BridgeMD
New treatment option for X-linked Severe Combined Immunodeficiency (XSCID)
Official title Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
ClinicalTrials.gov ID: NCT01306019
What this study is testing
What is Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector?
Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for x-linked severe combined immunodeficiency (xscid).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral vector incorporating additional features to improve safety and performance. The study will treat 35 patients with XSCID who are between 2 and 50 years of age and who have clinically significant impairment of immunity.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 50, men only
You may be able to join if
- A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA
- No available HLA matched sibling donor as determined before enrollment. (HLA typing will be performed prior to enrollment)
- Must be between 2 and 50 years of age and weigh greater than or equal to 10 kg
- If previously transplanted, must be greater than or equal to 18 months post HSCT
- Expected survival of at least 120 days.
You likely can't join if
- Any current or pre-existing hematologic malignancy
- Documented HIV-1 infection
- Documented active Hepatitis B infection
- Childhood malignancy (occurring before 18 years of age) in the patient or a first degree relative, or previously diagnosed known genotype of the...
See the full eligibility criteria
- A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA
- No available HLA matched sibling donor as determined before enrollment. (HLA typing will be performed prior to enrollment)
- Must be between 2 and 50 years of age and weigh greater than or equal to 10 kg
- If previously transplanted, must be greater than or equal to 18 months post HSCT
- Expected survival of at least 120 days.
- Participants of reproductive potential must agree to consistently use highly effective contraception throughout study participation and for at least 2 years post-treatment. Acceptable forms of contraception are: --For...
- Documented to be negative for HIV infection by genome PCR
- The patient must be judged by the primary evaluating physician to have a suitable family and social situation consistent with ability to comply with protocol procedures and the long-term follow-up requirements.
- Medical lab data (historical) of severe B cell dysfunction (low or absent IgG levels, failed immune response to vaccines); OR demonstrated requirement for intravenous gamma globulin (IVIG) (significant drop over 3 to 6...
- Must be willing to have blood and tissue samples stored IN ADDITION, patients must satisfy the following Laboratory Criteria AND Clinical Criteria Laboratory Criteria: (greater than or equal to 1 must be present) I...
- Treatment (not prophylaxis) with systemic antibacterial, antifungal or antiviral antibiotics greater than or equal to 14 days OR
- Hospitalization of any duration for infection OR
- Isolation of a bacteria, fungus, or virus from biopsy, skin lesion, blood, nasal washing, bronchoscopy, cerebrospinal fluid or stool likely to be an etiologic agent of infection II. Chronic pulmonary disease as defined...
- Bronchiectasis by x-ray computerized tomography OR
- Pulmonary function test (PFT) evidence for restrictive or obstructive disease that is 60 percent of Predicted for Age OR
- Pulse oximetry 94 percent in room air (if patient is too young to comply with performance of PFTs). III. Gastrointestinal enteropathy:
- Diarrhea-watery stools greater than or equal to 3 times per day (of at least 3 months duration that is not a result of infection as defined in criterion above) OR
- Endoscopic evidence (gross and histologic) for enteropathy (endoscopy will only be performed if medically indicated) OR
- Other evidence of enteropathy or bacterial overgrowth syndrome: including malabsorption of fat-soluble vitamin(s), abnormal D-xylose absorption, abnormal hydrogen breath test, evidence of protein losing enteropathy (for...
- Any current or pre-existing hematologic malignancy
- Documented HIV-1 infection
- Documented active Hepatitis B infection
- Childhood malignancy (occurring before 18 years of age) in the patient or a first degree relative, or previously diagnosed known genotype of the subject conferring a predisposition to cancer (no DNA or other testing for...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.