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New treatment option for Common Variable Immunodeficiency (CVID)
Official title Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
ClinicalTrials.gov ID: NCT07284641
What this study is testing
What is Hematopoietic stem cell transplant (HSCT)?
Hematopoietic stem cell transplant (HSCT) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for common variable immunodeficiency (cvid).
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 research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD).
- 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 5 to 40
You may be able to join if
- Patient, parent, or legal guardian must have given written informed consent. For pediatric people who are developmentally able, assent or affirmation...
- Male or female, 5 through 40 years old, inclusive, at the time of informed consent.
- Patients must have evidence of common variable immunodeficiency (CVID) or other autoimmune manifestation of a primary immune regulatory disorder...
- Common variable immunodeficiency (CVID)
- Combined Immunodeficiency (CID)
You likely can't join if
- Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product
- Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing...
- Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the...
See the full eligibility criteria
- Patient, parent, or legal guardian must have given written informed consent. For pediatric people who are developmentally able, assent or affirmation will be obtained.
- Male or female, 5 through 40 years old, inclusive, at the time of informed consent.
- Patients must have evidence of common variable immunodeficiency (CVID) or other autoimmune manifestation of a primary immune regulatory disorder (PIRD). Genetic screening is required by a targeting gene panel to...
- Common variable immunodeficiency (CVID)
- Combined Immunodeficiency (CID)
- Immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX syndrome), IPEX like syndromes
- Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome
- Chronic Granulomatous Disease (CGD)
- Signal Transducer and Activator of Transcription (STAT 1) Gain of Function (STAT1 GOF)
- Signal Transducer and Activator of Transcription (STAT 3) Gain of Function (STAT3 GOF)
- Hypomorphic Recombination-Activating Genes (RAG) 1 and RAG 2
- CD40 or CD40L deficiency
- Mendelian Susceptibility to Mycobacterial Disease
- GATA-binding factor 2 (GATA2) Associated Immunodeficiency
- Mouth and Genital Ulcers with Inflamed Cartilage Syndrome (MAGIC)
- Must have previously failed, due to lack of response or intolerance, mycophenolate mofetil and a B cell-depleting antibody, such as Rituximab
- Glomerular Filtration Rate (GFR) ≥50 mL/min/1.73 m2
- Aspartate Aminotransferase (AST) ≤4x upper limit of normal
- Alanine Aminotransferase (ALT) ≤4x upper limit of normal
- Direct bilirubin ≤ 2.5 mg/dL
- Human Immunodeficiency Virus (HIV) negative by serology and PCR
- Human T-cell Lymphotropic Virus (HTLV) negative by serology
- Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%
- Forced Vital Capacity (FVC) and Forced Expiratory Volume in 1 second (FEV1) ≥40% predicted for age
- Peripheral Capillary Oxygen Saturation (SpO2) of \>92% at rest on room air
- people must be a minimum of 8 weeks post-solid organ transplant prior to start of conditioning, if applicable
- Negative pregnancy test for females \>10 years old or who have reached menarche, unless surgically sterilized.
- All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 12 months after stem cell transplant or for as long as they are taking any medication...
- Subject and/or parent guardian informed of the potential risks of infertility following stem cell transplant and advised to discuss sperm banking or oocyte harvesting.
- Transplant endorsement from clinical immunologist
- Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product
- Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing, etc.
- Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the...
The study team makes the final eligibility decision.
Where it's taking place
- Pittsburgh, Pennsylvania, 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 all sexes, 5 years to 40 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Pittsburgh, Pennsylvania, 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.