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Format should be in the following format: Activity Code, Institute Abbreviation, and Serial Number. Grant Type, Support Year, and Suffix should be excluded. For example, grant 1R01MH123456-01A1 should be entered R01MH123456

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To add an existing Data Structure, enter its title in the search bar. If you need to request changes, select the indicator "No, it requires changes to meet research needs" after selecting the Structure, and upload the file with the request changes specific to the selected Data Structure. Your file should follow the Request Changes Procedure. If the Data Structure does not exist, select "Request New Data Structure" and upload the appropriate zip file.

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Collection Summary Collection Charts
Collection Title Collection Investigators Collection Description
Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome (FXS)
Elizabeth Berry-Kravis 
Fragile X syndrome FXS is the most common inherited cause of intellectual disability and autism spectrum disorder. Enormous progress in basic and translational FXS research has allowed identification of neuronal pathway targets for treatment of the underlying disorder. The most well-studied of these has been pharmacological using mGluR5 negative allosteric modulators NAMs and genetic reduction of excessive mGluR5 translational pathway signaling to correct abnormal synaptic plasticity, dendritic morphology, cellular signaling, electrophysiological, cognitive, social, behavioral and even growth phenotypes in FXS models. Human early phase trials of mGluR5 NAMs fenobam Neuropharm, AFQ056 Novartis and RO4917523 Roche have suggested possible benefit in FXS, but larger phase IIb studies of AFQ056 and RO4917523 failed to meet primary behavioral outcomes in adolescents adults. These trials may have failed due to lack of measurement of core FXS phenotypes of cognition and learning in a sufficiently young population over a long enough period of time. The proposed project will seek to determine whether the human mGluR5 NAM trials failed because the animal model is not adequate or because the trials were not conducted in a way that benefit could be seen in a neurodevelopmental disorder NDD. Indeed it may be that the benefits of translational work from preclinical models of NDDs can never be realized via standard drug development pathways. This NeuroNEXT trial seeks to use an innovative exploratory design to change the paradigm for translation of targeted treatments in FXS and determine whether AFQ056 can improve language learning in 100 very young age 3-6 years children with FXS during participation in an intensive language learning intervention LLI, as a surrogate for enhanced neural plasticity. The trial will use a double blind placebo-controlled parallel flexible- dose forced-titration design with a 4 month placebo-lead-in, randomization to AFQ056 or placebo and titration to maximum tolerated dose MTD, 6 months treatment with AFQ056 placebo combined with the LLI, an open- label extension with re-titration to MTD followed by 6 months of treatment of all participants with LLI and AFQ056, and follow up off AFQ056 at the end of the trial. The study will seek to assess effects of AFQ056 versus placebo on developmental language, cognitive, adaptive and maladaptive functioning in young children with FXS receiving the LLI Aim 1, evaluate safety and tolerability of long-term exposure to AFQ056 in young children with FXS Aim 2, validate biomarkers that interrogate neural functioning event-related potentials and eye tracking and cellular pathway signaling during treatment with AFQ056 and or LLI and correlate these with developmental outcomes Aim 3, validate the LLI by evaluating its effects on trajectories of language, cognitive, adaptive, and maladaptive functioning Aim 4. If the design is successful, this trial can serve as a model for future trials of mechanistically-targeted treatments operating on neural plasticity in other NDDs, and can accelerate the process of bringing needed treatments to these disorders with high unmet need.
NIMH Data Archive
09/29/2016
Funding Completed
Close Out
Yes
$11,530,344.00
97
Imaging+Omics0Clinical1879
setssize
2
3
118
1,279
2,3
1,3
1,2,3
NIH - Extramural None

QA-notification.txt Other Quality Assurance Notification Qualified Researchers
NN107 Protocol_V15.0.pdf Analysis Protocol Protocol Qualified Researchers
NN107_SAP_Version1.0_wSignatures.pdf Other Statistical Analysis Plan Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V4 to V5.pdf Other List of Changes Protocol V4 to V5 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V3 to V4.pdf Other List of Changes Protocol V3 to V4 Qualified Researchers
NN107 Model ICF v12.pdf Other Informed Consent Form Template Qualified Researchers
MOP Sections.zip Other Manual of Procedures Qualified Researchers
Supporting Documentation for Data Submissions 06DEC2022.docx Other Supporting documentation for QA of data submission Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V14 to V15.pdf Other List of Changes Protocol V14 to V15 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V13 to V14.pdf Other List of Changes Protocol V13 to V14 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V12 to V13.pdf Other List of Changes Protocol V12 to V13 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V11 to V12.pdf Other List of Changes Protocol V11 to V12 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V10 to V11.pdf Other List of Changes Protocol V10 to V11 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V9 to V10.pdf Other List of Changes Protocol V9 to V10 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V8 to V9.pdf Other List of Changes Protocol V8 to V9 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V7 to V8.pdf Other List of Changes Protocol V7 to V8 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V6 to V7.pdf Other List of Changes Protocol V6 to V7 Qualified Researchers
List of Changes_NN107 FX-LEARN_Protocol V5 to V6.pdf Other List of Changes Protocol V5 to V6 Qualified Researchers
NN107 Annotated CRF_Vital Signs_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Weighted Communication Site Collection_eCRF Version 4.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Weighted Intentional Communication Score_eCRF Version 3.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Protocol Deviation_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_ABC_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Adverse Event_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Auditory ERP_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Autism Diagnostic Observation Scale_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Blood for PK_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_CGI-I_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_CGI-S_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Child Sleep Habits_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Clinician Rating of Caregiver Quality of Involvement_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Concomitant Medications_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Concomitant Non-pharmacological Interventions_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Demographics_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Dose Titration_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Drug Accountability_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Drug Dispensing_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Electrocardiogram (ECG)_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Eye Tracking_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Funduscopic Exam_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Global Unique Identifier_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Informed Consent_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_MacArthur-Bates CDI_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Maximum Tolerated Dose_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Medical History General_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Medical History_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Medical History_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Mullen Scales of Early Learning_eCRF Version 3.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Neurological Exam_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Parent Education Session_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Physical Exam_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Preschool Language Scales - 5_eCRF Version 3.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Pre-Visit 3 Screen Fail_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Prior Medications for Fragile X_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Prior Non-Pharmacological Interventions for Fragile X_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Protocol Deviation Review_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Psychiatric-Behavioral Exam_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Reportable Event – Unanticipated Problem (UAP) Not an adverse event (AE)_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Research Labs for Biomarkers and Safety_eCRF Version 3.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_SAE Evaluation_eCRF Version 2.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Safety Follow-up_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Screening Eligibility_eCRF Version 5.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Study Drug Hold_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Study Termination_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Suicidality Screen_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_VAS-B_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_VAS-L_eCRF Version 1.00.pdf Other CRF Qualified Researchers
NN107 Annotated CRF_Vineland Adaptive Behavior Scales_eCRF Version 2.00.pdf Other CRF Qualified Researchers


U01NS096767-01 Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome (FXS) 09/30/2016 06/30/2023 100 71 RUSH UNIVERSITY MEDICAL CENTER $11,530,344.00

The purpose of this clinical trial is to investigate the impact of AFQ056 on language learning in 3-6 year old children with Fragile X Syndrome (FXS). Completed NCT02920892 ORA 15060903 Elizabeth Berry-Kravis, MD, PhD August 17, 2017 May 17, 2022
Edit
helpcenter.filter-cart

NDA Help Center

Filter Cart

Viewable at the top right of NDA pages, the Filter Cart is a temporary holder for filters and data they select. Filters are added to the Workspace first, before being submitted to The Filter Cart. Data selected by filters in the Filter Cart can be added to a Data Package or an NDA Study from the Data Packaging Page, by clicking the 'Create Data Package / Add Data to Study' button.

The filter cart supports combining multiple filters together, and depending on filter type will use "AND" or "OR" when combining filters.

Multiple selections from the same filter type will result in those selections being applied with an ‘OR’ condition. For example, if you add an NDA Collection Filter with selections for both collections 2112 and 2563 to an empty Workspace, the subjects from NDA Collection 2112 ‘OR’ NDA Collection 2563 will be added to your Workspace even if a subject is in both NDA Collections. You can then add other NDA Collections to your Workspace which further extends the ‘OR’ condition.

If a different filter type is added to your Workspace, or a filter has already been submitted to the Filter Cart, the operation then performs a logical ‘AND’ operation. This means that given the subjects returned from the first filter, only those subjects that matched the first filter are returned by the second filter (i.e., subjects that satisfied both filters).

When combining other filters with the GUID filter, please note the GUID filter should be added last. Otherwise, preselected data may be lost. For example, a predefined filter from Featured Datasets may select a subset of data available for a subject. When combined with a GUID filter for the same subject, the filter cart will contain all data available from that subject, data structure, and dataset; this may be more data than was selected in the predefined filter for that subject. Again, you should add the GUID Filter as the last filter to your cart. This ensures 'AND' logic between filters and will limit results to the subjects, data structures, and datasets already included in your filter cart.

Note that only the subjects specific to your filter will be added to your Filter Cart and only on data shared with the research community. Other data for those same subjects may exist (i.e., within another NDA Collection, associated with a data structure that was not requested in the query, etc.). So, users should select ‘Find all Subjects Data’ to identify all data for those specific subjects.

Additional Tips:

  • You may query the data without an account, but to gain access you will need to create an NDA user account and apply for access. Most data access requires that you or your lab are sponsored by an NIH recognized institution with Federal Wide Assurance (FWA). Without access, you will not be able to obtain individual-level data.

Once you have selected data of interest you can:

  • Create a data package - This allows you to specify format for access/download
  • Assign to Study Cohort - Associate the data to an NDA Study allowing for a DOI to be generated and the data to be linked directly to a finding, publication, or data release.
  • Find All Subject Data - Depending on filter types being used, not all data associated with a subject will be selected. Data may be restricted by data structure, NDA Collection, or outcome variables (e.g., NDA Study). ‘Find All Data’ expands the filter criteria by replacing all filters in your Filter Cart with a single Query by GUID filter for all subjects selected by those filters.

Please Note:

  • When running a query, it may take a moment to populate the Filter Cart. Queries happen in the background so you can define other queries during this time.
  • When you add your first filter, all data associated with your query will be added to the Filter Cart (e.g., a Concept, an NDA Collection, a Data Structure/Element, etc.). As you add additional filters, they will also display in the Filter Cart. Only the name of filter will be shown in the Filter Cart, not the underlying structures.
  • Information about the contents of the Filter Cart can be seen by clicking "Edit”.
  • Once your results appear in the Filter Cart, you can create a data package or assign subjects to a study by selecting the 'Package/Assign to Study' option. You can also 'Edit' or 'Clear' filters.

Frequently Asked Questions

  • What is a Filter Cart?
  • What do I do after filters are added to the Filter Cart?
  • Are there limitations on the amount of data a user can download?
  • How does Filter Cart Boolean logic work?

Glossary

  • Workspace
  • Filter Cart
helpcenter.collection.general-tab

NDA Help Center

Collection - General Tab

Fields available for edit on the top portion of the page include:

  • Collection Title
  • Investigators
  • Collection Description
  • Collection Phase
  • Funding Source
  • Clinical Trials

Collection Phase: The current status of a research project submitting data to an NDA Collection, based on the timing of the award and/or the data that have been submitted.

  • Pre-Enrollment: The default entry made when the NDA Collection is created.
  • Enrolling: Data have been submitted to the NDA Collection or the NDA Data Expected initial submission date has been reached for at least one data structure category in the NDA Collection.
  • Data Analysis: Subject level data collection for the research project is completed and has been submitted to the NDA Collection. The NDA Collection owner or the NDA Help Desk may set this phase when they’ve confirmed data submission is complete and submitted subject counts match at least 90% of the target enrollment numbers in the NDA Data Expected. Data submission reminders will be turned off for the NDA Collection.
  • Funding Completed: The NIH grant award (or awards) associated with the NDA Collection has reached its end date. NDA Collections in Funding Completed phase are assigned a subphase to indicate the status of data submission.
    • The Data Expected Subphase indicates that NDA expects more data will be submitted
    • The Closeout Subphase indicates the data submission is complete.
    • The Sharing Not Met Subphase indicates that data submission was not completed as expected.

Blinded Clinical Trial Status:

  • This status is set by a Collection Owner and indicates the research project is a double blinded clinical trial. When selected, the public view of Data Expected will show the Data Expected items and the Submission Dates, but the targeted enrollment and subjects submitted counts will not be displayed.
  • Targeted enrollment and subjects submitted counts are visible only to NDA Administrators and to the NDA Collection or as the NDA Collection Owner.
  • When an NDA Collection that is flagged Blinded Clinical Trial reaches the maximum data sharing date for that Data Repository (see https://nda.nih.gov/nda/sharing-regimen.html), the embargo on Data Expected information is released.

Funding Source

The organization(s) responsible for providing the funding is listed here.

Supporting Documentation

Users with Submission privileges, as well as Collection Owners, Program Officers, and those with Administrator privileges, may upload and attach supporting documentation. By default, supporting documentation is shared to the general public, however, the option is also available to limit this information to qualified researchers only.

Grant Information

Identifiable details are displayed about the Project of which the Collection was derived from. You may click in the Project Number to view a full report of the Project captured by the NIH.

Clinical Trials

Any data that is collected to support or further the research of clinical studies will be available here. Collection Owners and those with Administrator privileges may add new clinical trials.

Frequently Asked Questions

  • How does the NIMH Data Archive (NDA) determine which Permission Group data are submitted into?
  • How do I know when a NDA Collection has been created?
  • Is a single grant number ever associated with more than one Collection?
  • Why is there sometimes more than one grant included in a Collection?

Glossary

  • Administrator Privilege
  • Collection Owner
  • Collection Phase
  • Collection Title
  • Grant
  • Supporting Documentation
  • NIH Research Initiative
  • Permission Group
  • Planned Enrollment
  • Actual Enrollment
  • NDA Collection
  • Data Use Limitations
  • Total Subjects Shared
helpcenter.collection.experiments-tab

NDA Help Center

Collection - Experiments

The number of Experiments included is displayed in parentheses next to the tab name. You may download all experiments associated with the Collection via the Download button. You may view individual experiments by clicking the Experiment Name and add them to the Filter Cart via the Add to Cart button.

Collection Owners, Program Officers, and users with Submission or Administrative Privileges for the Collection may create or edit an Experiment.

Please note: The creation of an NDA Experiment does not necessarily mean that data collected, according to the defined Experiment, has been submitted or shared.

Frequently Asked Questions

  • Can an Experiment be associated with more than one Collection?

Glossary

  • Experiment Status
  • Experiment ID
helpcenter.collection.shared-data-tab

NDA Help Center

Collection - Shared Data

This tab provides a quick overview of the Data Structure title, Data Type, and Number of Subjects that are currently Shared for the Collection. The information presented in this tab is automatically generated by NDA and cannot be edited. If no information is visible on this tab, this would indicate the Collection does not have shared data or the data is private.

The shared data is available to other researchers who have permission to access data in the Collection's designated Permission Group(s). Use the Download button to get all shared data from the Collection to the Filter Cart.

Frequently Asked Questions

  • How will I know if another researcher uses data that I shared through the NIMH Data Archive (NDA)?
  • Can I get a supplement to share data from a completed research project?
  • Can I get a supplement to share data from a research project that is still ongoing?

Glossary

  • Data Structure
  • Data Type
  • Shared
helpcenter.collection.publications-tab

NDA Help Center

Collection - Publications

The number of Publications is displayed in parentheses next to the tab name. Clicking on any of the Publication Titles will open the Publication in a new internet browsing tab.

Collection Owners, Program Officers, and users with Submission or Administrative Privileges for the Collection may mark a publication as either Relevant or Not Relevant in the Status column.

Frequently Asked Questions

  • How can I determine if a publication is relevant?
  • Where does the NDA get the publications?

Glossary

  • Create Study
  • Not Determined Publication
  • Not Relevant Publication
  • PubMed
  • PubMed ID
  • Relevant Publication
helpcenter.collection.data-expected-tab

NDA Help Center

Collection - Data Expected

The Data Expected tab displays the list of all data that NDA expects to receive in association with the Collection as defined by the contributing researcher, as well as the dates for the expected initial upload of the data, and when it is first expected to be shared, or with the research community. Above the primary table of Data Expected, any publications determined to be relevant to the data within the Collection are also displayed - members of the contributing research group can use these to define NDA Studies, connecting those papers to underlying data in NDA.

The tab is used both as a reference for those accessing shared data, providing information on what is expected and when it will be shared, and as the primary tracking mechanism for contributing projects. It is used by both contributing primary researchers, secondary researchers, and NIH Program and Grants Management staff.

Researchers who are starting their project need to update their Data Expected list to include all the Data Structures they are collecting under their grant and set their initial submission and sharing schedule according to the NDA Data Sharing Regimen.

To add existing Data Structures from the Data Dictionary, to request new Data Structure that are not in the Dictionary, or to request changes to existing Data Structures, click "+New Data Expected".

For step-by-step instructions on how to add existing Data Structures, request changes to an existing Structure, or request a new Data Structure, please visit the Completing Your Data Expected Tutorial.

If you are a contributing researcher creating this list for the first time, or making changes to the list as your project progress, please note the following:

  • Although items you add to the list and changes you make are displayed, they are not committed to the system until you Save the entire page using the "Save" button at the bottom of your screen. Please Save after every change to ensure none of your work is lost.
  • If you attempt to add a new structure, the title you provide must be unique - if another structure exists with the same name your change will fail.
  • Adding a new structure to this list is the only way to request the creation of a new Data Dictionary definition.

Frequently Asked Questions

  • What is an NDA Data Structure?
  • What is the NDA Data Dictionary?

Glossary

  • Analyzed Data
  • Data Item
  • Data Structure
  • Data Structure Category
  • Data Structure Group
  • Evaluated Data
  • Imaging Data
  • Initial Share Date
  • Initial Submission Date
  • Research Subject and Pedigree
  • Submission Cycle
  • Submission Exemption
helpcenter.collection.associated-studies-tab

NDA Help Center

Collection - Associated Studies

Clicking on the Study Title will open the study details in a new internet browser tab. The Abstract is available for viewing, providing the background explanation of the study, as provided by the Collection Owner.

Primary v. Secondary Analysis: The Data Usage column will have one of these two choices. An associated study that is listed as being used for Primary Analysis indicates at least some and potentially all of the data used was originally collected by the creator of the NDA Study. Secondary Analysis indicates the Study owner was not involved in the collection of data, and may be used as supporting data.

Private v. Shared State: Studies that remain private indicate the associated study is only available to users who are able to access the collection. A shared study is accessible to the general public.

Frequently Asked Questions

  • How do I associate a study to my collection?

Glossary

  • Associated Studies Tab