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| Name | Class |
|---|---|
| Tuberous Sclerosis Alliance | OTHER |
| Autism Speaks | OTHER |
| Novartis Pharmaceuticals | INDUSTRY |
| Seizure Tracker LLC |
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Tuberous Sclerosis Complex (TSC) is a multi-system disease, usually presenting with seizures, mental retardation and autism, and exhibiting a high variability in clinical findings both among and within families. Investigators are doing research in order to identify possible neurocognitive benefits from treatment with RAD001 or placebo for a six month period. There may also be potential for improvements in seizure frequency, sleep and autistic behaviors. We hope this trial will lead to a better understanding of TSC and to new forms of treatment, to benefit children and adults with TSC in the future.
Individuals diagnosed with TSC will be asked to participate in this study if they are between the ages of 6 and 21 years of age and have an IQ of greater than or equal to 60. Both males and females will be asked to participate. Additionally, to be eligible for study participation, individuals must have been on the same seizure medication(s), if applicable, for at least 6 months. Individuals must also be able to participate in neuropsychological testing and meet certain medical criteria. They will need to sign an informed consent. If enrolled in the study, participants will have a number of screening tests to help determine if they are eligible for participation in the clinical trial. If eligible for the treatment phase of the trial, they will be asked to take either the study drug or a placebo (pill with no medicine), which is determined by chance.
The study involves about 9 visits, 3 of which can be done locally, over a six month period, as well as follow-up calls with our research nurse. Study visits will vary in length. Screening, three month and six month visits may last up to 8 hours, while all other visits will be less than 2 hours. The study visits include blood draws, laboratory tests and neuropsychological assessments. There is no fee to participate in this study. The study drug will be provided at no charge during the study.
After all study data has been analyzed, families will be informed of the overall results. Treatment on this study may or may not improve a child's learning skills (neurocognition). Future patients may benefit from what is learned.
This is a signal-seeking Phase II randomized, placebo-controlled trial of RAD001 in children and young adults with TSC with neurocognition as the primary outcome and autism spectrum disorder as a secondary outcome.
Specific Aims /Objectives Primary objective
Secondary objectives
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RAD001 | Experimental | RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. |
|
| Placebo | Placebo Comparator | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RAD001 | Drug | RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001 tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of RAD001 orally with a glass of water at regular intervals at the same time in the morning after a light, nonfat breakfast. |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | Evaluation of the safety of RAD001 compared with placebo in patients with TSC focusing on NCI CTCAE Grade 3 and 4 adverse events, serious adverse events, and Grade 3 and 4 laboratory toxicities. | 6 months |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Baseline and 6 month Timepoint scores are reported for the following primary outcome measures:
| 6 months |
| Evaluation of the Efficacy of RAD001 on Neurocognition (Cambridge Neuropsychological Test Automated Battery) in Patients With TSC Compared With Placebo. | Scores are reported for baseline and 6 month timepoints on the Cambridge Neuropsychological Test Automated Battery (CANTAB) subscales below. For all subscales, scores are reported as the mean difference between the study subjects and a normative population matched for age, gender and IQ (e.g., subject subscale score - mean of matched normative group = reported score). Higher scores represent a better outcome.
|
| Measure | Description | Time Frame |
|---|---|---|
| Comparison of Absolute Change From Baseline in Frequency of Epileptiform Events Between Patients Taking RAD001 vs. Placebo | Comparison of absolute change from baseline in frequency of epileptiform events as recorded on seizure diaries between patients taking RAD001 vs. placebo | 6 months |
| Comparison of Sleep Disturbances Between Patients Taking RAD001 vs. Placebo |
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Inclusion criteria:
Male or female patients ages 6 to 21 years of age.
IQ ≥60.
Ability to participate in direct neuropsychological and developmental testing.
English as primary language.
Diagnosis of tuberous sclerosis complex confirmed by genetic testing and/or clinically definite diagnosis of tuberous sclerosis complex according to the modified Gomez criteria and an IQ>60.
Stable anti-epileptic drugs (no changes in medications except dose for >6 months).
Adequate renal function. The GFR would be greater than 50 ml/min.m2 as determined by the Schwartz Formula for children and MDRD for adults:
http://www.nkdep.nih.gov/professionals/gfr\_calculators/index.htm
If female and of child bearing potential, documentation of negative pregnancy test prior to enrollment. Sexually active pre-menopausal female patients (and female partners of male patients) must use adequate contraceptive measures, excluding estrogen containing contraceptives, while on study. Abstinence will be considered an adequate contraceptive measure.
INR ≤1.5 (Anticoagulation is allowed if target INR ≤ 1.5 on a stable dose of warfarin or on a stable dose of LMW heparin for >2 weeks at time of randomization.)
Adequate liver function as shown by:
Written informed consent according to local guidelines.
Exclusion criteria:
Change of one or more antiepileptic medication in the past 6 months.
Prior exposure to the systemic use of an mTOR inhibitor.
Exposure to any investigational agent in the 30 days prior to randomization.
Neurosurgery within 6 months.
Known impaired lung function (e.g.FEV1 or DLCO <70% of predicted), if not resolved or if resolved within past 24 months.
Significant hematological or hepatic abnormality (i.e. transaminase levels > 2.5 x ULN or serum bilirubin > 1.5 x ULN, hemoglobin < 9 g/dL, platelets < 80,000/ mm3, absolute neutrophil count < 1,000/mm3).
Serum creatinine > 1.5 x ULN.
Uncontrolled hyperlipidemia: Fasting serum cholesterol > 300 mg/dL OR > 7.75 mmol/L AND Fasting triglycerides > 2.5 x ULN.
Uncontrolled diabetes mellitus as defined by fasting serum glucose > 1.5 x ULN.
Patients with bleeding diathesis or on oral anti-vitamin K medication (except low dose warfarin).
Patients with known history of HIV seropositivity.
Pregnancy or breast feeding.
Active infection at date of randomization.
Prior history of organ transplant.
Recent surgery (involving entry into a body cavity or requiring sutures) within the 4 weeks prior to randomization.
Inability to attend scheduled clinic visits.
History of malignancy in the past two years, other than squamous or basal cell skin cancer.
Patients should not receive immunization with attenuated live vaccines within one month of study entry or during study period. Close contact with those who have received attenuated live vaccines should be avoided during treatment with everolimus. Examples of live vaccines include intranasal influenza, measles, mumps, rubella, oral polio, BCG, yellow fever, varicella and TY21a typhoid vaccines.
Liver disease such as cirrhosis or severe hepatic impairment (Child-Pugh class C).
Note: A detailed assessment of Hepatitis B/C medical history and risk factors must be done at screening for all patients. HBV DNA and HCV RNA PCR testing are required at screening for all patients with a positive medical history based on risk factors and/or confirmation of prior HBV/HCV infection.
Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
Patients who have received an IQ score under 60 in the six months prior to the study screening visit will be deemed ineligible.
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| Name | Affiliation | Role |
|---|---|---|
| Mustafa Sahin, MD, PhD | Boston Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States | ||
| Cincinnati Children's Hospital Medical Center |
Not provided
| Label | URL |
|---|---|
| Boston Children's Hospital - Multi-Disciplinary Tuberous Sclerosis Program | View source |
| Tuberous Sclerosis Alliance | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | RAD001 | RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001: RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001 tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of RAD001 orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
| FG001 | Placebo | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Placebo: Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of the matching placebo orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | RAD001 | RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001: RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001 tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of RAD001 orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | Evaluation of the safety of RAD001 compared with placebo in patients with TSC focusing on NCI CTCAE Grade 3 and 4 adverse events, serious adverse events, and Grade 3 and 4 laboratory toxicities. | Posted | Number | Adverse Events | 6 months |
|
Not provided
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Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | RAD001 | RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001: RAD001 is formulated as tablets of 5.0 mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis. RAD001 tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of RAD001 orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Septicemia | Blood and lymphatic system disorders | CTCAE (3.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctivitis | Eye disorders | CTCAE (3.0) | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Mustafa Sahin | Boston Children's Hospital | 617-355-8994 | Mustafa.Sahin@childrens.harvard.edu |
Not provided
| ID | Term |
|---|---|
| D014402 | Tuberous Sclerosis |
| D001321 | Autistic Disorder |
| ID | Term |
|---|---|
| D006222 | Hamartoma |
| D009369 | Neoplasms |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
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| ID | Term |
|---|---|
| D000068338 | Everolimus |
| ID | Term |
|---|---|
| D020123 | Sirolimus |
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| UNKNOWN |
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|
|
| Placebo | Drug | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of the matching placebo orally with a glass of water at regular intervals at the same time in the morning after a light, nonfat breakfast. |
|
| 6 months |
Comparison of sleep disturbances between patients taking RAD001 vs. placebo, measured by the Pediatric Sleep Questionnaire (PSQ) and sleep logs |
| 6 months |
| Comparison of Autism Spectrum Disorders Features Between Patients Taking RAD001 vs. Placebo | Scores for the Baseline and 6 Month Timepoints are reported. The secondary outcome measure was the Social Responsiveness Scale (SRS). Standard scores are reported with a mean of 100 and standard deviation of 15. The range is 40-160 with higher scores indicating a better outcome. | 6 months |
| Comparison of Academic Skills Between Patients Taking RAD001 vs. Placebo | Scores are reported for Baseline and 6 Month Timepoints. The secondary outcome measure was the Wide Range Achievement Test 4 (WRAT4), which was used to assess academic skills. The Reading and Math subtests were used. Standard scores are reported which have a mean of 100 and a standard deviation of 15 (range=40-160 where higher is better). | 6 months |
| Comparison of Behavioral Problems Between Patients Taking RAD001 vs Placebo | Scores for Baseline and 6 Month Timepoints are reported for the following secondary outcome measures:
| 6 months |
| Cincinnati |
| Ohio |
| 45229 |
| United States |
| BG001 | Placebo | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Placebo: Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of the matching placebo orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 | Placebo | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Placebo: Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of the matching placebo orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. |
|
|
| Primary | Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Baseline and 6 month Timepoint scores are reported for the following primary outcome measures:
| Posted | Mean | Standard Deviation | units on a scale | 6 months |
|
|
|
| Primary | Evaluation of the Efficacy of RAD001 on Neurocognition (Cambridge Neuropsychological Test Automated Battery) in Patients With TSC Compared With Placebo. | Scores are reported for baseline and 6 month timepoints on the Cambridge Neuropsychological Test Automated Battery (CANTAB) subscales below. For all subscales, scores are reported as the mean difference between the study subjects and a normative population matched for age, gender and IQ (e.g., subject subscale score - mean of matched normative group = reported score). Higher scores represent a better outcome.
| Posted | Mean | Standard Deviation | units on a scale | 6 months |
|
|
|
| Secondary | Comparison of Absolute Change From Baseline in Frequency of Epileptiform Events Between Patients Taking RAD001 vs. Placebo | Comparison of absolute change from baseline in frequency of epileptiform events as recorded on seizure diaries between patients taking RAD001 vs. placebo | Data was not collected reliably and therefore was not analyzed. | Posted | No | 6 months |
|
|
| Secondary | Comparison of Sleep Disturbances Between Patients Taking RAD001 vs. Placebo | Comparison of sleep disturbances between patients taking RAD001 vs. placebo, measured by the Pediatric Sleep Questionnaire (PSQ) and sleep logs | Data was not collected reliably and therefore was not analyzed. | Posted | 6 months |
|
|
| Secondary | Comparison of Autism Spectrum Disorders Features Between Patients Taking RAD001 vs. Placebo | Scores for the Baseline and 6 Month Timepoints are reported. The secondary outcome measure was the Social Responsiveness Scale (SRS). Standard scores are reported with a mean of 100 and standard deviation of 15. The range is 40-160 with higher scores indicating a better outcome. | Posted | Mean | Standard Deviation | units on a scale (SRS) | 6 months |
|
|
|
| Secondary | Comparison of Academic Skills Between Patients Taking RAD001 vs. Placebo | Scores are reported for Baseline and 6 Month Timepoints. The secondary outcome measure was the Wide Range Achievement Test 4 (WRAT4), which was used to assess academic skills. The Reading and Math subtests were used. Standard scores are reported which have a mean of 100 and a standard deviation of 15 (range=40-160 where higher is better). | Posted | Mean | Standard Deviation | units on a scale | 6 months |
|
|
|
| Secondary | Comparison of Behavioral Problems Between Patients Taking RAD001 vs Placebo | Scores for Baseline and 6 Month Timepoints are reported for the following secondary outcome measures:
| Posted | Mean | Standard Deviation | units on a scale | 6 months |
|
|
|
| 3 |
| 32 |
| 30 |
| 32 |
| EG001 | Placebo | Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Placebo: Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10. Matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light-sensitive. Patients will be instructed to take 4.5 mg/m2 of the matching placebo orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, nonfat breakfast. | 0 | 15 | 10 | 15 |
| Pyelonephritis | Renal and urinary disorders | CTCAE (3.0) | Systematic Assessment |
|
| high fever/pneumonia | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Personality/Behavior | Social circumstances | CTCAE (3.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Stomach ache/pain/upset/irritation | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Stomach flu | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| stomach bug/virus/flu | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Bloody nose/nosebleed | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Body pain/swelling/generalized pain NOS | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Cavity | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Cold | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Cold Sores | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Congestion | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Cough | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Fall | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Fatigue | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Feeling Cold | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Fever | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Increased Energy | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Ingrown toenail | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Irritability | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Muscle cramp/soreness | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Runny nose | General disorders | CTCAE (3.0) | Systematic Assessment |
|
| Cellulitis | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Ear Infection | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Mouth Sore | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Mouth Ulcer | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Mouth irritation/pain | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Painful Urination | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Sore throat | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Stomach Infection | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Strep throat | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Viral infection | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
|
| Bruising/contusion | Injury, poisoning and procedural complications | CTCAE (3.0) | Systematic Assessment |
|
| Cuts/Abrasion | Injury, poisoning and procedural complications | CTCAE (3.0) | Systematic Assessment |
|
| Soreness due to injury | Injury, poisoning and procedural complications | CTCAE (3.0) | Systematic Assessment |
|
| Loss of appetite | Metabolism and nutrition disorders | CTCAE (3.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | CTCAE (3.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | CTCAE (3.0) | Systematic Assessment |
|
| Seizures | Nervous system disorders | CTCAE (3.0) | Systematic Assessment |
|
| Sleep Disturbance/Insomnia | Nervous system disorders | CTCAE (3.0) | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | CTCAE (3.0) | Systematic Assessment |
|
| Behavioral/personality changes | Psychiatric disorders | CTCAE (3.0) | Systematic Assessment |
|
| UTI/urinary frequency/incontinence | Renal and urinary disorders | CTCAE (3.0) | Systematic Assessment |
|
| Genital irritation | Reproductive system and breast disorders | CTCAE (3.0) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Phlegm | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Stuffy nose | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Upper respiratory infection/Upper Airways NOS | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | CTCAE (3.0) | Systematic Assessment |
|
| Abscess | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Acne/Pimples | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Athlete's foot | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Dry/itchy skin | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Rash/hives | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
|
| Tremor | Nervous system disorders | CTCAE (3.0) | Systematic Assessment |
|
| Triglyceride-serum high | Metabolism and nutrition disorders | CTCAE (3.0) | Systematic Assessment |
|
Not provided
Not provided
| D065703 |
| Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D000067877 | Autism Spectrum Disorder |
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
| EVT2 (Expressive Language) |
|
| WRAML2 Verbal Learning |
|
| WRAML2 Verbal Recall |
|
| VABS2 Adaptive Behavior Composite |
|
| VABS2 Communication |
|
| VABS2 Socialization |
|
| VABS2 Daily Living Skills |
|
| VABS2 Motor Skills |
|
| Pegboard, Dominant Hand |
|
| Pegboard, Non-Dominant Hand |
|
| CANTAB Spatial Recognition Memory |
|
| CANTAB Spatial Span |
|
| CANTAB Spatial Working Memory |
|
| CANTAB Reaction Time |
|
| CANTAB Rapid Visual Processing |
|
| CANTAB Stockings of Cambridge |
|
| CANTAB IDED |
|
| SRS Social Awareness |
|
| SRS Social Cognition |
|
| SRS Social Communication |
|
| SRS Social Motivation |
|
| SRS Autism Mannerisms |
|
| Math Computation (WRAT 4) |
|
| BRIEF Behavioral Regulation Index |
|
| BRIEF Metacognition Index |
|
| BASC2 Behavioral Symptoms Index |
|
| BASC2 Externalizing Problems |
|
| BASC2 Internalizing Problems |
|
| BASC2 Adaptive Skills |
|
| SDQ Total Difficulties Scale |
|