Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Description

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

Conditions

Glycogen Storage Disease Type Ia

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Study Overview

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Study Details

Study overview

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

GSDIa Disease Monitoring Program

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Condition
Glycogen Storage Disease Type Ia
Intervention / Treatment

-

Contacts and Locations

Orange

Children's Hospital of Orange County, Orange, California, United States, 92868

Denver

Children's Hospital Colorado, Denver, Colorado, United States, 80045

Hartford

University of Connecticut Health Center, Hartford, Connecticut, United States, 06106

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

New York

Icahn School of Medicine at Mount Sinai, New York, New York, United States, 10029

Durham

Duke University Medical Center, Durham, North Carolina, United States, 27710

Cleveland

The Cleveland Clinic Foundation, Cleveland, Ohio, United States, 44195

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Houston

University of Texas Health Science Center at Houston, Houston, Texas, United States, 77030

Salt Lake City

Primary Children's Hospital, Salt Lake City, Utah, United States, 84108

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Patient who had:
  • * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
  • * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
  • * Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.
  • * Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ultragenyx Pharmaceutical Inc,

Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceuticals Inc.

Study Record Dates

2036-12