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A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

NCT04140539 · Status: WITHDRAWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL

Last updated 2022-08-03

No results posted yet for this study

Summary

The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.

Conditions

  • Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

BIOLOGICAL

OTL-101

Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo using a lentiviral vector (LV) encoding the human adenosine deaminase (ADA) gene.

Sponsors & Collaborators

Principal Investigators

  • Orchard Therapeutics Clinical Trials · Orchard Therapeutics (Europe) Limited

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Days
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-15
Primary Completion
2021-08-30
Completion
2021-08-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04140539 on ClinicalTrials.gov