28 days
8 secondary outcomes
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 - Maximum Observed Plasma Concentration After Final Dose on Day 28
Time frame: Day 28-Day 31 (3 days)
Pharmacokinetics - Terminal Half-life Estimation After Final Dose on Day 28
Time frame: Day 28-Day 31 (3 days)
Change From Baseline at 28 Days in the Timed 25 Foot Walk (T25FW)
Time frame: 28 days
Change From Baseline at 28 Days in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score (Minimum Score 0, Maximum Score 125, Lower is Better)
Time frame: 28 days
Change From Baseline at 28 Days in Peak Workload for the Treated Population vs. the Comparator Population
Time frame: 28 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Curtis Scribner, MD study director
Biojiva LLC
Theresa Zesiewicz, MD principal investigator
USF Ataxia Research Center
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 Low dose cohort and High dose cohort in people with Friedreich's Ataxia, run by Biojiva LLC 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 10 inclusion and 15 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 27 Nov 2020. 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 27 Nov 2020). 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?
Number of Patients With Adverse Events (time frame: 28 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 27 Nov 2020.