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New treatment option for Chronic Granulomatous Disease (CGD)
Official title Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
ClinicalTrials.gov ID: NCT06325709
What this study is testing
What is Plerixafor?
Plerixafor is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for chronic granulomatous disease (cgd).
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
- Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly and are at greater risk of getting infections.
- 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 18 to 75, men only
You may be able to join if
- -\>= 18 years of age.
- Confirmed CYBB c.676 C\>T mutation.
- Male patients.
- Clinically stable and eligible to undergo apheresis and conditioning chemotherapy. -\>=5 x 10\^6 cryopreserved cells/kg body weight available for...
- History of at least one prior serious infection or inflammatory complication requiring hospitalization despite conventional therapy.
You likely can't join if
- Individuals meeting any of the following criteria will be excluded from study participation:
- Untreated, acute infection.
- Elevated anti-gp91 specific autoantibodies \>2 x ULN
- Elevated anti-gp91 specific T cells (\>10 fold)
- Anti-platelet antibody screening with \>1 anti-platelet antibody positive in the presence of an ongoing brain infection; OR \>1 anti-platelet...
- Known hypersensitivity to busulfan or any component of the product.
See the full eligibility criteria
- -\>= 18 years of age.
- Confirmed CYBB c.676 C\>T mutation.
- Male patients.
- Clinically stable and eligible to undergo apheresis and conditioning chemotherapy. -\>=5 x 10\^6 cryopreserved cells/kg body weight available for study product manufacturing.
- History of at least one prior serious infection or inflammatory complication requiring hospitalization despite conventional therapy.
- In the experience of a qualified clinical investigator, the patient has a poor prognosis.
- Able and willing to use a highly effective method of contraception, AND partner has communicated her willingness through subject to do same, if engaging in potentially reproductive sex from the signing of the informed...
- Hormonal contraception in continuously effective use by female partner.
- Male or female condom with spermicide as indicated.
- Diaphragm or cervical cap in consistent and effective pattern of use with a spermicide by female partner.
- Intrauterine device in-situ throughout above period by female partner.
- Individuals meeting any of the following criteria will be excluded from study participation:
- Untreated, acute infection.
- Elevated anti-gp91 specific autoantibodies \>2 x ULN
- Elevated anti-gp91 specific T cells (\>10 fold)
- Anti-platelet antibody screening with \>1 anti-platelet antibody positive in the presence of an ongoing brain infection; OR \>1 anti-platelet antibody positive and considered unsafe for study participation after...
- Known hypersensitivity to busulfan or any component of the product.
- Contraindications for administration of busulfan.
- Any current or pre-existing hematologic malignancy.
- Chronic infections that are considered unsafe for participation in the study by Infectious Disease Consultant.
- Cardiac abnormalities and neurological abnormalities that are deemed unsafe to participate in the study.
- Childhood malignancy (occurring before 18 years of age) in the patient or a first degree relative, or previously diagnosed known genotype of the participant conferring a predisposition to cancer (no DNA or other testing...
- Hematological parameters unsafe for apheresis or above Grade 2 Common Terminology Criteria for Adverse Events (CTCAE) criteria until improved.
- Hepatic dysfunction- alanine aminotransferase (ALT \>3.0 - 5.0 x upper limit of normal [ULN]), aspartate aminotransferase (AST \>3.0 - 5.0 x ULN), bilirubin (\>1.5 - 3.0 x ULN).
- Renal dysfunction-serum creatinine \>1.5 - 3.0 x ULN or creatinine clearance 59-30 mL/min/1.73 m\^2.
- Coagulation dysfunction- Prothrombin INR or Partial thromboplastin time \>2 x ULN (patients on controlled anticoagulation agents will not be excluded for therapeutic levels).
- Uncontrolled hypertension- Systolic BP 140-159 mm Hg or diastolic BP 90-99 mm Hg.
- Abnormal blood chemistries- Hyperkalemia (K \>5.5 - 6.0 mmol/L), Hypokalemia (\ 11.5 - 12.5 mg/dL), Hypocalcemia (corrected serum calcium \<8.0 -7.0 mg/dL) These values exclude false abnormalities secondary to hemolysis.
- Cytogenetic abnormalities evidenced on bone marrow aspirate.
- Pulmonary dysfunction FEV1\<25% predicted.
- Previous treatment with gene therapy or gene editing products.
- Previous receipt of non-HLA matched donor granulocyte transfusions.
- Any other condition that, in the opinion of the investigator, may unduly compromise the safety or compliance of the patient, or would make successful study completion highly unlikely.
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, 18 years to 75 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.