Amyotrophic Lateral SclerosisPhase 1/2NCT01384162
An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
A Phase 1/2 study testing sNN0029 in people with Amyotrophic Lateral Sclerosis, run by Newron Sweden AB at 1 site in Belgium.
Trial summary
This is an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- sNN0029
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteBelgium
- Study dates
- Jun 2009to Oct 2015
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria (Appendix B).
- Previous participation in study sNN0029-001 with completion of 12 weeks study drug administration.
- To have completed the investigations associated with safety in study sNN0029-001 without development of clinically significant safety concerns.
- Patient has been given written and verbal information about the continuation study, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.
- Patient has given oral and / or signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.
Who cannot join
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.
- Proliferative retinopathy.
- Non-proliferative retinopathy of moderate severity or higher.
- Concurrent clinically significant dementia as determined by the investigator.
- Concurrent clinically significant depression as determined by the investigator.
- Need for administration of any antiplatelet or anticoagulant medication (e.g. aspirin, Plavix, non-steroidal anti-inflammatory drugs [NSAIDs]). Low dose aspirin or occasional use of NSAIDs is allowed (See Appendix E).
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- Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.
- For female patients, ongoing pregnancy or planned pregnancy
- Breast feeding
Trial locations and contacts
1 site in 1 country.
Belgium1 site
University Hospital Leuven, Department of Neurology
Leuven, 3000
About this study
Official title: An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump
This is an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. The intention of the study is to investigate safety and tolerability of intracerebroventricular administration of sNN0029 and whether it can improve motor function and prolong survival in patients with ALS.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- sNN0029
- Keywords
- ALS, VEGF165, Safety, Intracerebroventricular, Infusion, Implantable
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sNN0029Drug
ICV infusion
| Study group | Role | What they receive |
|---|---|---|
| sNN0029, ICV infusion | Experimental | Not described Receives: Drug: sNN0029 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability through assessments of adverse events, vital signs, clinical laboratory tests, MRI of brain, fundus photography and device performance as characterized by catheter tip placement and infusion accuracy
Measured: multiple assessments over 120 weeks
Secondary outcomes (1)
Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma
Measured: Multiple assessments over 120 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2011 (estimated)
- Study startJun 2009
- Primary completionOct 2015
- Study completionOct 2015
Why it stopped: Issues with development and supply of infusion system for delivery of IMP. Lack of favorable benefit risk ratio in sNN0029-003 study (review of interim data).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Newron Sweden AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medtronic, ICON Clinical Research
- Responsible party
- Sponsor
- Investigators
- Wim Robberecht, MD, PhD, Principal investigator, University Hospital Leuven, Department of Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01384162
- Sponsor's protocol ID
- sNN0029-002
- Status last verified
- Jan 2016
- First submitted
- 23 Jun 2011
- Last updated
- 27 Jan 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump
- ClinicalTrials.gov ID
- NCT01384162
- Acronym / sponsor ID
- sNN0029-002
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, VEGF165, Safety, Intracerebroventricular, Infusion, Implantable
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Issues with development and supply of infusion system for delivery of IMP. Lack of favorable benefit risk ratio in sNN0029-003 study (review of interim data).
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Newron Sweden ABIndustry (pharma, biotech, devices)
- Collaborators
- Medtronic, ICON Clinical Research
- Study start
- Jun 2009
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 28 Jun 2011 (estimated)
- 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 an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. The intention of the study is to investigate safety and tolerability of intracerebroventricular administration of sNN0029 and whether it can improve motor function and prolong survival in patients with ALS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| sNN0029, ICV infusion | Experimental | Drug: sNN0029 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria (Appendix B).
- Previous participation in study sNN0029-001 with completion of 12 weeks study drug administration.
- To have completed the investigations associated with safety in study sNN0029-001 without development of clinically significant safety concerns.
- Patient has been given written and verbal information about the continuation study, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.
- Patient has given oral and / or signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.
Key exclusion criteria (9 listed)
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.
- Proliferative retinopathy.
- Non-proliferative retinopathy of moderate severity or higher.
- Concurrent clinically significant dementia as determined by the investigator.
- Concurrent clinically significant depression as determined by the investigator.
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 |
|---|---|---|
| sNN0029ICV infusion | Drug | sNN0029, ICV infusion |
Primary outcome
Safety and tolerability through assessments of adverse events, vital signs, clinical laboratory tests, MRI of brain, fundus photography and device performance as characterized by catheter tip placement and infusion accuracy
Time frame: multiple assessments over 120 weeks
1 secondary outcome
Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma
Time frame: Multiple assessments over 120 weeks
Results and safety
The study was stopped early (reason given: Issues with development and supply of infusion system for delivery of IMP. Lack of favorable benefit risk ratio in sNN0029-003 study (review of interim data).). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2016.
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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.
Wim Robberecht, MD, PhD principal investigator
University Hospital Leuven, Department of Neurology
1 site 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 sNN0029 in people with Amyotrophic Lateral Sclerosis, run by Newron Sweden AB at 1 site in Belgium.
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 5 inclusion and 9 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: stopped early, as of the sponsor's last update on 27 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. 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 stopped early (reason given: Issues with development and supply of infusion system for delivery of IMP. Lack of favorable benefit risk ratio in sNN0029-003 study (review of interim data).). Results are not available in the source record.
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?
Safety and tolerability through assessments of adverse events, vital signs, clinical laboratory tests, MRI of brain, fundus photography and device performance as characterized by catheter tip placement and infusion accuracy (time frame: multiple assessments over 120 weeks). 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 Jan 2016.