Amyotrophic Lateral SclerosisPhase 1NCT02269436
A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS
A Phase 1 study testing sNN0029 infusion solution in people with Amyotrophic Lateral Sclerosis, run by Newron Sweden AB at 2 sites in 2 countries.
Trial summary
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 μg sNN0029/day in patients with ALS who previously participated in study sNN0029-003 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 infusion solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jan 2015to 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
- Previous participation in sNN0029-003 with completion of 12 weeks study without clinically significant safety concerns
- Intact continuity of the Medtronic SynchroMed® II Infusion System as judged by X-ray of head and abdominal area
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria
- 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 informed consent form on the patient's behalf
Who cannot join
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic
- Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.
- Diagnosis of diabetes mellitus
- 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)
- Underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions)
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- Presence of additional risk factors for thromboembolism such as obesity (Body mass index [BMI] > 35) or use of oestrogens including combined contraceptive pills
- Clinically significant abnormalities in haematology 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 patient to undergo study procedures (e.g., MRI), or to give informed consent
- 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 Multi-centre, Open-label, follow-on Study to Assess Long-term Safety and Tolerability of Intracerebroventricular Administration of sNN0029 in Patients With Amyotrophic Lateral Sclerosis
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 μg sNN0029/day in patients with ALS who previously participated in study sNN0029-003
Read the detailed description
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of sNN0029 infusion solution in patients with ALS.
Eighteen patients who previously participated in study sNN0029-003 will receive continuous administration of 4 μg sNN0029/day via i.c.v. infusion with the Medtronic SynchroMed® II Infusion System. The sNN0029 infusion solution is intended to be an add-on treatment to other treatments for ALS.
The assessments performed at the last visit in study sNN0029-003 will serve as the baseline values for patients included in study sNN0029-004 and be the start of sNN0029 treatment for all patients (Study Day 1; Visit 1). In order to not reveal the blind in study sNN0029-003, the patients will return to hospital on Day 3 and stay to Day 6 (Visit 2). During these days, the sNN0029 begins to exit the i.c.v. catheter tip in the ventricle of the brain.
Patients will return to the hospital on Day 11 (Visit 3) for a refill of sNN0029 and adjustment of the infusion pump flow rate. After this, the patient will return to the clinic on Day 39 (Visit 4) for a sNN0029-refill of infusion pump and assessments. Thereafter the patients will visit the hospital on a monthly basis (every 28 days ± 2) to perform refills and make assessments every 3 months.
Treatment in study sNN0029-004 may continue unless safety concerns warrant discontinuation of therapy, until patients choose to withdraw from the study, experience treatment related toxicity or intolerance, are deemed to be unsuitable to continue treatment by the investigator, or die
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- sNN0029 infusion solution
- 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.
sNN0029 infusion solutionDrug
4 µg/day, continuous i.c.v administration
| Study group | Role | What they receive |
|---|---|---|
| sNN0029 infusion solution | Experimental | Not described Receives: Drug: sNN0029 infusion solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number Patients with Adverse Events as a measure of Safety and Tolerability
Number of patients with events will be analysed primarily at 24 months of study duration. The study may be extended on a 6-monthly basis for as long as there are patients that tolerate, and are still eligible to receive, the study treatment.
Measured: At 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2014 (estimated)
- Study startJan 2015
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Newron Sweden AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02269436
- Sponsor's protocol ID
- sNN0029-004
- Other IDs
- 2012-005034-11
- Status last verified
- Jan 2016
- First submitted
- 8 Oct 2014
- 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 Multi-centre, Open-label, follow-on Study to Assess Long-term Safety and Tolerability of Intracerebroventricular Administration of sNN0029 in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT02269436
- Acronym / sponsor ID
- sNN0029-004
- 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
- 11 participants (actual)
- Lead sponsor
- Newron Sweden ABIndustry (pharma, biotech, devices)
- Study start
- Jan 2015
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 21 Oct 2014 (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, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 μg sNN0029/day in patients with ALS who previously participated in study sNN0029-003
Detailed description
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of sNN0029 infusion solution in patients with ALS.
Eighteen patients who previously participated in study sNN0029-003 will receive continuous administration of 4 μg sNN0029/day via i.c.v. infusion with the Medtronic SynchroMed® II Infusion System. The sNN0029 infusion solution is intended to be an add-on treatment to other treatments for ALS.
The assessments performed at the last visit in study sNN0029-003 will serve as the baseline values for patients included in study sNN0029-004 and be the start of sNN0029 treatment for all patients (Study Day 1; Visit 1). In order to not reveal the blind in study sNN0029-003, the patients will return to hospital on Day 3 and stay to Day 6 (Visit 2). During these days, the sNN0029 begins to exit the i.c.v. catheter tip in the ventricle of the brain.
Patients will return to the hospital on Day 11 (Visit 3) for a refill of sNN0029 and adjustment of the infusion pump flow rate. After this, the patient will return to the clinic on Day 39 (Visit 4) for a sNN0029-refill of infusion pump and assessments. Thereafter the patients will visit the hospital on a monthly basis (every 28 days ± 2) to perform refills and make assessments every 3 months.
Treatment in study sNN0029-004 may continue unless safety concerns warrant discontinuation of therapy, until patients choose to withdraw from the study, experience treatment related toxicity or intolerance, are deemed to be unsuitable to continue treatment by the investigator, or die
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| sNN0029 infusion solution | Experimental | Drug: sNN0029 infusion solution |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Previous participation in sNN0029-003 with completion of 12 weeks study without clinically significant safety concerns
- Intact continuity of the Medtronic SynchroMed® II Infusion System as judged by X-ray of head and abdominal area
- Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria
- 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 informed consent form on the patient's behalf
Key exclusion criteria (13 listed)
- Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic
- Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.
- Diagnosis of diabetes mellitus
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that cannot be not managed optimally due to:
- Presence of additional risk factors for thromboembolism such as obesity (Body mass index [BMI] > 35) or use of oestrogens including combined contraceptive pills
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 |
|---|---|---|
| sNN0029 infusion solution4 µg/day, continuous i.c.v administration | Drug | sNN0029 infusion solution |
Primary outcome
Number Patients with Adverse Events as a measure of Safety and Tolerability
Time frame: At 24 Months
Number of patients with events will be analysed primarily at 24 months of study duration. The study may be extended on a 6-monthly basis for as long as there are patients that tolerate, and are still eligible to receive, the study treatment.
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.
The record lists no contact. Use the official record to reach the sponsor.
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 infusion solution 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 5 inclusion and 13 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. 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 Patients with Adverse Events as a measure of Safety and Tolerability (time frame: At 24 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 27 Jan 2016.