Amyotrophic Lateral SclerosisPhase 1NCT01999803
A Safety Study of sNN0029 Administration Via Intracerebroventricular Route to Patients With ALS
A Phase 1 study testing sNN0029 in people with Amyotrophic Lateral Sclerosis, run by Newron Sweden AB at 2 sites in 2 countries.
Trial summary
This is a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v. administration of sNN0029 infusion solution at a dose of 4µg/day in patients with Amyotrophic Lateral Sclerosis (ALS). 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
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Sep 2014to Oct 2015
- Phase
- Phase 1
- 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 World Federation of Neurology (WFN) El Escorial criteria.
- If patients are being treated with riluzole, they must have been on a stable dose for at least the past 30 days prior to screening.
- The patient is, in the opinion of the investigator, medically fit to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.
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.
- Values for coagulation parameters including platelet count, normalised prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) outside normal ranges.
- Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.
- Diagnosis of diabetes mellitus.
- History of structural brain disease other than ALS, including tumours and hyperplasia.
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- An MRI of the brain and cervical spine, and an Magnetic Resonance Angiography (MRA) of the brain with findings of tumours or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgment of the investigator, represent a surgical risk to the patient. If an MRI and/or MRA has been performed within 1 month prior to screening, the results from that examination can be used.
- 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 i.c.v. delivery or device implants medically unsuitable.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that cannot be not managed optimally due to:
- anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), ii. underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions) iii. administration of any antiplatelet or anticoagulant medication in the preoperative period
- A personal history of thromboembolic disease. A family history of thromboembolic disease will prompt a laboratory assessment to exclude hereditary liability before the patient is declared eligible.
- Presence of additional risk factors for thromboembolism such as obesity (BMI > 35) or use of oestrogens including combined contraceptive pills.
- Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter.
- Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.
- Serological evidence of Hepatitis B virus (HBV), Hepatitis C virus (HCV) or Human immunodeficiency virus (HIV)
- 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 patient 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 women only: pregnant, breast feeding and/or for fecund women unwillingness to use adequate contraception during the trial such as:
- Established use of oral, injected or implanted hormonal methods of contraception that do NOT contain oestrogens.
- Placement of an intrauterine device.
- Barrier methods of contraception: Condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Phase I, Randomised, Double-blind, Placebo-controlled Study in Patients With Amyotrophic Lateral Sclerosis to Further Assess the Safety and Tolerability of Intracerebroventricular Administration of sNN0029 Infusion Solution
This is a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v. administration of sNN0029 infusion solution at a dose of 4µg/day in patients with Amyotrophic Lateral Sclerosis (ALS).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- sNN0029, Placebo
- 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
Also called telbermin, rhVEGF165
PlaceboDrug
Also called Artificial CSF
| Study group | Role | What they receive |
|---|---|---|
| sNN0029 (VEGF) | Experimental | 4 µg/d of sNN0029 administered by continuous intracerebral infusion during12 weeks Receives: Drug: sNN0029 |
| Placebo | Placebo comparator | Placebo administered by continuous intracerebral infusion during12 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Events (AEs)
The safety and tolerability of i.c.v. administration of sNN0029 infusion solution at a dose of 4 µg/day delivered via a Medtronic SynchroMed® II Infusion System will be evaluated by comparing tabulated number of events over 12 weeks by body system, preferred term and by severity and relationship to study medication/device. Serious Adverse Events/Serious Adverse Device Events will also be presented in separate tabulations.
Measured: 12 weeks
Secondary outcomes (2)
VEGF165 levels in Cerebrospinal Fluid (CSF)
Levels of the active ingredient of sNN0029 infusion solution (VEGF165) in CSF will be summarised using descriptive statistics by time point and treatment.
Measured: 12 weeks
Medical Device performance
Device performance (number of values +/-25% of expected) will be presented by time point and treatment.
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 posted3 Dec 2013 (estimated)
- Study startSep 2014
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Newron Sweden AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Philip VanDamme, Prof, MD, Principal investigator, University Hospital Leuven, Herestraat 49, B-3000 Leuven, BelgiumLeonard van den Berg, MD, Prof, Principal investigator, University Medical Center Utrecht, Department of Neurology G03.228, P.O. Box 85500, NL-3508 GA Utrecht, The Netherlands
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01999803
- Sponsor's protocol ID
- sNN0029-003
- Other IDs
- 2012-001026-10
- Status last verified
- Jan 2016
- First submitted
- 22 Nov 2013
- 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
- A Phase I, Randomised, Double-blind, Placebo-controlled Study in Patients With Amyotrophic Lateral Sclerosis to Further Assess the Safety and Tolerability of Intracerebroventricular Administration of sNN0029 Infusion Solution
- ClinicalTrials.gov ID
- NCT01999803
- Acronym / sponsor ID
- sNN0029-003
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- 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)
- Study start
- Sep 2014
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 3 Dec 2013 (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 a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v. administration of sNN0029 infusion solution at a dose of 4µg/day in patients with Amyotrophic Lateral Sclerosis (ALS).
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 (VEGF) | Experimental | Drug: sNN00294 µg/d of sNN0029 administered by continuous intracerebral infusion during12 weeks |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo administered by continuous intracerebral infusion during12 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised World Federation of Neurology (WFN) El Escorial criteria.
- If patients are being treated with riluzole, they must have been on a stable dose for at least the past 30 days prior to screening.
- The patient is, in the opinion of the investigator, medically fit to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.
Key exclusion criteria (21 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.
- Values for coagulation parameters including platelet count, normalised prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) outside normal ranges.
- Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.
- Diagnosis of diabetes mellitus.
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 |
|---|---|---|
| sNN0029Also: telbermin, rhVEGF165 | Drug | sNN0029 (VEGF) |
| PlaceboAlso: Artificial CSF | Drug | Placebo |
Primary outcome
Number of Adverse Events (AEs)
Time frame: 12 weeks
The safety and tolerability of i.c.v. administration of sNN0029 infusion solution at a dose of 4 µg/day delivered via a Medtronic SynchroMed® II Infusion System will be evaluated by comparing tabulated number of events over 12 weeks by body system, preferred term and by severity and relationship to study medication/device. Serious Adverse Events/Serious Adverse Device Events will also be presented in separate tabulations.
2 secondary outcomes
VEGF165 levels in Cerebrospinal Fluid (CSF)
Time frame: 12 weeks
Medical Device performance
Time frame: 12 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.
Philip VanDamme, Prof, MD principal investigator
University Hospital Leuven, Herestraat 49, B-3000 Leuven, Belgium
Leonard van den Berg, MD, Prof principal investigator
University Medical Center Utrecht, Department of Neurology G03.228, P.O. Box 85500, NL-3508 GA Utrecht, The Netherlands
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing sNN0029 in people with Amyotrophic Lateral Sclerosis, run by Newron Sweden AB at 2 sites in 2 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 3 inclusion and 21 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 2 sites in Belgium, Netherlands. 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 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.
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?
Number of Adverse Events (AEs) (time frame: 12 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.