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We are terminating due to loss of funding because of Covid-19.
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| Name | Class |
|---|---|
| Novartis | INDUSTRY |
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Following the publication of two case studies that reported behavioral benefit in ASD patients treated with omalizumab, the investigators will conduct a pilot trial to test the proof-of-concept efficacy of omalizumab in ASD patients with comorbid atopic disease. Investigators will evaluate behavioral improvement using three questionnaires. Investigators will also perform fMRI on all subjects and obtain serum samples for quantification of immunological biomarkers. If the trial is conclusive, the investigators will conduct a larger-scale, randomized-controlled trial to further understand the pathology of allergy in this subpopulation of ASD patients and the efficacy of this intervention.
This clinical trial is a Phase I, single-arm, open-label study. All 20 subjects will be given the same dosage of the study drug. Following baseline testing, subjects will undergo treatment during a 24 week treatment period and will receive six subcutaneous injections total, one injection every 4 weeks. A 24-week follow-up period after the treatment period will be concluded with patient interview. Behavioral questionnaires will be administered throughout the duration of the trial. fMRI will be conducted at baseline and at the conclusion of the treatment period. Blood will be drawn for serum testing at baseline, at Week 12 during the treatment period, and at the conclusion of the treatment period. Vitals and CGI will also be assessed throughout the trial.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group | Experimental | Subjects will receive subcutaneous treatment of 300mg of omalizumab during the 24-week treatment period. One injection will be administered every 4 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Omalizumab Injection [Xolair] | Drug | 300mg via 2 subcutaneous injections every 4 weeks |
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| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline to Week 12 in Social Responsiveness Scale (SRS) Edition 2 | Social communication and behavior - The scoring is based on T-score which is based on the sum of responses as follows (76 to higher - severe, 66 to 75- moderate deficiencies, 60 to 65 - mild deficiencies, 59 and below is not clinically significant for ASD). | Baseline and Week 12 |
| Change from Baseline to Week 24 in Social Responsiveness Scale (SRS) Edition 2 | Social communication and behavior - The scoring is based on T-score which is based on the sum of responses as follows (76 to higher - severe, 66 to 75- moderate deficiencies, 60 to 65 - mild deficiencies, 59 and below is not clinically significant for ASD). | Baseline and Week 24 |
| Change from Baseline to Week 48 in Social Responsiveness Scale (SRS) Edition 2 | Social communication and behavior - The scoring is based on T-score which is based on the sum of responses as follows (76 to higher - severe, 66 to 75- moderate deficiencies, 60 to 65 - mild deficiencies, 59 and below is not clinically significant for ASD). | Baseline and Week 48 |
| Change from Week 24 to Week 48 in Social Responsiveness Scale (SRS) Edition 2 | Social communication and behavior - The scoring is based on T-score which is based on the sum of responses as follows (76 to higher - severe, 66 to 75- moderate deficiencies, 60 to 65 - mild deficiencies, 59 and below is not clinically significant for ASD). | Week 24 (conclusion of treatment period) and Week 48 (follow-up) |
| Change from Baseline to Week 12 in Aberrant Behavior Checklist (ABC) | Social behavior test - The total score is calculated based on 5 sub-scales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech). There are 58 questions that are scored on a 0-3 scale 0 -"not at all a problem", 1- "the behavior is a problem but slight in degree", 2- "the problem is moderately serious" and 3- "the problem is severe in degree". Based on this sub-scores are calculated and added to get the total score. |
| Measure | Description | Time Frame |
|---|---|---|
| Inflammatory Marker Levels | Inflammatory marker serum concentration quantification (free IgE, total IgE, interleukin 6, tumor necrosis factor alpha, tryptase). | Baseline, Week 12, and Week 24 (conclusion of treatment period) |
| Structural MRI |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impressions Scale - Improvement (CGI-I) | Psychological Clinical Assessment - Conducted by a physician, this 7-point scale characterizes changes in the subject's clinical presentation. | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
Inclusion criteria:
Exclusion criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Xue-Jun Kong, MD | Beth Israel Deaconess Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | United States |
There is no plan to share data with researchers not directly involved in this study and on the research team.
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| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D000069444 | Omalizumab |
| ID | Term |
|---|---|
| D000888 | Antibodies, Anti-Idiotypic |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
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Baseline testing, followed by 24-week treatment period, followed by 24-week follow-up period.
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| Baseline and Week 12 |
| Change from Baseline to Week 24 in Aberrant Behavior Checklist (ABC) | Social behavior test - The total score is calculated based on 5 sub-scales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech). There are 58 questions that are scored on a 0-3 scale 0 -"not at all a problem", 1- "the behavior is a problem but slight in degree", 2- "the problem is moderately serious" and 3- "the problem is severe in degree". Based on this sub-scores are calculated and added to get the total score. | Baseline and Week 24 (conclusion of treatment period) |
| Change from Baseline to Week 48 in Aberrant Behavior Checklist (ABC) | Social behavior test - The total score is calculated based on 5 sub-scales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech). There are 58 questions that are scored on a 0-3 scale 0 -"not at all a problem", 1- "the behavior is a problem but slight in degree", 2- "the problem is moderately serious" and 3- "the problem is severe in degree". Based on this sub-scores are calculated and added to get the total score. | Baseline and Week 48 (follow-up) |
| Change from Week 24 to Week 48 in Aberrant Behavior Checklist (ABC) | Social behavior test - The total score is calculated based on 5 sub-scales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech). There are 58 questions that are scored on a 0-3 scale 0 -"not at all a problem", 1- "the behavior is a problem but slight in degree", 2- "the problem is moderately serious" and 3- "the problem is severe in degree". Based on this sub-scores are calculated and added to get the total score. | Week 24 (conclusion of treatment period) and Week 48 (follow-up) |
| Change from Baseline to Week 1 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 1 |
| Change from Baseline to Week 4 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 4 |
| Change from Baseline to Week 8 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 8 |
| Change from Baseline to Week 12 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 12 |
| Change from Baseline to Week 16 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 16 |
| Change from Baseline to Week 20 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 20 |
| Change from Baseline to Week 24 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 24 (conclusion of treatment period) |
| Change from Baseline to Week 48 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Baseline and Week 48 (follow-up) |
| Change from Week 24 to Week 48 in Autism Treatment Evaluation Checklist (ATEC) | Social Behavior Test - The total score is calculated by adding together the scores of 4 subtests (Speech/Language Communication, Sociability, Sensory/Cognitive Awareness, and Health/Physical/Behavior). This 67 question test will provide scores for each subtest and a total score, for which a higher score indicates greater symptom severity. | Week 24 (conclusion of treatment period) and Week 48 (follow-up) |
The software Freesurfer will be used to calculate volume of brain regions and diffusion parameters (e.g. fractional anisotropy and mean diffusivity) from structural T1 and diffusion tensor images respectively. Paired t-test will be used to compare brain volume at baseline and post-treatment.
| Baseline and Week 24 (conclusion of treatment period) |
| Functional MRI (resting state) | Correlation analysis will be used to calculate the connectivity across different brain regions. Paired t-test will be used to compare differences in activity at baseline and post-treatment. | Baseline, and Week 24 (conclusion of treatment period) |
| Generalized Anxiety Disorder 7-item (GAD-7) | Anxiety symptom questionnaire - Scoring is based on how frequently the subject experienced 7 GAD-related symptoms in the last two weeks. Each question is rated on a 0-3 scale: 0 - "Not at all", 1 - "Several days", 2 - "More than half the days", 3 - "Nearly every day." Defined cutoffs will be used to define severity. | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Epworth Sleepiness Scale (ESS) | Sleepiness Questionnaire - Scoring is based on how likely the subject is to doze off or fall asleep during 8 daily activities. Higher scores indicate more severe sleepiness. | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Pittsburgh Sleep Quality Index (PSQI) | Sleep Quality Questionnaire - Scoring is based on 19 self-administered questions and 5 roommate questions. An algorithm provided with the questionnaire will be used to calculate global sleep quality score, for which a higher score indicates greater severity. | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Asthma Control Test (ACT) | A 5 question patient survey designed to identify patients with poorly controlled asthma based on the symptom severity over the last 4 weeks. | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Rhinitis Control Assessment Test (RCAT) | A 6 question patient survey designed to evaluate rhinitis symptom control based on symptom severity and frequency over the past week. | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Validated Investigator Global Assessment of Atopic Dermatitis (vIGA-AD) | A clinical assessment tool used to describe severity of atopic dermatitis. The vIGA-AD includes a single scale from 0-4, in which 0 is "clear" and 4 is "severe." | Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Clinical Global Impressions Scale - Severity (CGI-S) |
Psychological Clinical Assessment - Conducted by a physician, this 7-point scale defines severity of the subject's psychopathology. |
| Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Autonomic indices 1 | Blood volume pulse | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Autonomic indices 2 | Heart Rate | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Autonomic indices 3 | Blood oxygen saturation | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Autonomic indices 4 | Carboxyhemoglobin blood saturation | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| Autonomic indices 5 | Blood pressure | Baseline, Week 0, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 (conclusion of treatment period), and Week 48 (follow-up) |
| D001798 |
| Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D012712 | Serum Globulins |
| D005916 | Globulins |