Infantile Neuroaxonal DystrophyPhase 2/3NCT03570931
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
A Phase 2/3 study testing RT001 in people with Infantile Neuroaxonal Dystrophy, run by Biojiva LLC at 2 sites in United States.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD). Read more about this study
- Who is it for?
- Ages 18 months to 10 years
- Conditions the study is for
- Infantile Neuroaxonal Dystrophy
- What is being tested?
- RT001
- 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
- 5 Nov 2018to 30 Jun 2022
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 months to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female 18 months to 10 years of age
- Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
- Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
- Must have impairment in at least 2 of the assessed categories at baseline
- Signed informed consent form (ICF) prior to entry into the study
- Able to provide the necessary blood samples
Who cannot join
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
- Have a life expectancy of less than one year
- Diagnosis of atypical NAD (ANAD)
- Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Jacobs Levy Genomics and Research Program
Morristown, New Jersey, 07960
University of California San Francisco, Benioff Children's Hospital
San Francisco, California, 94158
About this study
Official title: A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
Read the detailed description
This is a single arm open-label study with a structured observation of INAD patients treated with RT001. Enrolled subjects will undergo observation and testing to determine the effect of RT001 treatment. Fifteen to twenty eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.
- Conditions
- Infantile Neuroaxonal Dystrophy
- Being tested
- RT001
- Keywords
- INAD, Infantile Neuroaxonal Dystrophy
- Medical subject headings
- Neuroaxonal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RT001Drug
Also called Deuterated linoleic acid
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.
| Study group | Role | What they receive |
|---|---|---|
| RT001 | Experimental | RT001, oral, 3.84 g/day Receives: Drug: RT001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Modified Ashworth Spasticity Scale
Change from baseline in the Modified Ashworth spasticity scale.
Measured: 12 months
Secondary outcomes (2)
INAD Progression Composite
Change from baseline in an INAD composite score to assess the overall treatment effect on the most progressive aspects of the disease
Measured: 12 months
Progression Free Survival Time
Progression free survival time (mortality or pneumonia)
Measured: All available data
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2018
- Study start5 Nov 2018
- Primary completion9 Aug 2020
- Study completion30 Jun 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile Neuroaxonal DystrophyStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single arm open-label study
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biojiva LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter Milner, MD, Study director, Chief Medical Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03570931
- Sponsor's protocol ID
- RT001-008
- Status last verified
- Oct 2021
- First submitted
- 11 Jun 2018
- Last updated
- 14 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
- ClinicalTrials.gov ID
- NCT03570931
- Acronym / sponsor ID
- RT001-008
- Conditions
- Infantile Neuroaxonal Dystrophy
- Keywords
- INAD, Infantile Neuroaxonal Dystrophy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Biojiva LLCIndustry (pharma, biotech, devices)
- Study start
- 5 Nov 2018 (actual)
- Primary completion
- 9 Aug 2020 (actual)
- Study completion
- 30 Jun 2022 (estimated)
- First posted
- 27 Jun 2018 (actual)
- 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.
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
Detailed description
This is a single arm open-label study with a structured observation of INAD patients treated with RT001. Enrolled subjects will undergo observation and testing to determine the effect of RT001 treatment. Fifteen to twenty eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm open-label study
| Arm | Type | Receives |
|---|---|---|
| RT001 | Experimental | Drug: RT001RT001, oral, 3.84 g/day |
Who may qualify
- Ages
- 18 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male or female 18 months to 10 years of age
- Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
- Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
- Must have impairment in at least 2 of the assessed categories at baseline
- Signed informed consent form (ICF) prior to entry into the study
Key exclusion criteria (5 listed)
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
- Have a life expectancy of less than one year
- Diagnosis of atypical NAD (ANAD)
- Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled
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 |
|---|---|---|
| RT001Also: Deuterated linoleic acidRT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001. | Drug | RT001 |
Primary outcome
Modified Ashworth Spasticity Scale
Time frame: 12 months
Change from baseline in the Modified Ashworth spasticity scale.
2 secondary outcomes
INAD Progression Composite
Time frame: 12 months
Progression Free Survival Time
Time frame: All available data
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2021.
- Individual participant dataSharing planned: Undecided
Related trials for Infantile Neuroaxonal Dystrophy
RecruitingObservational study
TIRCON International NBIA Registry
Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN)
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.
Peter Milner, MD study director
Chief Medical Officer
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing RT001 in people with Infantile Neuroaxonal Dystrophy, 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 6 inclusion and 5 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: under way, not looking for participants, as of the sponsor's last update on 14 Oct 2021. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Modified Ashworth Spasticity Scale (time frame: 12 months). 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 14 Oct 2021.