Spinal Muscular AtrophyPhase 3CHERISHNCT02292537
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
A Phase 3 study testing Nusinersen in people with Spinal Muscular Atrophy, run by Biogen at 24 sites in 10 countries.
Trial summary
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). Read more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Nusinersen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 126 participants
- Where
- 24 sitesin 10 countries
- Study dates
- 24 Nov 2014to 20 Feb 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent or guardian has signed informed consent and, if indicated per participant's age and institutional guidelines, participant has signed informed assent
- Be medically diagnosed with Spinal Muscular Atrophy (SMA)
- Have onset of clinical signs and symptoms consistent with SMA at greater than 6 months of age
- Be able to sit independently, but has never had the ability to walk independently
- Have Motor Function Score (Hammersmith Functional Motor Scale - Expanded) greater than or equal to 10 and less than or equal to 54 at Screening
- Be able to complete all study procedures, measurements and visits and parent or guardian and subject has adequately supportive psychosocial circumstances, in the opinion of the Investigator
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- Have an estimated life expectancy of greater than 2 years from Screening, in the opinion of the Investigator
- Meet age-appropriate institutional criteria for use of anesthesia and sedation, if use is planned for study procedures
- For subjects who have reached reproductive maturity, satisfy study contraceptive requirements
Who cannot join
- Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for greater than 6 hours during a 24 hour period, at Screening
- Medical necessity for a gastric feeding tube, where the majority of feeds are given by this route, as assessed by the Site Investigator
- Severe contractures or severe scoliosis evident on X-ray examination at Screening
- Hospitalization for surgery (i.e., scoliosis surgery, other surgery), pulmonary event, or nutritional support within 2 months of Screening or planned during the duration of the study
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period
- History of brain or spinal cord disease, including tumors, or abnormalities by magnetic resonance imaging (MRI) or computed tomography (CT) that would interfere with the LP procedures or cerebrospinal fluid (CSF) circulation
Show all 14Show fewer
- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
- History of bacterial meningitis
- Dosing with IONIS-SMN Rx in any previous clinical study
- Prior injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject's ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline
- Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the subject unsuitable for inclusion
- Treatment with another investigational drug (e.g., oral albuterol or salbutamol, riluzole, carnitine, creatine, sodium phenylbutyrate, et.c), biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Treatment with valproate or hydroxyurea within 3-months of Screening. Any history of gene therapy, antisense oligonucleotide therapy, or cell transplantation.
- Ongoing medical condition that according to the Site Investigator would interfere with the conduct and assessments of the study. Examples are medical disability (e.g., wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromise the ability of the subject to undergo study procedures.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
24 sites in 10 countries.
24 sites in 10 countries
United States11 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Children's Medical Center
Dallas, Texas, 75235
UCLA Clinical and Translational Research Center
Los Angeles, California, 90095
Columbia University Medical Center
New York, New York, 10032
Nemours Children's Hospital
Orlando, Florida, 32827
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304
Children's Hospital of Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104
Oregon Health & Science University
Portland, Oregon, 97239
Washington University School of Medicine
St Louis, Missouri, 63110
Canada2 sites
Children's Hospital - London Health Sciences Centre
London, Ontario, N6A 5W9
McGill University Health Centre-Glen Site-CIM
Montreal, Quebec, H4A 3JI
Germany2 sites
Universitatsklinikum Essen
Essen
University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease
Freiburg im Breisgau
Italy2 sites
AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud
Messina
Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile
Rome
Japan2 sites
Hyogo College of Medicine
Nishinomya-shi, Hyōgo
Tokyo Women's Medical University
Shinjuku-ku, Tokyo
France1 site
Armand Trousseau Hospital, I-Motion, Clinical Trials Platform
Paris
Hong Kong1 site
The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine
Hong Kong, Hong Kong SAR
South Korea1 site
Seoul National University Children's Hospital
Seoul, Korea
Sweden1 site
University of Gothenburg, The Queen Silvia Children's Hospital
Gothenburg
About this study
Official title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
Read the detailed description
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.
In August 2016, sponsorship of the trial was transferred to Biogen.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Nusinersen, Sham procedure
- Keywords
- Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, CHERISH, IONIS-SMNRx, IONIS-SMN Rx
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NusinersenDrug
Also called Sprinraza, ISIS 396443, IONIS-SMN Rx, BIIB058
Administered by intrathecal (IT) lumbar puncture (LP) injection
Sham procedureProcedure or surgery
Small needle prick on the lower back at the location where the IT injection is normally made
| Study group | Role | What they receive |
|---|---|---|
| Nusinersen | Experimental | Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. Receives: Drug: Nusinersen |
| Sham procedure | Sham comparator | Sham comparator on Days 1, 29, 85 and 274. Receives: Procedure: Sham procedure |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Measured: Baseline and Month 15
Secondary outcomes (14)
Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Measured: Baseline and Month 15
Proportion of Participants That Achieved Any New Motor Milestone at Month 15
New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone.
Measured: Month 15
Number of New Motor Milestones Achieved Per Participant
New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone.
Measured: Month 15
Change From Baseline in Revised Upper Limb Module (RULM) Test
The RULM Test is used in patients with SMA to assess upper limb functional ability items that are reflective of activities of daily living (i.e., raise a can to mouth as if drinking, take a coin and place it in a box, remove the lid of a container). The RULM test has a total of 20 items with an entry item that serves as functional class identification and does not contribute to the total score. The remaining 19 scorable items reflect different functional domains and are graded on a 3-point system with a score of 0 (unable), 1 (able, with modification), and a maximum of 2 (able, no difficulty). There is only 1 item (item I) that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for a total score range of 0-37, with higher scores increased great upper limb function. A positive change from Baseline indicates improvement.
Measured: Baseline and Month 15
Proportion of Participants That Achieved Standing Alone
If the participant was unable to achieve standing alone at Baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder.
Measured: Month 15
Proportion of Participants That Achieved Walking With Assistance
If the participant was unable to achieve walking with assistance at baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder.
Measured: Month 15
Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs)
AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death; an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.
Measured: Baseline through Month 15
Number of Participants With Clinically Significant Vital Sign Abnormalities
Vital signs assessed for clinical significance include resting blood pressure, pulse, respiratory rate, and temperature.
Measured: Baseline through Month 15
Number of Participants With Clinically Significant Weight Abnormalities
Weight changes assessed from Baseline to Month 15.
Measured: Baseline through Month 15
Number of Participants With Clinically Significant Neurological Examination Abnormalities
Neurological changes assessed for clinical significance include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes.
Measured: Baseline through Month 15
Number of Participants With Clinically Significant Physical Examination Abnormalities
Physical examination changes were assessed for clinical significance.
Measured: Baseline through Month 15
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Laboratory parameter changes assessed for clinical significance include serum chemistry, hematology, coagulation and urinalysis.
Measured: Baseline through Month 15
Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results
The number of participants with abnormal, clinically relevant worsening, defined as participants with an ECG interpreted as abnormal and clinically relevant, with a comparison with Baseline value is reported.
Measured: Baseline through Month 15
Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure
Concomitant medications include prescription and over-the-counter medications administered to participants on or after the first day of study treatment.
Measured: Baseline through Month 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2014 (estimated)
- Study start24 Nov 2014
- Primary completion20 Feb 2017
- Study completion20 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 126 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02292537
- Sponsor's protocol ID
- ISIS 396443-CS4
- Other IDs
- 2014-001947-18, 2014
- Acronym
- CHERISH
- Status last verified
- Feb 2021
- First submitted
- 12 Nov 2014
- Last updated
- 17 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Expanded access
- Available
Publications
Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w.
PubMed 31420846Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
PubMed 29443664
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT02292537
- Acronym / sponsor ID
- CHERISH / ISIS 396443-CS4
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, CHERISH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 126 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 24 Nov 2014 (actual)
- Primary completion
- 20 Feb 2017 (actual)
- Study completion
- 20 Feb 2017 (actual)
- First posted
- 17 Nov 2014 (estimated)
- Results
- Posted 22 Feb 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
Detailed description
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.
In August 2016, sponsorship of the trial was transferred to Biogen.
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 |
|---|---|---|
| Nusinersen | Experimental | Drug: NusinersenNusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. |
| Sham procedure | Sham comparator | Procedure: Sham procedureSham comparator on Days 1, 29, 85 and 274. |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Parent or guardian has signed informed consent and, if indicated per participant's age and institutional guidelines, participant has signed informed assent
- Be medically diagnosed with Spinal Muscular Atrophy (SMA)
- Have onset of clinical signs and symptoms consistent with SMA at greater than 6 months of age
- Be able to sit independently, but has never had the ability to walk independently
- Have Motor Function Score (Hammersmith Functional Motor Scale - Expanded) greater than or equal to 10 and less than or equal to 54 at Screening
Key exclusion criteria (14 listed)
- Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for greater than 6 hours during a 24 hour period, at Screening
- Medical necessity for a gastric feeding tube, where the majority of feeds are given by this route, as assessed by the Site Investigator
- Severe contractures or severe scoliosis evident on X-ray examination at Screening
- Hospitalization for surgery (i.e., scoliosis surgery, other surgery), pulmonary event, or nutritional support within 2 months of Screening or planned during the duration of the study
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period
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 |
|---|---|---|
| NusinersenAlso: Sprinraza, ISIS 396443, IONIS-SMN Rx, BIIB058Administered by intrathecal (IT) lumbar puncture (LP) injection | Drug | Nusinersen |
| Sham procedureSmall needle prick on the lower back at the location where the IT injection is normally made | Procedure or surgery | Sham procedure |
Primary outcome
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15
Time frame: Baseline and Month 15
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
14 secondary outcomes
Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15
Time frame: Baseline and Month 15
Proportion of Participants That Achieved Any New Motor Milestone at Month 15
Time frame: Month 15
Number of New Motor Milestones Achieved Per Participant
Time frame: Month 15
Change From Baseline in Revised Upper Limb Module (RULM) Test
Time frame: Baseline and Month 15
Proportion of Participants That Achieved Standing Alone
Time frame: Month 15
Proportion of Participants That Achieved Walking With Assistance
Time frame: Month 15
Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline through Month 15
Number of Participants With Clinically Significant Vital Sign Abnormalities
Time frame: Baseline through Month 15
Number of Participants With Clinically Significant Weight Abnormalities
Time frame: Baseline through Month 15
Number of Participants With Clinically Significant Neurological Examination Abnormalities
Time frame: Baseline through Month 15
Number of Participants With Clinically Significant Physical Examination Abnormalities
Time frame: Baseline through Month 15
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Time frame: Baseline through Month 15
Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results
Time frame: Baseline through Month 15
Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure
Time frame: Baseline through Month 15
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31420846Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w.
- Derived from this study: PubMed 29443664Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
- Cure SMALink provided by the sponsor.
- Muscular Dystrophy AssociationLink provided by the sponsor.
- National Organization for Rare DiseasesLink provided by the sponsor.
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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.
Medical Director study director
Biogen
24 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nusinersen in people with Spinal Muscular Atrophy, run by Biogen at 24 sites in 10 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 9 inclusion and 14 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 17 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Canada, Germany, Italy, Japan and 5 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Feb 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15 (time frame: Baseline and Month 15). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02292537, which also lists 2 linked publications.
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 17 Feb 2021.