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Gene Therapy
Lenti-D for Adrenoleukodystrophy
Phase 3
Waitlist Available
Research Sponsored by bluebird bio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to month 24 (+or- 1 month) post-transplant
Awards & highlights
Study Summary
This trial will enroll males with CALD who are less than or equal to 17 years old. The trial will be open-label, meaning that both the participants and the study staff will know which participants are receiving the Lenti-D Drug Product. The trial will be conducted at multiple sites internationally. Approximate 35 participants will be infused with Lenti-D Drug Product.
Eligible Conditions
- Adrenoleukodystrophy (ALD)
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to month 24 (+or- 1 month) post-transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to month 24 (+or- 1 month) post-transplant
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Percentage of participants who are alive and have none of the 6 major functional disabilities (MFDs) at Month 24
Percentage of participants with neutrophil engraftment after drug product infusion
Secondary outcome measures
Change in total neurologic function Score (NFS) from baseline to protocol scheduled visits
Detectable vector copy number (VCN) in peripheral blood cells by Month 6
Duration of in-patient hospitalizations (post-neutrophil engraftment) by Month 24
+20 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Lenti-D Drug ProductExperimental Treatment1 Intervention
Participants received a single intravenous (IV) infusion of Lenti-D Drug Product at a dose of > or = 5.0*10^6 CD34+ cells/kilogram (kg) (autologous CD34+ cell-enriched population that contains cells transduced with lentiviral vector encoding ABCD1 cDNA for human adrenoleukodystrophy protein, suspended in a cryopreservative solution) following myeloablative conditioning with busulfan and fludarabine on Day 1.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Lenti-D
2019
Completed Phase 3
~40
Find a Location
Who is running the clinical trial?
bluebird bioLead Sponsor
20 Previous Clinical Trials
2,008 Total Patients Enrolled
4 Trials studying Adrenoleukodystrophy
275 Patients Enrolled for Adrenoleukodystrophy
Andrew Dietz, MDStudy Directorbluebird bio, Inc.
1 Previous Clinical Trials
64 Total Patients Enrolled
1 Trials studying Adrenoleukodystrophy
64 Patients Enrolled for Adrenoleukodystrophy
Himal Lal Thakar, MDStudy Directorbluebird bio, Inc.
4 Previous Clinical Trials
358 Total Patients Enrolled
2 Trials studying Adrenoleukodystrophy
184 Patients Enrolled for Adrenoleukodystrophy
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have previously received a transplant from another person or have undergone gene therapy treatment.You are allergic to protamine sulfate.You have a condition that prevents you from having an MRI scan, such as allergies to anesthesia or contrast agents.Someone in your close family has a known or suspected genetic condition that increases the risk of cancer.You are currently taking statins, Lorenzo's Oil, or following a special diet to lower certain levels in your body. You will need to stop taking these medications or following the diet if you want to participate in the study.You have any serious heart, blood, lung, or other health condition that would make it unsafe for you to participate in the study procedures.
Research Study Groups:
This trial has the following groups:- Group 1: Lenti-D Drug Product
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any available vacancies for participants in this experiment?
"Unfortunately, this specific clinical trial is not currently looking for patients. Although the last update to the postings was on March 22nd, 2022, it is possible that the study will begin searching for participants again in the future. As of right now, there are 14 other trials that are actively enrolling patients."
Answered by AI
Are there any dangers to using Lenti-D for treatment?
"Lenti-D has received a score of 3 for safety. This is due to the fact that it is a phase 3 trial, meaning there are multiple rounds of data supporting efficacy and safety."
Answered by AI
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