Your session is about to expire
← Back to Search
Gene Therapy
Gene Therapy for Severe Combined Immunodeficiency
Phase 1 & 2
Recruiting
Led By Morton Cowan, MD
Research Sponsored by University of California, San Francisco
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
≥2.0 months of age at initiation of busulfan conditioning
Have no prior exposure to high dose busulfan
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 years
Awards & highlights
Study Summary
This trial will investigate if a new gene transfer method is safe and effective in treating Artemis-deficient Severe Combined Immunodeficiency (ART-SCID).
Who is the study for?
This trial is for patients with Artemis-deficient Severe Combined Immunodeficiency (ART-SCID) who haven't been treated with high-dose busulfan, don't have a suitable sibling donor, and are over 2 months old. It's also open to those who didn’t respond well to a previous transplant if they meet certain conditions.Check my eligibility
What is being tested?
The study tests a gene therapy method where stem cells are modified with a normal DCLRE1C gene and infused back into the patient after low-dose busulfan conditioning. The goal is to see if this can safely rebuild the immune system in ART-SCID patients.See study design
What are the potential side effects?
Potential side effects include reactions related to busulfan such as nausea, vomiting, liver problems, or mouth sores; complications from general anesthesia; and risks associated with stem cell infusion like infections or graft failure.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am at least 2 months old starting the busulfan treatment.
Select...
I have never been treated with high dose busulfan.
Select...
I don't have a healthy sibling match for a bone marrow donation.
Select...
I have been diagnosed with ART-SCID.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 15 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~15 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Survival of patients with ART-SCID who receive self-inactivating (SIN) lentiviral vector (AProArt)-transduced CD34 cells through autologous stem cell transplant
Secondary outcome measures
Dose of AProArt transduced cells
Hematopoietic recovery in patients with ART-SCID who receive self-inactivating (SIN) lentiviral vector (AProArt)-transduced CD34 cells through autologous stem cell transplant.
Immunoglobulin levels to measure establishment of B cell immune function in patients who have received AProArt lentiviral vector-transduced autologous CD34 hematopoietic stem cell transplant after low dose busulfan conditioning
+5 moreOther outcome measures
Dose of AProArt transduced cells with a repeat infusion of gene-corrected cells
Effect of prophylactic sirolimus to reduce the occurrence of autoimmune hemolytic anemia following infusion of gene-corrected cells.
Effects of treatment with a repeat infusion of gene-corrected cells on survival for patients who do not develop adequate immunity
+16 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Gene therapy (AProArt)Experimental Treatment3 Interventions
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells. The CliniMACS® CD34 Reagent System sorter device will be used to select CD34 cells. Patients will be conditioned with low dose busulfan prior to transplant.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120
Find a Location
Who is running the clinical trial?
University of California, San FranciscoLead Sponsor
2,506 Previous Clinical Trials
15,238,530 Total Patients Enrolled
3 Trials studying Severe Combined Immunodeficiency
1,807 Patients Enrolled for Severe Combined Immunodeficiency
Morton Cowan, MDPrincipal Investigator - University of California, San Francisco
UCSF Medical Center at Parnassus
University Of Pennsylvania School Of Medicine (Medical School)
Moffitt Hosp University Of California (Residency)
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have tested positive for HIV using specific testing methods.I have a sibling who is a medical match for me (newly diagnosed patients only).I have had a liver condition called veno-occlusive disease.I have ART-SCID and my previous transplant did not work.My doctor expects I have less than 4 months to live due to my condition.I am at least 2 months old starting the busulfan treatment.I have never been treated with high dose busulfan.I cannot undergo general anesthesia or procedures to collect stem cells.My liver tests are more than three times the normal limit or my total bilirubin is over 1.50 mg/dl.I have been newly diagnosed with ART-SCID and have low CD3 levels.I don't have a healthy sibling match for a bone marrow donation.I have been diagnosed with ART-SCID.
Research Study Groups:
This trial has the following groups:- Group 1: Gene therapy (AProArt)
Awards:
This trial has 1 awards, including:- 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.
Is there still room for new participants in this experiment?
"That is correct. As of February 4th, 2022, this trial indicated on clinicaltrials.gov that it was actively recruiting participants. The study had been originally posted on May 31st, 2018 and was last updated on the date just mentioned. Currently, the study aims to enroll 25 patients from a single site."
Answered by AI
What other medical research studies have used the CliniMACS® CD34 Reagent System cell sorter device?
"As of now, there are 104 ongoing clinical trials studying the CliniMACS® CD34 Reagent System cell sorter device. The majority of these studies are in their third phase with 13 total trials. Most of the research is taking place in Philadelphia, Pennsylvania; however, there are 1,072 locations conducting some form of study for the CliniMACS® CD34 Reagent System cell sorter device."
Answered by AI
What is the size of the sample group for this clinical trial?
"That is correct. The information available on clinicaltrials.gov reveals that this study is still recruiting patients. This trial was first posted on May 31st, 2018 and the most recent update was February 4th, 2022. They are looking for 25 individuals to participate at 1 location."
Answered by AI
What blood-related disorders does the CliniMACS® CD34 Reagent System cell sorter device help alleviate?
"The CliniMACS® CD34 Reagent System cell sorter device is most often used in allogeneic hematopoietic stem cell transplantation and chronic myelogenous leukemia treatment. It can also be employed to help patients with conditions like thrombocythemia, essential, and polycythemia vera (pv) during allogeneic stem cell transplant conditioning regimens ."
Answered by AI
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger