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Virus Therapy

cohort a for Bubble Boy Disease

Phase 1 & 2
Recruiting
Led By Suk S De Ravin, M.D.
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA
Must be between 2 and 40 years of age and weigh greater than or equal to 10 kg
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

This trial aims to test a new treatment for X-linked severe combined immunodeficiency (XSCID), also known as SCID-X1. The treatment involves transferring a corrected gene into the patient's

Who is the study for?
This trial is for children and adults aged 2 to 40 with X-linked severe combined immunodeficiency (XSCID) who lack a matched sibling bone marrow donor, may have had an unsuccessful half-matched transplant, and show significant immune impairment. Participants must weigh at least 10 kg, be HIV negative, have documented B cell dysfunction or need for IVIG therapy, and be able to comply with the study's long-term follow-up.Check my eligibility
What is being tested?
The trial tests gene transfer using lentiviral vectors in patients' own blood stem cells to treat XSCID. The process involves collecting these cells from the patient, treating them with a corrective gene vector in culture, then returning them via vein after pre-treatment with low-dose busulfan chemotherapy and palifermin to reduce mucositis side effects.See study design
What are the potential side effects?
Potential side effects include mouth, stomach, and bowel inflammation due to busulfan (mucositis), which palifermin aims to prevent. There are also risks associated with gene therapy such as immune reactions or unintended genetic changes that could lead to complications like leukemia.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My DNA test shows a mutation in the common gamma chain gene.
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I am between 2 and 40 years old and weigh at least 10 kg.
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I have a history of severe B cell dysfunction or need IVIG due to low IgG levels.
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I do not have a sibling donor match for a transplant.
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My HLA type has been determined.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Early evidence for efficacy will be defined by appearance and expansion in the circulation of autologous transduced T-lymphocytes with functional gmama-c and improved laboratory measures of immune function in the interim evaluation of these para...
Secondary outcome measures
Therapeutic procedure

Trial Design

2Treatment groups
Experimental Treatment
Group I: cohort bExperimental Treatment3 Interventions
Patients 9 and Beyond
Group II: cohort aExperimental Treatment3 Interventions
First 8 Patients Treated
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120
Palifermin
2006
Completed Phase 3
~1200

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,270 Previous Clinical Trials
5,485,197 Total Patients Enrolled
Suk S De Ravin, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
4 Previous Clinical Trials
81 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
Journal of Clinical InvestigationJournal
Insertional Mutagenesis Combined with Acquired Somatic Mutations Causes Leukemogenesis Following Gene Therapy of SCID-X1 Patients
Howe, Steven J., Marc R. Mansour, Kerstin Schwarzwaelder, Cynthia Bartholomae, Michael Hubank, Helena Kempski, Martijn H. Brugman, et al.. 2008. “Insertional Mutagenesis Combined with Acquired Somatic Mutations Causes Leukemogenesis Following Gene Therapy of SCID-X1 Patients”. Journal of Clinical Investigation. American Society for Clinical Investigation. doi:10.1172/jci35798.
clinicaltrials.govStudy
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
2012. "Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01306019.
All > Paid Studies Maryland
~17 spots leftby Dec 2032
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