Spinal Muscular Atrophy Type IPhase 3NCT04042025
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
A Phase 3 study testing Onasemnogene Abeparvovec-xioi in people with Spinal Muscular Atrophy Type I and Spinal Muscular Atrophy Type II, run by Novartis Gene Therapies at 31 sites in 9 countries.
Trial summary
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
- What is being tested?
- Onasemnogene Abeparvovec-xioi
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 85 participants
- Where
- 31 sitesin 9 countries
- Study dates
- 10 Feb 2020to 31 Dec 2035
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
- Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Who cannot join
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
Trial locations and contacts
31 sites in 9 countries.
31 sites in 9 countries
United States18 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
John Hopkins Hospital - David M. Rubenstein Child Health Building
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Health Specialty Center Dallas Campus
Dallas, Texas, 75235
Duke University
Durham, North Carolina, 27713
Spectrum Health Hospitals Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
University of Wisconsin, Madison
Madison, Wisconsin, 53792
Columbia University Medical Center
New York, New York, 10032
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Stanford University Medical Center
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Utah Health
Salt Lake City, Utah, 84112
Washington Unviersity School of Medicine in Saint Louis
St Louis, Missouri, 63110
Clinic for Special Children
Strasburg, Pennsylvania, 17579
Italy4 sites
Instituto Gianninia Gaslini
Genova, 16147
Universita Degli Studi Di Milano
Milan, 20122
Istituto Neurologico di Ricerca
Milan, 20133
Fondazione Policlinico Universitario Agostino Gemelli
Roma, Roma, 00168
Belgium2 sites
Universitair Ziekenhuis Gent
Ghent, 9000
Centre de Référence des Maladies Neuromusculaires
Liège, Liège, B-4000
United Kingdom2 sites
Great Ormond Street Hospital for Children NHS Foundation Trust
London, London, WC1N 3JH
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 4LP
Canada1 site
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, K1H8L1
About this study
Official title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Being tested
- Onasemnogene Abeparvovec-xioi
- Keywords
- Gene replacement
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Onasemnogene Abeparvovec-xioiBiological
Also called Zolgensma
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
| Study group | Role | What they receive |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi | Other | Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene. Receives: Biological: Onasemnogene Abeparvovec-xioi |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Reach Developmental Milestones
Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
Measured: Up to 5 years
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.
Measured: Up to 15 years
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
Measured: Up to 5 years
Change From Baseline in Height Measurements
Measured: Up to 5 years
Change From Baseline in Weight Measurements
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.
Measured: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support.
Measured: Year 6 to Year 15
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
An SAE is defined as any adverse event (appearance of [or worsening of any pre existing]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:
- Fatal
- Life-threatening
- Results in persistent or significant disability/incapacity
- Constitutes a congenital abnormality or birth defect
- Requires in-patient hospitalization or prolongation of existing hospitalization
- Is medically significant e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
Measured: Up to 15 years
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:
- Hepatotoxicity
- Thrombotic microangiopathy
- Cardiac adverse events
- Dorsal root ganglia toxicity
- New malignancies
- New incidence of a neurologic disorder
- New incidence of an autoimmune disorder
- New incidence of hematologic disorder
Measured: Up to 15 years
Change From Baseline in Bayley Scales of Infant and Toddler Development
Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age.
Measured: Up to 42 months, 15 days of age
Change From Baseline in Revised Upper Limb Module (RULM) Score
RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.
Measured: Up to 5 years
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)).
Measured: Up to 5 years
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced.
Measured: Up to 5 years
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience
Measured: Up to 5 years
Number of Participants With Concomitant Medications Overall and by Type of Medications
Measured: Up to 5 years
Number of Participants With Other SMA Therapies Overall and by Type of Medications
Measured: Year 6 to Year 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2019
- Study start10 Feb 2020
- Primary completion31 Dec 2035 (estimated)
- Study completion31 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular Atrophy Type IStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 85 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Gene Therapies
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sitra Tauscher-Wisniewski, MD, Study chair, Novartis Gene Therapies, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04042025
- Sponsor's protocol ID
- AVXS-101-LT-002
- Other IDs
- 2019-002611-26, 205305, COAV101A12103, 2019
- Status last verified
- Sep 2026
- First submitted
- 31 Jul 2019
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Publications
Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12.
PubMed 34383289
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
- ClinicalTrials.gov ID
- NCT04042025
- Acronym / sponsor ID
- AVXS-101-LT-002
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Keywords
- Gene replacement
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 85 participants (actual)
- Lead sponsor
- Novartis Gene TherapiesIndustry (pharma, biotech, devices)
- Study start
- 10 Feb 2020 (actual)
- Primary completion
- 31 Dec 2035 (estimated)
- Study completion
- 31 Dec 2035 (estimated)
- First posted
- 1 Aug 2019 (actual)
- 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 long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi | Other | Biological: Onasemnogene Abeparvovec-xioiParticipants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
- Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Key exclusion criteria (1 listed)
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
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 |
|---|---|---|
| Onasemnogene Abeparvovec-xioiAlso: ZolgensmaOnasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight. | Biological | Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi |
Primary outcome
Number of Participants Who Reach Developmental Milestones
Time frame: Up to 5 years
Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Time frame: Up to 5 years
The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Time frame: Up to 15 years
Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Time frame: Up to 5 years
Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
Time frame: Up to 5 years
The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Time frame: Up to 5 years
Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
Change From Baseline in Height Measurements
Time frame: Up to 5 years
Change From Baseline in Weight Measurements
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Time frame: Up to 5 years
Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Time frame: Up to 5 years
Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
Time frame: Year 6 to Year 15
The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support.
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
Time frame: Up to 15 years
An SAE is defined as any adverse event (appearance of [or worsening of any pre existing]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * Fatal * Life-threatening * Results in persistent or significant disability/incapacity * Constitutes a congenital abnormality or birth defect * Requires in-patient hospitalization or prolongation of existing hospitalization * Is medically significant e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
Time frame: Up to 15 years
An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria: * Hepatotoxicity * Thrombotic microangiopathy * Cardiac adverse events * Dorsal root ganglia toxicity * New malignancies * New incidence of a neurologic disorder * New incidence of an autoimmune disorder * New incidence of hematologic disorder
Change From Baseline in Bayley Scales of Infant and Toddler Development
Time frame: Up to 42 months, 15 days of age
Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age.
Change From Baseline in Revised Upper Limb Module (RULM) Score
Time frame: Up to 5 years
RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
Time frame: Up to 5 years
The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)).
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
Time frame: Up to 5 years
The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced.
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
Time frame: Up to 5 years
ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience
Number of Participants With Concomitant Medications Overall and by Type of Medications
Time frame: Up to 5 years
Number of Participants With Other SMA Therapies Overall and by Type of Medications
Time frame: Year 6 to Year 15
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- Derived from this study: PubMed 34383289Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
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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.
Sitra Tauscher-Wisniewski, MD study chair
Novartis Gene Therapies, Inc.
31 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Onasemnogene Abeparvovec-xioi in people with Spinal Muscular Atrophy Type I and Spinal Muscular Atrophy Type II, run by Novartis Gene Therapies at 31 sites in 9 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 2 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Italy, Belgium, United Kingdom, Australia and 4 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. 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?
This study is still under way. Results are not available in the source record yet.
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 Participants Who Reach Developmental Milestones (time frame: Up to 5 years). The study lists 20 primary outcomes in all. 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 NCT04042025, which also lists 1 linked publication.
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 24 Sep 2026.