X-Linked AdrenoleukodystrophyPhase 1/2NCT03196765
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
A Phase 1/2 study testing Sobetirome (NV1205) in people with X-Linked Adrenoleukodystrophy, run by NeuroVia, Inc. at 13 sites in 8 countries.
Trial summary
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase. Read more about this study
- Who is it for?
- Ages 4 to 18 years, men only
- Conditions the study is for
- X-Linked Adrenoleukodystrophy
- What is being tested?
- Sobetirome (NV1205)
- Type of treatment
- Pill or oral medicine
- How many people
- 0 participants
- Where
- 13 sitesin 8 countries
- Study dates
- Aug 2018to 1 Dec 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 4 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males ≥4 years and <18 years of age
- CCALD diagnosis confirmed by genetic testing
- Loes score of >0 and ≤15
- Patients on VLCFA lowering agents such as Lorenzo's oil must stop the medication and have high VLCFA levels before enrollment
Who cannot join
- Significant medical conditions such as heart, thyroid, or liver disease
- HSCT recipients
Trial locations and contacts
13 sites in 8 countries.
13 sites in 8 countries
Russia3 sites
Endocrinology Research Center
Moscow
Moscow Morozov's Children Clinical Hospital
Moscow
Saint Petersburg State Pediatric Medical University
Saint Petersburg
Argentina2 sites
Hospital Austral
Buenos Aires
Hospital General de ninos Pedro de Elizalde
Buenos Aires
Chile2 sites
Hospital Clínico San Borja Arriarán
Santiago
Hospital Dr. Luis Calvo Mackenna
Santiago
United Kingdom2 sites
Great Ormond Street Hospital for Children
London
Manchester Children's Hospital
Manchester
About this study
Official title: Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
Read the detailed description
This study is to evaluate the safety, pharmacokinetics, and efficacy of NV1205 in pediatric patients diagnosed with childhood cerebral adrenoleukodystrophy (CCALD).
The study consists of:
- Screening period: within 30 days of first dose
- Main treatment period of 12 weeks (Part 1- from Screening to Week 12)
- Long Term Treatment (LTT) period (Part 2- Week 13 through Week 96)
In Part 1, subjects will have an initial 4-week treatment period at the assigned dose and, if no safety concerns are noted, subjects continue for another 8 weeks of extended safety assessment.
There will be several cohorts of subjects enrolled. After each Cohort has completed the 4-week initial safety assessment, the safety data will be reviewed by an independent Data Safety Monitoring Board (DSMB) and, subject to DSMB recommendation, Cohort 2 will be enrolled and receive the next dose level. After Cohort 2 has completed 4 weeks of treatment, the DSMB will review all available safety data (Cohorts 1 and 2) and, subject to DSMB recommendation, Cohort 3 will be enrolled and receive the next dose level. Additional Cohorts may be enrolled at the recommendation of the DSMB with an incremental dose increase.
In Part 2, subjects will continue to receive treatment in the LTT period of the study.
- Conditions
- X-Linked Adrenoleukodystrophy
- Being tested
- Sobetirome (NV1205)
- Keywords
- X-ALD
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sobetirome (NV1205)Drug
Also called NV1205
Once a day oral dose of the study drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Adverse events
Measured: 2 years
Secondary outcomes (1)
Area under the curve concentration of NV1205 in plasma
A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2017
- Study startAug 2018 (estimated)
- Primary completion1 Dec 2020 (estimated)
- Study completion1 Dec 2020 (estimated)
Why it stopped: No funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked AdrenoleukodystrophyStudies 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
- Intervention model
- Sequential
- Model description
- Sequential dose escalation
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NeuroVia, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Henderson, MD, Study director, NeuroVia, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03196765
- Sponsor's protocol ID
- NV1205-009
- Status last verified
- May 2018
- First submitted
- 5 May 2017
- Last updated
- 7 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)
- ClinicalTrials.gov ID
- NCT03196765
- Acronym / sponsor ID
- NV1205-009
- Conditions
- X-Linked Adrenoleukodystrophy
- Keywords
- X-ALD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NeuroVia, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2018 (estimated)
- Primary completion
- 1 Dec 2020 (estimated)
- Study completion
- 1 Dec 2020 (estimated)
- First posted
- 23 Jun 2017 (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.
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
Detailed description
This study is to evaluate the safety, pharmacokinetics, and efficacy of NV1205 in pediatric patients diagnosed with childhood cerebral adrenoleukodystrophy (CCALD).
The study consists of:
- Screening period: within 30 days of first dose
- Main treatment period of 12 weeks (Part 1- from Screening to Week 12)
- Long Term Treatment (LTT) period (Part 2- Week 13 through Week 96)
In Part 1, subjects will have an initial 4-week treatment period at the assigned dose and, if no safety concerns are noted, subjects continue for another 8 weeks of extended safety assessment.
There will be several cohorts of subjects enrolled. After each Cohort has completed the 4-week initial safety assessment, the safety data will be reviewed by an independent Data Safety Monitoring Board (DSMB) and, subject to DSMB recommendation, Cohort 2 will be enrolled and receive the next dose level. After Cohort 2 has completed 4 weeks of treatment, the DSMB will review all available safety data (Cohorts 1 and 2) and, subject to DSMB recommendation, Cohort 3 will be enrolled and receive the next dose level. Additional Cohorts may be enrolled at the recommendation of the DSMB with an incremental dose increase.
In Part 2, subjects will continue to receive treatment in the LTT period of the study.
Study design
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Model description
- Sequential dose escalation
Who may qualify
- Ages
- 4 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Males ≥4 years and <18 years of age
- CCALD diagnosis confirmed by genetic testing
- Loes score of >0 and ≤15
- Patients on VLCFA lowering agents such as Lorenzo's oil must stop the medication and have high VLCFA levels before enrollment
Key exclusion criteria (2 listed)
- Significant medical conditions such as heart, thyroid, or liver disease
- HSCT recipients
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 |
|---|---|---|
| Sobetirome (NV1205)Also: NV1205Once a day oral dose of the study drug | Drug | – |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 2 years
Adverse events
1 secondary outcome
Area under the curve concentration of NV1205 in plasma
Time frame: 12 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2019.
- Individual participant dataSharing planned: No
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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.
John Henderson, MD study director
NeuroVia, Inc.
13 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sobetirome (NV1205) in people with X-Linked Adrenoleukodystrophy, run by NeuroVia, Inc. at 13 sites in 8 countries.
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 4 inclusion and 2 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: withdrawn, as of the sponsor's last update on 7 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Russia, Argentina, Chile, United Kingdom, Australia and 3 more countries. 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?
The study was withdrawn before enrolling participants, so there are no results.
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 participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: 2 years). 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 7 Mar 2019.