A Phase 1/2 interventional study of SOBI003 in Sanfilippo Syndrome Type A (MPS IIIA), sponsored by Swedish Orphan Biovitrum. Completed at 4 sites in 3 countries. Open to participants aged 12 Months to 72 Months. Per ClinicalTrials.gov, last updated 2021-11-19.
Sponsored by Swedish Orphan Biovitrum · Phase 1/2, Interventional, and Treatment
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Among 43 studies in Mucopolysaccharidosis III with a site in United States — 12 open to participants now, 15 under way, 4 of them phase 3. Mucopolysaccharidosis III · United States.
Among the 43 studies in Mucopolysaccharidosis III with a site in United States, this one is the 19th largest by enrollment of the 23 interventional ones that give a count, is one of 4 that started in 2018, is one of 10 in phase 1/2, and was the 22nd registered.
58 studies on the registry are indexed under Mucopolysaccharidosis III; 16 are open to participants now.
Browse Mucopolysaccharidosis III studies →12 of the 43 studies in Mucopolysaccharidosis III with a site in United States are open now. Recruiting first, the newest first:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Sites in United States
Ages 4+
Ozlem Goker-Alpan · Sites in United States
Ages 18–99
Phoenix Nest · Sites in United States, France
Ages 12 mo+
Denali Therapeutics Inc. · Sites in United States
Ages 0–18
Lysosomal and Rare Disorders Research and Treatment Center, Inc. · Sites in United States
Ages 5–99
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 8 Mucopolysaccharidosis III studies recruiting now, 5 set no upper age limit and 1 stops at 75 or younger; 7 take children under 18; none take healthy volunteers.
This study: 12 Months to 72 Months · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
43 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 11 · Phase 2 18 · Phase 3 6 · Phase 4 1
First in 2006; this study’s year is marked. 4 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 6 is below the median of 12 across 33 interventional studies indexed under Mucopolysaccharidosis III.
Median enrollment 17 across the 23 interventional studies, 30 across the 19 observational. Of the 25 that completed or were terminated, 10 (40%) have results posted.
This tab as a report to print or save as PDF → With an account.
30 of 43 have an industry lead sponsor. The 10 most frequent:
Swedish Orphan Biovitrum is the lead sponsor of 82 studies on the registry; 6 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.
Swedish Orphan Biovitrum has 2 studies filed under Mucopolysaccharidosis III — fewer than ten, so there is no comparison to draw.
5 of the studies in Mucopolysaccharidosis III with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06333041Phase 3Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterStudy of Cannabidiol in Sanfilippo Syndrome | 2027-04 |
| NCT04360265Phase 3Ultragenyx Pharmaceutical IncFollow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | 2027-08 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT06567769GC Biopharma CorpPhase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | 2027-06 |
| NCT05492799Allievex CorporationSafety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE | 2027-10 |
Add all 5 to your calendar (.ics) → With an account.
Among the 43 studies in Mucopolysaccharidosis III with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS IIIA is characterized by devastating neurodegeneration with initially mild somatic involvement. The aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients with MPS IIIA, as confirmed by both:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
SOBI003 dose 3 mg/kg once weekly for 24 weeks
Drug: SOBI003
SOBI003 dose 10 mg/kg once weekly for 24 weeks
Drug: SOBI003
Weekly i.v.infusion
Also known as: Modified recombinant human sulphamidase
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Number of adverse events, by type and severity, from start of infusion up to 24 weeks
Time frame: From start of first infusion up to Week 24
The Observed Serum Concentration Immediately Before the Start of Infusion of SOBI003
The observed serum concentration immediately before the start of infusion of SOBI003 (CPre-dose).
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Observed Serum Concentration at the End of Infusion of SOBI003
The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf)
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Time of the End of the Infusion of SOBI003
The time of the end of infusion of SOBI003 (tEnd of inf)
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Maximum Observed Serum Concentration of SOBI003
The Maximum Observed Serum Concentration of SOBI003 (Cmax)
Time frame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Weeks 1, 4, 12, and 24
The Time at Which the Maximum Serum Concentration of SOBI003 is Observed
The time after start of infusion at which the maximum serum concentration is observed (tmax)
Time frame: Weeks 1, 4, 12, and 24
The Minimum Observed Serum Concentration of SOBI003
The minimum observed serum concentration of SOBI003 (CTrough)
Time frame: Weeks 1, 4, 12, and 24
Clearance
Clearance (CL) of SOBI003
Time frame: Weeks 1, 4, 12, and 24
Area Under the Serum Concentration-time Curve From Time 0 to 168 Hours
Area under the serum concentration-time curve from time 0 to 168 hours (AUC 0-168h)
Time frame: 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 1, 4,12, and 24
The Half-life
The half-life of SOBI003 in serum (T1/2)
Time frame: Weeks 1, 4, 12, and 24
SOBI003 Concentration in Cerebrospinal Fluid
SOBI003 concentration in cerebrospinal fluid
Time frame: Weeks 12 and 24
Number of Patients Having Anti-drug Antibodies in Serum
Number of patients in each dose group having anti-drug antibodies in serum
Time frame: Weeks 2,4,8,12 and 24
Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Percent of patients having anti-drug antibodies in cerebrospinal fluid
Time frame: Weeks 12 and 24
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
Time frame: Baseline, weeks 12, and 24
Change From Baseline in Heparan Sulfate Levels in Serum
Change from baseline in Heparan sulfate levels in serum
Time frame: Weeks 2, 3, 4, 8, 12 and 24
Change From Baseline in Heparan Sulfate Levels in Urine
Change from baseline in Heparan sulfate levels in urine
Time frame: Weeks 2, 3, 4, 8, 12 and 24
Change From Baseline in Neurocognitive Development Quotient
Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
Time frame: Week 24
Change From Baseline in Age-equivalence Score
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
Time frame: Week 24
Change From Baseline in Age-equivalence Score as Assessed by VABS-II
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Time frame: Week 24
Change From Baseline in Gray Matter Volume
Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI).
Time frame: Week 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144.
Time frame: Week 24
Change From Baseline in PedsQL™ Family Impact Module Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The measure includes a scale, from where the categorical score "4", "3", "2", "1", and "0" was reversed and linearly transformed to a 0-100 scale to 4=0, 3=25, 2=50, 1=75 and 0=100, where 100 = minimum and 0 = maximum. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning.
Time frame: Week 24
| Milestone | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Started | 3 | 3 |
| Completed | 3 | 3 |
| Not completed | 0 | 0 |
Number of adverse events, by type and severity, from start of infusion up to 24 weeks
| number of events | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Any adverse event | 53 | 128 |
| Any non-treatment emergent serious adverse event | 0 | 1 |
| Any treatment emergent adverse event (TEAE) | 53 | 124 |
| Any drug-related TEAE | 27 | 74 |
| Any non-serious TEAE | 52 | 121 |
| Any serious TEAE | 1 | 3 |
| Any serious drug-related TEAE | 0 | 0 |
| Any TEAE leading to study and/or treatment withdrawal | 0 | 0 |
| Any drug-related TEAE leading to study and/or treatment withdrawal | 0 | 0 |
| Any serious TEAE leading to study and/or treatment withdrawal | 0 | 0 |
| Any TEAE leading to death | 0 | 0 |
| Any Infusion related Reaction | 17 | 27 |
The observed serum concentration immediately before the start of infusion of SOBI003 (CPre-dose).
| ng/mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | NA (NA to NA) | NA (NA to NA) |
| Week 2 | 58.4 (33.0 to 62.2) | 109.0 (85.7 to 137.0) |
| Week 3 | 68.85 (55.2 to 82.5) | 135.5 (114.0 to 157.0) |
| Week 4 | 71.4 (NA to 97.7) | 49.8 (31.4 to 603.0) |
| Week 8 | NA (NA to 27.0) | 25.5 (NA to 51) |
| Week 12 | 33.75 (26.6 to 40.9) | NA (NA to 10.3) |
| Week 24 | NA (NA to NA) | 16.4 (NA to 90.0) |
The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf)
| ng/mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 33800 (29500 to 36400) | 87500 (75300 to 114000) |
| Week 2 | 34950 (33300 to 36600) | 108200 (99400 to 117000) |
| Week 3 | 36750 (31300 to 42200) | 82700 (73300 to 92100) |
| Week 4 | 35700 (16150 to 36600) | 77400 (71800 to 102000) |
| Week 8 | 20800 (65.5 to 36600) | 50900 (33300 to 68600) |
| Week 12 | 30500 (32.5 to 36700) | 56900 (56100 to 71000) |
| Week 24 | 17500 (47.5 to 35400) | 93500 (69700 to 103000) |
The time of the end of infusion of SOBI003 (tEnd of inf)
| Hours | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 4.57 (4.27 to 4.62) | 4.33 (4.02 to 4.33) |
| Week 2 | 4.565 (4.30 to 4.83) | 4.245 (4.07 to 4.42) |
| Week 3 | 4.28 (4.13 to 4.43) | 4.1 (3.98 to 4.22) |
| Week 4 | 4.28 (4.05 to 4.50) | 4.42 (4.27 to 4.90) |
| Week 8 | 4.85 (4.42 to 6.58) | 8.17 (8.17 to 8.17) |
| Week 12 | 4.23 (4.10 to 6.42) | 6.83 (5.08 to 7.03) |
| Week 24 | 4.45 (4.08 to 4.53) | 4.75 (4.47 to 5.57) |
The Maximum Observed Serum Concentration of SOBI003 (Cmax)
| ng/mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 33800 (29500 to 36400) | 87500 (75300 to 114000) |
| Week 4 | 35700 (16150 to 36600) | 77400 (71800 to 102000) |
| Week 12 | 20800 (65.5 to 36400) | 56900 (56100 to 71000) |
| Week 24 | 17500 (47.5 to 35400) | 93500 (69700 to 103000) |
The time after start of infusion at which the maximum serum concentration is observed (tmax)
| Hours | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 4.57 (4.27 to 4.62) | 4.33 (4.02 to 4.33) |
| Week 4 | 4.28 (4.05 to 4.50) | 4.42 (4.27 to 4.90) |
| Week 12 | 4.23 (4.10 to 6.42) | 6.83 (5.08 to 7.03) |
| Week 24 | 4.45 (4.08 to 4.53) | 4.75 (4.47 to 5.57) |
The minimum observed serum concentration of SOBI003 (CTrough)
| ng/mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | NA (NA to 2010) | NA (NA to NA) |
| Week 4 | 28.9 (NA to 61.3) | 115 (20.8 to 7730) |
| Week 12 | 26.2 (17.0 to 32.5) | NA (NA to 29.7) |
| Week 24 | 44.8 (NA to 47.5) | NA (14.6 to 9010) |
Clearance (CL) of SOBI003
| (mL/h)/kg | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 10.4 (9.7 to 11.1) | 12.8 (12.0 to 13.6) |
| Week 4 | 8.1 (8.0 to 8.2) | 12.75 (12.4 to 13.1) |
| Week 12 | 9.5 (7.8 to 11.2) | 13.45 (12.1 to 14.8) |
| Week 24 | 19.5 (10.2 to 28.8) | 10.4 (9.9 to 10.9) |
Area under the serum concentration-time curve from time 0 to 168 hours (AUC 0-168h)
| h*ng/mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 290044.5 (275152 to 304937) | 834411 (737687 to 2492658) |
| Week 4 | 369915.5 (365435 to 374396) | 789106.5 (761760 to 816453) |
| Week 12 | 324999.5 (267722 to 382277) | 749864 (674188 to 828540) |
| Week 24 | 198973 (104009 to 293937) | 961516.5 (915941 to 1007092) |
The half-life of SOBI003 in serum (T1/2)
| Hours | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 1 | 19.8 (5.4 to 34.2) | 22.75 (5.8 to 39.7) |
| Week 4 | 34.3 (29.7 to 38.9) | 7.65 (6.3 to 9.0) |
| Week 12 | 40.95 (34.4 to 47.5) | 10.75 (8.8 to 12.7) |
| Week 24 | 20.85 (9.3 to 32.4) | 15.95 (6.0 to 25.9) |
SOBI003 concentration in cerebrospinal fluid
| mg/L | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 12 | NA (NA to NA) | 17.8 (17.8 to 17.8) |
| Week 24 | NA (NA to NA) | 47.2 (12.2 to 64.6) |
Number of patients in each dose group having anti-drug antibodies in serum
| Participants | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Weeks 2 | 1 | 1 |
| Weeks 4 | 2 | 3 |
| Weeks 8 | 3 | 3 |
| Weeks 12 | 3 | 3 |
| Weeks 24 | 3 | 3 |
Percent of patients having anti-drug antibodies in cerebrospinal fluid
| percent of participants in dose group | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 12 | 33.3 | 66.7 |
| Week 24 | 66.7 | 100 |
Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
| percent change | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 12 | -5.4 (-33.0 to 7.7) | -52.8 (-64.6 to -28.4) |
| Week 24 | -17.5 (-26.5 to -14.7) | -50.9 (-59.4 to -21.2) |
Change from baseline in Heparan sulfate levels in serum
| mg/L | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Week 2 | -1.8070 (-1.891 to -0.660) | -1.7460 (-3.409 to -1.705) |
| Week 3 | -1.22 (-2.006 to -0.220) | -1.9190 (-3.576 to -1.856) |
| Week 4 | -1.9040 (-2.083 to -1.850) | -1.9229 (-3.770 to -1.268) |
| Week 8 | -2.1030 (-2.210 to -1.615) | -1.8290 (-3.730 to -1.772) |
| Week 12 | -1.8170 (-2.131 to -1.597) | -1.8340 (-3.729 to -1.722) |
| Week 24 | -1.7480 (-2.207 to -1.657) | -1.8310 (-3.722 to -1.818) |
Change from baseline in Heparan sulfate levels in urine
| g/mol | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Weeks 2 | -456.2 (-496.1 to -298) | -561.1 (-592.1 to -231.9) |
| Weeks 3 | -494.67 (-592.1 to -390.0) | -597.4 (-618.8 to -309.3) |
| Weeks 4 | -498.53 (-575.8 to -422.79) | -503.465 (-672.17 to -334.76) |
| Weeks 8 | -474.7 (-542.22 to -460.95) | -666.81 (-672.42 to -341.70) |
| Weeks 12 | -534.1 (-540.71 to -442.33) | -640.2 (-668.83 to -326.13) |
| Weeks 24 | -421.98 (-557.33 to -258.1) | -695.9 (-698.03 to -348.05) |
Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
| unitless | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in Neurocognitive Development Quotient | -8.7 (-11.2 to -4.9) | -15.68 (-53.3 to 4.1) |
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
| Months | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in Age-equivalence Score | -1.0 (-4 to 1) | -3.0 (-6 to 6) |
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
| Months | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in Age-equivalence Score as Assessed by VABS-II | 1.0 (-9 to 2) | 0.0 (-3 to 24) |
Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI).
| mL | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in Gray Matter Volume | 10.858 (-6.22 to 42.27) | 39.129 (-33.28 to 111.53) |
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144.
| Units on a scale | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score | -9.3 ± 15.52 | -7.3 ± 14.9 |
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The measure includes a scale, from where the categorical score "4", "3", "2", "1", and "0" was reversed and linearly transformed to a 0-100 scale to 4=0, 3=25, 2=50, 1=75 and 0=100, where 100 = minimum and 0 = maximum. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning.
| Units on a scale | Dose Group 1 | Dose Group 2 |
|---|---|---|
| Change From Baseline in PedsQL™ Family Impact Module Total Score | -9.77 ± 19.05 | 1.3 ± 3.11 |
Collected over The period for recording adverse events, including Serious Adverse Events (SAEs), began upon receiving the first dose of SOBI003 and ended at completion of the week 24 visit. In addition SAEs was reported from the time for signing the informed consent form until 28 days past the last dose of SOBI003.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Dose Group 1 | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Dose Group 2 | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Event | Dose Group 1 | Dose Group 2 |
|---|---|---|
| PyrexiaGeneral disorders | 1/3 | 0/3 |
| Device related infestationsInfections and infestations | 0/3 | 1/3 |
| EndocarditisInfections and infestations | 0/3 | 1/3 |
| PneumoniaInfections and infestations | 0/3 | 1/3 |
| Event | Dose Group 1 | Dose Group 2 |
|---|---|---|
| TachycardiaCardiac disorders | 1/3 | 3/3 |
| VomitingGastrointestinal disorders | 1/3 | 3/3 |
| Upper respiratory tract infectionInfections and infestations | 3/3 | 2/3 |
| UrticariaSkin and subcutaneous tissue disorders | 2/3 | 3/3 |
| PyrexiaGeneral disorders | 0/3 | 3/3 |
| Vascular access site occlusionInjury, poisoning and procedural complications | 1/3 | 2/3 |
| TremorNervous system disorders | 1/3 | 2/3 |
| RestlessnessPsychiatric disorders | 0/3 | 2/3 |
| Iron deficiency anaemiaBlood and lymphatic system disorders | 1/3 | 1/3 |
| Medical device site haemorrhageGeneral disorders | 1/3 | 0/3 |
| Age, Continuous(months) | Dose Group 1 | Dose Group 2 | Total |
|---|---|---|---|
| Mean | 46.3 ± 24.2 | 29.0 ± 12.2 | 37.7 ± 19.6 |
| Sex: Female, Male(Participants) | Dose Group 1 | Dose Group 2 | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 2 | 2 | 4 |
| Ethnicity (NIH/OMB)(Participants) | Dose Group 1 | Dose Group 2 | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 3 | 3 | 6 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Dose Group 1 | Dose Group 2 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 3 | 3 | 6 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Undecided
Ran from Jun 2018 to primary completion in Oct 2019, 16 months.
Results were first posted in Apr 2021, 1 year 5 months after primary completion.
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