Amyotrophic Lateral SclerosisPhase 1/2NCT00800501
A Safety and Tolerability 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 study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed® II pump. 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?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 1 siteBelgium
- Study dates
- Dec 2008to Jun 2011
- 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.
- Age 18 to 75 years inclusive.
- If patients are being treated with riluzole, they must have been on a stable dose for at least 30 days.
- Ophthalmological examination at screening with normal findings regarding vascular structure and function.
- MRI/magnetic resonance angiography (MRA) examination of the brain and cervical spinal cord at screening with no findings of tumors or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgement of the investigator, represent a surgical risk to the subject.
- Values of coagulation parameters including platelet count, normalized prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) within normal ranges.
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- Patient is medically able to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.
- Patient has been given written and verbal information, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.
- Patient has given 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
- Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.
- Diagnosis of diabetes mellitus.
- Proliferative retinopathy.
- Non-proliferative retinopathy of moderate severity or higher.
- Concurrent clinically significant dementia as determined by the investigator.
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- Concurrent clinically significant depression as determined by the investigator.
- History of structural brain disease other than ALS, including tumours and hyperplasia.
- Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection), alters wound healing (e.g., including bleeding disorders), or renders chronic ICV delivery or device implants medically unsuitable.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally. Physicians should specifically investigate anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions), and the administration of any antiplatelet or anticoagulant medication (e.g., aspirin, Plavix, NSAIDs) in the pre- or perioperative period. Any of those conditions or drugs could place a patient at an increased risk for intraoperative or postoperative bleeding.
- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.
- Presence of cardiac pacemakers, spinal cord stimulators, implantable programmable intraspinal drug pumps, or any other device that may interfere or interact with the programmer, without prior approval by Medtronic.
- Clinically significant abnormalities in hematology or clinical chemistry parameters as assessed by the investigator.
- Ongoing medical condition that according to the investigator would interfere with the conduct and assessments in the study. Examples are medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of safety and efficacy of investigational product or device performance, or would compromise the ability of the subject to undergo study procedures (e.g., MRI), or to give informed consent.
- Participation in another clinical trial with an investigational drug or device within 3 months prior to Screening visit.
- For female subjects, ongoing pregnancy or planned pregnancy during the period of treatment with study drug.
- Breast feeding during the period of treatment with study drug.
Trial locations and contacts
1 site in 1 country.
Belgium1 site
University Hospital Leuven, Department of Neurology
Leuven, 3000
About this study
Official title: A Double-blind, Randomised, Parallel Group Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump
This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed® II pump. Patients with Amyotrophic Lateral Sclerosis will be enrolled.
Read the detailed description
Vascular endothelial growth factor (VEGF) is an endogenous human protein fundamental to the development of the vascular and nervous systems in the body. A role for VEGF in ALS has been suggested from observations in animal models of the disease as well as observations of a dysregulation of VEGF production in patients with ALS.
NeuroNova intends to investigate whether intracerebroventricular administration of VEGF165 in the form of the drug product sNN029 can improve motor function and prolong survival in patients with ALS, and in this first study the safety and tolerability of treatment for 3 months will be evaluated.
Assessments will include:
- Electrocardiograms, vital signs and clinical laboratory tests
- Adverse events and withdrawals related to adverse events
- Possible pathological changes in the brain, spinal cord or retina identified through magnetic resonance imaging and funduscopy
- Possible signs of intracranial bleeding or loss of blood-brain-barrier integrity through measurements of bilirubin and albumin levels in cerebrospinal fluid collected through lumbar and cervical puncture
- Device performance as characterized by catheter tip placement (determined by imaging) and infusion accuracy (pump residual volume)
The secondary objective of this study is:
- To explore the effect of ICV administration of sNN0029 on the time course of Amyotrophic Lateral Sclerosis related parameters including:
- Disease activity as measured by Amyotrophic Lateral Sclerosis Functional Rating Scale
- Quality of life as measured by the EQ-5D rating scale
- To explore the levels of VEGF165 in cerebrospinal fluid collected through lumbar and cervical puncture.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- sNN0029, Placebo
- 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
Continuous ICV infusion at one of three dose levels
PlaceboDrug
Continuous ICV infusion
| Study group | Role | What they receive |
|---|---|---|
| sNN0029 | Experimental | Not described Receives: Drug: sNN0029 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability through assessment of adverse events, ECGs, vital signs, clinical laboratory variables, MRI of brain and spinal cord, CSF sampling, and device performance as characterized by catheter tip placement and infusion accuracy.
Measured: Multiple assessments over 3 months
Secondary outcomes (1)
Time course of Amyotrophic Lateral Sclerosis Functional Rating Scale, Quality of life as measured by the EQ-5D rating scale
Measured: Multiple assessments over 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2008 (estimated)
- Study startDec 2008
- Primary completionJun 2011
- Study completionJun 2011
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 18 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
- NCT00800501
- Sponsor's protocol ID
- sNN0029-001
- Status last verified
- Dec 2011
- First submitted
- 29 Nov 2008
- Last updated
- 6 Dec 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomised, Parallel Group Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump
- ClinicalTrials.gov ID
- NCT00800501
- Acronym / sponsor ID
- sNN0029-001
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, VEGF165, Safety, Intracerebroventricular, Infusion, Implantable
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Newron Sweden ABIndustry (pharma, biotech, devices)
- Collaborators
- Medtronic, ICON Clinical Research
- Study start
- Dec 2008
- Primary completion
- Jun 2011 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 2 Dec 2008 (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 study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed® II pump. Patients with Amyotrophic Lateral Sclerosis will be enrolled.
Detailed description
Vascular endothelial growth factor (VEGF) is an endogenous human protein fundamental to the development of the vascular and nervous systems in the body. A role for VEGF in ALS has been suggested from observations in animal models of the disease as well as observations of a dysregulation of VEGF production in patients with ALS.
NeuroNova intends to investigate whether intracerebroventricular administration of VEGF165 in the form of the drug product sNN029 can improve motor function and prolong survival in patients with ALS, and in this first study the safety and tolerability of treatment for 3 months will be evaluated.
Assessments will include:
- Electrocardiograms, vital signs and clinical laboratory tests
- Adverse events and withdrawals related to adverse events
- Possible pathological changes in the brain, spinal cord or retina identified through magnetic resonance imaging and funduscopy
- Possible signs of intracranial bleeding or loss of blood-brain-barrier integrity through measurements of bilirubin and albumin levels in cerebrospinal fluid collected through lumbar and cervical puncture
- Device performance as characterized by catheter tip placement (determined by imaging) and infusion accuracy (pump residual volume)
The secondary objective of this study is:
- To explore the effect of ICV administration of sNN0029 on the time course of Amyotrophic Lateral Sclerosis related parameters including:
- Disease activity as measured by Amyotrophic Lateral Sclerosis Functional Rating Scale
- Quality of life as measured by the EQ-5D rating scale
- To explore the levels of VEGF165 in cerebrospinal fluid collected through lumbar and cervical puncture.
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 |
|---|---|---|
| sNN0029 | Experimental | Drug: sNN0029 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria.
- Age 18 to 75 years inclusive.
- If patients are being treated with riluzole, they must have been on a stable dose for at least 30 days.
- Ophthalmological examination at screening with normal findings regarding vascular structure and function.
- MRI/magnetic resonance angiography (MRA) examination of the brain and cervical spinal cord at screening with no findings of tumors or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgement of the investigator, represent a surgical risk to the subject.
Key exclusion criteria (17 listed)
- Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.
- Diagnosis of diabetes mellitus.
- Proliferative retinopathy.
- Non-proliferative retinopathy of moderate severity or higher.
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 |
|---|---|---|
| sNN0029Continuous ICV infusion at one of three dose levels | Drug | sNN0029 |
| PlaceboContinuous ICV infusion | Drug | Placebo |
Primary outcome
Safety and tolerability through assessment of adverse events, ECGs, vital signs, clinical laboratory variables, MRI of brain and spinal cord, CSF sampling, and device performance as characterized by catheter tip placement and infusion accuracy.
Time frame: Multiple assessments over 3 months
1 secondary outcome
Time course of Amyotrophic Lateral Sclerosis Functional Rating Scale, Quality of life as measured by the EQ-5D rating scale
Time frame: Multiple assessments over 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2011.
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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 9 inclusion and 17 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 6 Dec 2011. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 assessment of adverse events, ECGs, vital signs, clinical laboratory variables, MRI of brain and spinal cord, CSF sampling, and device performance as characterized by catheter tip placement and infusion accuracy. (time frame: Multiple assessments over 3 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 6 Dec 2011.