NFXF Data Repository

There are currently three available datasets, learn more about each below.

1. Review the NFXF Data Use Policy information
   Applicants will need to review the information in our Data Use Policy. This document is also available in the application. If approved, you’ll be asked to sign this document and return it to us.
2. Complete and submit a Data Access Application
   See below.
3. NFXF Data Request Committee review
   The NFXF Data Request Committee will review the Data Access Application for its methodological soundness, anticipated contribution to the community, and lack of overlap with other completed, ongoing, or previously approved planned analyses using the same data. All requests will be reviewed within 4 weeks of submission.
4. Dataset delivered
   If approved, a raw dataset and accompanying documentation will be provided securely to the principal investigator. All data access, use, and storage information is provided in our Data Use Policy document.

The application is available below, and you’ll also need to read the Data Use Policy

The goal of the NFXF Data Repository is to advance scientific understanding and treatment research for Fragile X by leveraging previously collected data for new proof-of-concept drug treatments, outcome measure development, and other meaningful scientific work.

Given our status as the leading patient advocacy organization for Fragile X, and our experience with collecting data in partnership with the FXCRC clinics and the CDC for the FORWARD-MARCH registry and database, the NFXF is the best equipped to centralize all de-identified clinical trial data for future research. The NFXF can provide unbiased access to all reputable projects wanting to utilize these data, while at the same time ensuring that all data remains de-identified for maximal patient safety and confidentiality.

Patient data can live on beyond initial clinical trials, creating opportunities for patient samples and clinical data to aid in multiple future treatment discovery programs. Researchers can feel confident knowing that the NFXF Data Repository has quality data from individuals with Fragile X syndrome, and the Fragile X community will know and trust that their data is safely housed within the NFXF.

Through the NFXF Data Repository, the paradigm shifts from the current model — where clinical trial data stays locked up with the sponsor, with no future use for the data — to a new NFXF model — where data is set free to become part of a treatment development cycle.

Supporting All Stakeholders

FACT: Multiple clinical trials for treatments specific to Fragile X syndrome have failed, leaving the community searching for treatments to improve quality of life in FXS.

FACT: Clinical trials take an average of 11 years and $2.6 billion to bring a drug to market.

We want to leverage participants’ contributions in clinical trials and make the de-identified data widely available to the Fragile X research community to expedite treatment development. This is part of the NFXF’s effort to support all stakeholders — Fragile X professionals, families, and researchers — to be engaged in the process to promote better outcomes for families living with Fragile X

A Readily Available, Inclusive Repository

The NFXF Data Repository™ is an inclusive repository of all valuable data, including NFXF survey data and NFXF Biobank data. The Data Repository allows research data to be readily available to the broader community for future research.

• Leveraging patient data from previous trials will enable new trialists to complete their proof-of-concept work without having to recruit participants and collect samples.
• Leveraging survey data from prior NFXF work enables development of better outcome measures and serves as a jumping off point for collaborative conversations to better research in Fragile X.

This is important because we know that there are many important things that lead to advancing treatment research. Having data living on past its initial collection is best for families and cost effective for researchers. Both clinical and biological data are valuable, and the Data Repository captures the importance of both. We have multiple sources contributing data, including our NFXF Biobank pilot project, the STX209 Reconsent project (more on STX209 below), and data rollover from future trials. The paired clinical and biological de-identified data in the Data Repository will allow researchers to jumpstart projects in meaningful ways, including treatment development.

Biomarkers are a big piece of the researcher puzzle, and so are better outcome measures specific to Fragile X and better representation in research so that treatments are effective for any individual living with Fragile X. The NFXF Data Repository allows us to look at research from these angles, doing parallel work in impactful areas. Including qualitative data in the NFXF Data Repository helps us examine additional angles.

The STX209 trials were medication trials studying arbaclofen in Fragile X syndrome. Many families participated in these studies over multiple years. In the two Phase III trials, arbaclofen did not meet the primary outcome of improved social avoidance in FXS in either study. This resulted in the STX209 program shutting down. The data from these trials stayed with the sponsor organization for safe keeping.

Families and professionals expressed their disappointment in the STX209 program ending. There was hope that the data would be made available for future research and possibly to even help with future trials to study arbaclofen in FXS for a drug approval.

For more information on these trials:

• Effects of STX209 (Arbaclofen) on Neurobehavioral Function in Children and Adults with Fragile X Syndrome: A Randomized, Controlled, Phase 2 Trial
• Arbaclofen in Fragile X Syndrome: Results of Phase 3 Trials

Also see: What We’ve Done, What We’re Doing and What’s the Plan