Myelodysplastic Syndrome Clinical Trial

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Summary

This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study.

After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
Have received Lenti-D Drug Product in a parent clinical study
Able to comply with study requirements

Exclusion Criteria:

There are no exclusion criteria for this Study

Study is for people with:

Myelodysplastic Syndrome

Estimated Enrollment:

60

Study ID:

NCT02698579

Recruitment Status:

Enrolling by invitation

Sponsor:

bluebird bio

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There are 8 Locations for this study

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Mattel Children's Hospital-UCLA
Los Angeles California, 90095, United States
Lucile Packard Children's Hospital - Stanford
Palo Alto California, 94304, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston Massachusetts, 02115, United States
University of Minnesota
Minneapolis Minnesota, 55455, United States
Instituto Neurogenia
Caba , , Argentina
Women's and Children's Hospital
North Adelaide , , Australia
Hospital das Clínicas da Universidade de São Paulo
São Paulo , 05403, Brazil
Hôpital Bicêtre
Le Kremlin-Bicêtre Cedex, 94275, France
Universitätsklinikum Leipzig AöR
Leipzig , 04103, Germany
Prinses Maxima Center
Utrecht , 3584, Netherlands
Great Ormond Street Hospital
London , , United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Estimated Enrollment:

60

Study ID:

NCT02698579

Recruitment Status:

Enrolling by invitation

Sponsor:


bluebird bio

How clear is this clinincal trial information?

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