To Evaluate the Effects of EryDex in Patients With A-T (NEAT)

April 11, 2024 updated by: Erydel

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex in Subjects With Ataxia Telangiectasia (NEAT)

This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangectasia (A-T).

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient.

In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution.

Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.

Study Type

Interventional

Enrollment (Estimated)

106

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • UZ Leuven
        • Contact:
          • Isabelle Meyts, MD
  • Denmark
      • Copenhagen, Denmark, 2100
        • Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
        • Contact:
          • Malene Landbo Børresen, MD
  • Germany
      • Frankfurt, Germany, 60590
        • University Hospital Frankfurt, Pediatric and Adolescent Clinic
        • Contact:
          • Jordis Trischler, MD
  • Italy
      • Brescia, Italy, 25123
        • Spedali Civili di Brescia, Pediatric immunology department
        • Contact:
          • Raffaele Badolato, MD
      • Rom, Italy, 00161
        • Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
        • Contact:
          • Francesco Pisani, MD
  • Norway
      • Oslo, Norway, 0372
        • Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
        • Contact:
          • Asbjørg Stray-Pedersen, MD
  • Poland
      • Warsaw, Poland, 04-736
        • Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
        • Contact:
          • Barbara Pietrucha, MD
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron, Department of pediatric neurology
        • Contact:
          • David Gómez Andrés, MD
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz, Department of pediatric neurology
        • Contact:
          • María del Mar García Romero, MD
  • United Kingdom
      • London, United Kingdom, WC1N 1DZ
        • Great Ormond Street Hospital for Children, Zayed Centre for Research
        • Contact:
          • Winnie Ip, MD
      • Nottingham, United Kingdom, NG7 2UH
        • Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
        • Contact:
          • Susan L. Perlman, MD
    • Maryland
      • Baltimore, Maryland, United States, 21289
        • The Johns Hopkins Hospital, Division of pediatric allergy and immunology
        • Contact:
          • Howard M. Lederman, MD
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital, Division of neurology
        • Contact:
          • Steve W. Wu, MD
    • Texas
      • Houston, Texas, United States, 77030
        • UT Health Houston, Department of pediatrics, division of child & adolescent neurology
        • Contact:
          • Sam Nicholas Russo, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Clinical diagnosis of A-T
  • In autonomous gait or is helped by periodic use of a support
  • Genetic confirmation of A-T
  • Body weight ≥15 kg

Exclusion Criteria:

  • Participation in another clinical study
  • Immune impairment
  • History of severe impairment of the immunological system
  • Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years
  • Severe or unstable pulmonary disease
  • Uncontrolled diabetes
  • Current chronic or acute significant renal and/or hepatic impairment
  • Any previous oral or parenteral steroid use within 6 weeks before Baseline. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted
  • A disability that may prevent the subject from completing all study requirements

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dexamethasone sodium phosphate
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
Drug: Dexamethasone sodium phosphate
Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion
Other Names:
  • DSP
Placebo Comparator: Placebo
IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
Other: Placebo
Placebo encapsulated in autologous erythrocytes and administered via IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)
Time Frame: Baseline to Visit 9 (approximately 6 months)
Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old)
Baseline to Visit 9 (approximately 6 months)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical Global Impression of Severity (CGI-S)
Time Frame: Baseline to Visit 9 (approximately 6 months)
Change in CGI-S from baseline to Visit 9
Baseline to Visit 9 (approximately 6 months)
Clinical Global Impression of Change (CGI-C)
Time Frame: Baseline to Visit 9 (approximately 6 months)
Clinical Global Impression of Change (CGI-C) at Visit 9
Baseline to Visit 9 (approximately 6 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Erydel

Collaborators

Biotrial

Investigators

  • Study Director: Dirk Thye, MD, Erydel

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2024

Primary Completion (Estimated)

August 1, 2025

Study Completion (Estimated)

September 1, 2025

Study Registration Dates

First Submitted

December 11, 2023

First Submitted That Met QC Criteria

December 21, 2023

First Posted (Actual)

January 5, 2024

Study Record Updates

Last Update Posted (Actual)

April 12, 2024

Last Update Submitted That Met QC Criteria

April 11, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IEDAT-04-2022

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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