Overview

The Critical Path to Therapeutics for the Ataxias (CPTA) is a public-private partnership with the mission to optimize clinical trials for inherited ataxias. Through facilitating a collaborative and consensus-driven community of patient advocates, researchers, and regulators, CPTA aims to work collaboratively to develop tools and complete analyses to accelerate the development of new therapies for inherited ataxias.

Please visit the CPTA overview to learn more.

Data contributed to CPTA is hosted on C-Path's Rare Disease Cures Accelerator - Data and Analytics Platform (RDCA-DAP), an FDA-funded initiative that provides a centralized and standardized infrastructure for data across rare and orphan diseases. Click here for more information.

Who we are

Officially launched in February 2021, CPTA is a public-private partnership with the mission to optimize clinical trials for inherited ataxias. CPTA is a collaborative effort between C-Path, the Ataxia Global Initiative, the National Ataxia Foundation, Ataxia UK, the Ataxia Charlevoix-Saguenay Foundation and key partners in the industry and academic research communities, including Biogen, Biohaven Pharmaceuticals, Ionis, PTC Therapeutics, Roche, Servier Pharmaceuticals and VICO Therapeutics.

Our mission

Inherited ataxias are a diverse collection of genetic disorders with unique genetic causes and a spectrum of clinical presentation, affecting many different body systems. Although the pipeline of new medical products for the ataxias is expanding, there remain large gaps in our understanding of these diverse disorders.

There is an urgent need for actionable tools to understand the natural history, progression, and variance in these diseases, as well as how to measure therapeutic effects in clinical trials.

Our mission is to bring together experts from across different fields of ataxia research, advocacy, and medical product development to create regulatory tools and strategies that will catalyze research in the ataxias. In doing so, we aim to increase the efficiency and effectiveness of medical product development for these disorders and deliver innovative treatments to patients in need.

Quick Start

New users: please sign up here before visiting Workspaces and FAIR. Platform access will be reviewed within 3-5 business days. More info here.

Use these Quick Start steps to explore the platform and available data. If you are interested in using CPTA data for research or study, please request a research workspace prior to submitting a Data Access Request in FAIR. A member of the C-Path team will address workspace requests within 3-5 business days.

1. Discover Data
  1. Go to FAIR Data ServicesFAIR logoData Services
  2. Review the existing datasets: Click 'Datasets' and select ‘All datasets’ in the dropdown.Datasets
  3. Click on an individual dataset to review its metadata.
2. Request Access
  1. When you have found a dataset of interest, request a workspace for data access.
  2. Once approved, you will be invited to join a workspace.
  3. To request data access, go to FAIR and click the Request Icon  in the menubar.
  4. Fill out the form and submit – once approved, you can transfer the dataset to your workspace.
3. Conduct Research
  1. Go to WorkspacesWorkspaces logoWorkspaces
  2. From the Workspaces home page, you can view your workspaces and invitations.
  3. Accept any pending invites and click to enter your workspace.
4. Contribute Datasets
Interested in contributing research, study, or clinical trials data to RDCA-DAP?

  1. Share your interest in contributing data to C-Path and provide supporting information.Connect
  2. C-Path will review and reach out with more information within 10 business days.

If you are a patient or patient advocate seeking a place for single patient data, or help on establishing data collection efforts, please reach out to NORD (US based) or EURORDIS (EU based) to identify available databases.

Getting Started

Using C-Path DAP

The Data and Analytics Platform includes FAIR Data Services and Workspaces.

  • FAIR includes integrated data from clinical trials, observational studies, real-world data, patient registries and other sources.
  • In Workspaces, advanced analytics capabilities allow efficient and effective interrogation of data to generate insights, analyses and solutions.

When using the C-Path DAP, please refer to the current list of supported browsers.

Workspaces Fundamentals

FAIR Standard
Using FAIR Data Services
Workspaces Advanced
Workspaces Advanced Learning

Useful Information

Data security

Data can be highly sensitive: it can be at risk for re-identification even when explicit personally identifiable information is removed. Find out more about the measures that we use to keep data safe.

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