Down Syndrome
Conditions
Keywords
cognitive decline
Brief summary
The purpose of this study is to test in adults with Down Syndrome the safety, tolerability and immunogenicity of a vaccine, ACI-24.
Detailed description
This is a prospective multi-center, placebo controlled, double-blind and randomized dose escalation study of 2 doses of ACI-24 versus Placebo over 24 months with a total of 21 visits. All subjects will receive the study medication (ACI-24 or Placebo) 7 times via s.c. injection (12 months) and will be followed up for 12 months after the last dose with a final safety and efficacy assessment.
Interventions
BIOLOGICALACI-24 low dose
ACI-24 administered as a sterile suspension in PBS via s.c. injection.
BIOLOGICALACI-24 high dose
ACI-24 administered as a sterile suspension in PBS via s.c. injection.
BIOLOGICALPlacebo
Placebo is a standard PBS sterile solution administrated via s.c. injection.
Sponsors
National Institute on Aging (NIA)
Alzheimer's Disease Cooperative Study (ADCS)
LuMind IDSC Foundation
AC Immune SA
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
25 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* Males or females with Down Syndrome aged ≥25 to ≤45 years, with a cytogenetic diagnosis being either Trisomy 21 or Complete Unbalanced Translocation of the Chromosome 21. * Subjects and their study partner/legal representative in the opinion of the investigator able to understand and to provide written informed consent. * Written informed consent obtained from subjects and their study partner/legal representative before any trial-related activities. * In the opinion of the investigator able to fully participate in the trial and sufficiently proficient in English to be capable of reliably completing study assessments. * Subjects have a study partner/legal representative who have direct contact with the subjects at least 10 hours per week and who can be asked questions about the subjects.
Exclusion criteria
* Subjects weighing less than 40 kg. * IQ less than 40 (as assessed by Kaufman Brief Intelligence Test, Second Edition (KBIT-2). * In the investigators opinion, any clinically significant current psychiatric or neurologic illness, including a past illness with a risk of recurrence, other than Down syndrome. * Any medical condition likely to significantly hamper the evaluation of safety of the study drug. * DSM-IV criteria for drug or alcohol abuse or dependence currently met within the past five years. * History or presence of uncontrolled seizures. If history of seizures, they must be well controlled with no occurrence of seizures in the past 2 years prior to study screening. The use of anti-epileptic medications is permitted. * History of meningitis or meningoencephalitis. * History of malignant neoplasms within 3 years prior to study screening or where there is current evidence of recurrent or metastatic disease. * History of persistent cognitive deficits immediately following head trauma. * History of inflammatory neurology disorders. * History of autoimmune disease with potential for CNS involvement. * MRI scan at screening showing a single area of cerebral vasogenic edema, superficial siderosis, or evidence of a prior macrohemorrhage, or showing more than four cerebral microhemorrhages (regardless of their anatomical location or diagnostic characterization as possible or definite). * MRI examination cannot be done for any reason, including metal implants contraindicated for MRI studies and/or severe claustrophobia. * Significant hearing or visual impairment or other issues judged relevant by the investigator preventing to comply with the protocol and to perform the outcome measures. * Severe infections or a major surgical operation within 3 months prior to screening. * History of chronic or recurrent infections judged to be clinically significant by the investigator. * History or presence of immunological or inflammatory conditions which are judged to be clinically significant by the investigator. * Celiac disease not on a gluten free diet for at least 3 months prior to study screening. * Chronic benign skin pathologies, unless viewed as clinically insignificant in the investigator's opinion. * Any vaccine received within the past 2 months before baseline, except influenza vaccine which if indicated must be given at least 2 weeks prior to baseline. * Clinically significant arrhythmias or other abnormalities on ECG at screening. (Minor abnormalities documented as clinically insignificant by the investigator will be allowed.) * Clinically significant abnormal vital signs including sustained sitting blood pressure greater than 160/90 mmHg. * In the opinion of the site investigator, deviations from normal values for hematologic parameters, liver function tests, and other biochemical measures, that are judged to be clinically significant. * Subjects with treated hypothyroidism not on a stable dose of medication for at least 3 months prior to screening and having clinically significant abnormal serum T-4 and TSH at screening. * Subjects with diabetes mellitus with an HbA1c of ≥ 8.0%. * Subjects who have been receiving any experimental drug for Down Syndrome with a washout less than 30 days or less than five halflives of the drug, whichever is longer. * Female subjects being pregnant as confirmed by serum testing at screening or planning to be pregnant or lactating. * Female subjects not using a reliable method of contraception (unless abstaining). * Patient receiving any anticoagulant drug, or aspirin at doses greater than 100 mg daily in the 7 days prior to lumbar puncture (in order to avoid risk of bleeding during scheduled or unscheduled lumbar puncture) * Use of antidepressants other than SSRI/SNRIs at stable dose, antipsychotics (typical or atypical), GABA agonists (e.g. gabapentin), or stimulants (e.g. methylphenidate, modafinil). In exceptional cases, low doses of atypical antipsychotics (e.g. risperidone up to 0.5 mg/day or quetiapine up to 50 mg/day) or benzodiazepines are only allowed after review by the site principal investigator, in consultation with the project director and/or medical monitors. * Current use of immunosuppressant or immunomodulating drugs or their use within the past 6 months prior to study screening. Current use of steroids or their use within the past 3 months prior to study screening. * Use of Cholinesterase Inhibitor or use of Glutamatergic drugs (Topiramate, Memantine, Lamotrigine) if not on stable dose for at least 3 months prior to screening. * Subjects who have donated blood or blood products during the 30 days prior to screening who plan to donate blood while participating in the study or within four weeks after completion of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. The measure is expressed in Arbitrary Units per mL (AU/mL). AU/mL in a sample is obtained by back-calculation towards the standard curve. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| CANTAB - MOT Latency Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Cambridge Neuropsychological Test Automated Battery (CANTAB), Motor Screening Task (MOT) is a cognitive scale to be completed by the subject. Range score from 0 to ∞, lower score means a better outcome |
| CANTAB - PAL First Attempt Memory Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Cambridge Neuropsychological Test Automated Battery (CANTAB), Paired Associate Learning (PAL) is a cognitive scale to be completed by the subject. Range score from 0 to 20, higher score means a better outcome |
| Brief Praxis Test (BPT) - Total Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Brief Praxis Test (BPT) is a cognitive scale to be completed by the subject. Range score from 0 to 80, higher score means better outcome |
| Vineland II - Communication Domain Standard Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 113, higher score means a better outcome |
| Amyloid Beta 1-40 in Blood - Mean Absolute Value | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. |
| Vineland II - Socialisation - Domain Standard Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 115, higher score means a better outcome |
| NPI - Total Score | Values at baseline (week 0) and week 50 are reported. | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Neuropsychiatric Inventory (NPI) is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 144, higher score means a worse outcome |
| Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Clinical Global Impression of Change (CGIC) is a global assessment to be completed by the investigator. |
| Vineland II - Daily Living Skill - Domain Standard Score | Values at baseline (week 0) and week 50 are reported | All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 114, higher score means a better outcome |
Countries
United States
Participant flow
Pre-assignment details
Twenty subjects were screened (signed the informed consent) but four participants were considered screen failures
Participants by arm
| Arm | Count |
|---|---|
| ACI-24 300µg Cohort 1: Low dose | 6 |
| ACI-24 1000µg Cohort 2: High dose | 6 |
| Placebo Placebo cohort 1 + cohort 2 | 4 |
| Screen Failure Screen failure cohort 1 + cohort 2 | 4 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Withdrawal by Subject | 0 | 2 | 0 |
Baseline characteristics
| Characteristic | ACI-24 300µg | Total | Screen Failure | Placebo | ACI-24 1000µg |
|---|---|---|---|---|---|
| Age, Continuous | 33.5 Years STANDARD_DEVIATION 4.6 | 32.9 Years STANDARD_DEVIATION 4.1 | 34.0 Years STANDARD_DEVIATION 2.6 | 33.0 Years STANDARD_DEVIATION 4.2 | 31.5 Years STANDARD_DEVIATION 4.9 |
| BMI | 37.0 kg/m^2 STANDARD_DEVIATION 9.9 | 40.2 kg/m^2 STANDARD_DEVIATION 10.1 | 42.8 kg/m^2 STANDARD_DEVIATION 11.6 | 43.1 kg/m^2 STANDARD_DEVIATION 12.7 | 39.9 kg/m^2 STANDARD_DEVIATION 9.4 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 19 Participants | 4 Participants | 4 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| KBIT 2: IQ Composite | 44.2 IQ score STANDARD_DEVIATION 5.5 | 47.8 IQ score STANDARD_DEVIATION 10.4 | 52.8 IQ score STANDARD_DEVIATION 18.9 | 46.0 IQ score STANDARD_DEVIATION 8 | 49.2 IQ score STANDARD_DEVIATION 9.5 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 6 Participants | 20 Participants | 4 Participants | 4 Participants | 6 Participants |
| Region of Enrollment United States | 6 participants | 20 participants | 4 participants | 4 participants | 6 participants |
| Sex: Female, Male Female | 2 Participants | 11 Participants | 2 Participants | 3 Participants | 4 Participants |
| Sex: Female, Male Male | 4 Participants | 9 Participants | 2 Participants | 1 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 6 | 0 / 2 | 0 / 2 |
| other Total, other adverse events | 5 / 6 | 6 / 6 | 2 / 2 | 2 / 2 |
| serious Total, serious adverse events | 0 / 6 | 0 / 6 | 0 / 2 | 0 / 2 |
Outcome results
Primary
Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. The measure is expressed in Arbitrary Units per mL (AU/mL). AU/mL in a sample is obtained by back-calculation towards the standard curve.
Time frame: Values at baseline (week 0) and week 50 are reported
Population: Modified Intent to Treat Population (mITT)
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 0 | 632.3 AU/mL (AU: Arbitrary Unit) | Standard Deviation 570.9 |
| ACI-24 300µg | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 50 | 751.5 AU/mL (AU: Arbitrary Unit) | Standard Deviation 825.6 |
| ACI-24 1000µg | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 0 | 335.4 AU/mL (AU: Arbitrary Unit) | Standard Deviation 157.4 |
| ACI-24 1000µg | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 50 | 434.3 AU/mL (AU: Arbitrary Unit) | Standard Deviation 224.3 |
| Placebo | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 0 | 162.8 AU/mL (AU: Arbitrary Unit) | Standard Deviation 84.5 |
| Placebo | Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value | Week 50 | 153.3 AU/mL (AU: Arbitrary Unit) | Standard Deviation 140.7 |
Secondary
Amyloid Beta 1-40 in Blood - Mean Absolute Value
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered.
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 0 | 81.7 ng/L | Standard Deviation 56.6 |
| ACI-24 300µg | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 50 | 183.8 ng/L | Standard Deviation 18.7 |
| ACI-24 1000µg | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 0 | 138.5 ng/L | Standard Deviation 41.1 |
| ACI-24 1000µg | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 50 | 186.2 ng/L | Standard Deviation 37.8 |
| Placebo | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 0 | 169.5 ng/L | Standard Deviation 43.5 |
| Placebo | Amyloid Beta 1-40 in Blood - Mean Absolute Value | Week 50 | 181.3 ng/L | Standard Deviation 11.2 |
Secondary
Brief Praxis Test (BPT) - Total Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Brief Praxis Test (BPT) is a cognitive scale to be completed by the subject. Range score from 0 to 80, higher score means better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Brief Praxis Test (BPT) - Total Score | Week 0 | 72.0 score on a scale | Standard Deviation 5.8 |
| ACI-24 300µg | Brief Praxis Test (BPT) - Total Score | Week 50 | 75.5 score on a scale | Standard Deviation 4.8 |
| ACI-24 1000µg | Brief Praxis Test (BPT) - Total Score | Week 0 | 73.7 score on a scale | Standard Deviation 3.2 |
| ACI-24 1000µg | Brief Praxis Test (BPT) - Total Score | Week 50 | 77.5 score on a scale | Standard Deviation 2.5 |
| Placebo | Brief Praxis Test (BPT) - Total Score | Week 0 | 73.8 score on a scale | Standard Deviation 5.6 |
| Placebo | Brief Praxis Test (BPT) - Total Score | Week 50 | 76.5 score on a scale | Standard Deviation 2.5 |
Secondary
CANTAB - MOT Latency Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Cambridge Neuropsychological Test Automated Battery (CANTAB), Motor Screening Task (MOT) is a cognitive scale to be completed by the subject. Range score from 0 to ∞, lower score means a better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | CANTAB - MOT Latency Score | week 0 | 1784.5 score on a scale | Standard Deviation 904.4 |
| ACI-24 300µg | CANTAB - MOT Latency Score | Week 50 | 1781.2 score on a scale | Standard Deviation 960.4 |
| ACI-24 1000µg | CANTAB - MOT Latency Score | week 0 | 997.1 score on a scale | Standard Deviation 271.8 |
| ACI-24 1000µg | CANTAB - MOT Latency Score | Week 50 | 1046.3 score on a scale | Standard Deviation 152.1 |
| Placebo | CANTAB - MOT Latency Score | week 0 | 1024.3 score on a scale | Standard Deviation 193.9 |
| Placebo | CANTAB - MOT Latency Score | Week 50 | 1002.1 score on a scale | Standard Deviation 138.5 |
Secondary
CANTAB - PAL First Attempt Memory Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Cambridge Neuropsychological Test Automated Battery (CANTAB), Paired Associate Learning (PAL) is a cognitive scale to be completed by the subject. Range score from 0 to 20, higher score means a better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | CANTAB - PAL First Attempt Memory Score | week 0 | 8.0 score on a scale | Standard Deviation 5.1 |
| ACI-24 300µg | CANTAB - PAL First Attempt Memory Score | Week 50 | 7.5 score on a scale | Standard Deviation 4.1 |
| ACI-24 1000µg | CANTAB - PAL First Attempt Memory Score | week 0 | 9.2 score on a scale | Standard Deviation 5.1 |
| ACI-24 1000µg | CANTAB - PAL First Attempt Memory Score | Week 50 | 6.7 score on a scale | Standard Deviation 4.3 |
| Placebo | CANTAB - PAL First Attempt Memory Score | week 0 | 7.8 score on a scale | Standard Deviation 5.3 |
| Placebo | CANTAB - PAL First Attempt Memory Score | Week 50 | 9.8 score on a scale | Standard Deviation 4.6 |
Secondary
Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Clinical Global Impression of Change (CGIC) is a global assessment to be completed by the investigator.
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ACI-24 300µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | no change | 5 Participants |
| ACI-24 300µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Moderate improvement | 1 Participants |
| ACI-24 300µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Marked improvement | 0 Participants |
| ACI-24 1000µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | no change | 6 Participants |
| ACI-24 1000µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Marked improvement | 0 Participants |
| ACI-24 1000µg | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Moderate improvement | 0 Participants |
| Placebo | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | no change | 2 Participants |
| Placebo | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Moderate improvement | 1 Participants |
| Placebo | Clinical Global Impression of Change (CGIC) - Change From Baseline at Week 50 | Marked improvement | 1 Participants |
Secondary
NPI - Total Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Neuropsychiatric Inventory (NPI) is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 144, higher score means a worse outcome
Time frame: Values at baseline (week 0) and week 50 are reported.
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | NPI - Total Score | Week 0 | 4.5 score on a scale | Standard Deviation 3.6 |
| ACI-24 300µg | NPI - Total Score | Week 50 | 1.5 score on a scale | Standard Deviation 2.5 |
| ACI-24 1000µg | NPI - Total Score | Week 0 | 0.3 score on a scale | Standard Deviation 0.8 |
| ACI-24 1000µg | NPI - Total Score | Week 50 | 0.3 score on a scale | Standard Deviation 0.8 |
| Placebo | NPI - Total Score | Week 0 | 8.5 score on a scale | Standard Deviation 10 |
| Placebo | NPI - Total Score | Week 50 | 4.0 score on a scale | Standard Deviation 8 |
Secondary
Vineland II - Communication Domain Standard Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 113, higher score means a better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Vineland II - Communication Domain Standard Score | Week 0 | 47.7 score on a scale | Standard Deviation 10.2 |
| ACI-24 300µg | Vineland II - Communication Domain Standard Score | Week 50 | 52.2 score on a scale | Standard Deviation 11.3 |
| ACI-24 1000µg | Vineland II - Communication Domain Standard Score | Week 0 | 59.3 score on a scale | Standard Deviation 8.5 |
| ACI-24 1000µg | Vineland II - Communication Domain Standard Score | Week 50 | 65.2 score on a scale | Standard Deviation 9.4 |
| Placebo | Vineland II - Communication Domain Standard Score | Week 0 | 59.0 score on a scale | Standard Deviation 5.3 |
| Placebo | Vineland II - Communication Domain Standard Score | Week 50 | 61.0 score on a scale | Standard Deviation 6.8 |
Secondary
Vineland II - Daily Living Skill - Domain Standard Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 114, higher score means a better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Vineland II - Daily Living Skill - Domain Standard Score | Week 0 | 54.2 score on a scale | Standard Deviation 9.9 |
| ACI-24 300µg | Vineland II - Daily Living Skill - Domain Standard Score | Week 50 | 55.8 score on a scale | Standard Deviation 11.4 |
| ACI-24 1000µg | Vineland II - Daily Living Skill - Domain Standard Score | Week 0 | 68.3 score on a scale | Standard Deviation 4.1 |
| ACI-24 1000µg | Vineland II - Daily Living Skill - Domain Standard Score | Week 50 | 72.8 score on a scale | Standard Deviation 4.1 |
| Placebo | Vineland II - Daily Living Skill - Domain Standard Score | Week 0 | 58.0 score on a scale | Standard Deviation 9.3 |
| Placebo | Vineland II - Daily Living Skill - Domain Standard Score | Week 50 | 61.5 score on a scale | Standard Deviation 13.5 |
Secondary
Vineland II - Socialisation - Domain Standard Score
All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. Vineland II is a behavioral questionnaire to be completed by the study partner of the subject. Range score from 0 to 115, higher score means a better outcome
Time frame: Values at baseline (week 0) and week 50 are reported
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| ACI-24 300µg | Vineland II - Socialisation - Domain Standard Score | Week 0 | 64.3 score on a scale | Standard Deviation 17.2 |
| ACI-24 300µg | Vineland II - Socialisation - Domain Standard Score | Week 50 | 66.7 score on a scale | Standard Deviation 16.1 |
| ACI-24 1000µg | Vineland II - Socialisation - Domain Standard Score | Week 0 | 77.0 score on a scale | Standard Deviation 9.1 |
| ACI-24 1000µg | Vineland II - Socialisation - Domain Standard Score | Week 50 | 83.0 score on a scale | Standard Deviation 10.4 |
| Placebo | Vineland II - Socialisation - Domain Standard Score | Week 0 | 59.0 score on a scale | Standard Deviation 15.1 |
| Placebo | Vineland II - Socialisation - Domain Standard Score | Week 50 | 70.3 score on a scale | Standard Deviation 9.7 |