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New treatment option for Chronic Granulomatous Disease
Official title Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
ClinicalTrials.gov ID: NCT05463133
What this study is testing
What is Sirolimus?
Sirolimus is an investigational medicine, being studied as a potential treatment for chronic granulomatous disease.
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) affects the immune system. People with CGD are more likely to get infections.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 65
You may be able to join if
- In order to be eligible to participate in this study, an individual must meet all the following criteria:
- Must have the ability to comprehend and a willingness to sign the informed consent. For pediatric patients, must have a parent/guardian who can sign...
- Must have confirmed diagnosis of CGD.
- Must have sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing...
- Ages 4 years-65 years.
You likely can't join if
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Ejection fraction of less than 30% by echocardiography.
- Forced expiratory volume (FEV1%) of less than 35% and/or an adjusted diffusing capacity of lung of carbon monoxide (adj DLCO) of less than 30%.
- Transaminases \>5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
- Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT treatment unlikely, and/or to make regulatorily and...
- Major anticipated illness or organ failure incompatible with survival from allogeneic peripheral blood stem cell (AlloPBSC) transplant.
See the full eligibility criteria
- In order to be eligible to participate in this study, an individual must meet all the following criteria:
- Must have the ability to comprehend and a willingness to sign the informed consent. For pediatric patients, must have a parent/guardian who can sign consent if the donor is a minor; assent will be obtained from minors...
- Must have confirmed diagnosis of CGD.
- Must have sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation/CGD-related autoimmunity OR a CGD-related infection while on prophylaxis) OR...
- Ages 4 years-65 years.
- HLA-matched family donor graft or an HLA-matched unrelated PBSC graft (10/10 or 9/10 mismatch) available.
- Must be human immunodeficiency virus (HIV) negative.
- When discharged from the hospital the participant must be able to stay within 1 hour s travel of the NIH for the first 3 months after transplantation.
- Must have a family member or other designated care provider to assist with care during the post-transplant period when the patient is in the outpatient setting.
- Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance with NIH 200 'NIH Durable Power of Attorney for Health Care Decision Making.'
- Females of child-bearing potential must agree to consistently use one form of contraception from 1 month prior to study entry and for at least 1 year post transplant. Male participants must agree to consistently use...
- Contraceptive pills or patch, Norplant [Registered], Depo-Provera [Registered], or other FDA-approved contraceptive method.
- Male partner has previously undergone a vasectomy.
- Male participants will be advised to consistently use contraception throughout study participation and for 3 months post-transplant.
- Stated willingness to comply with all study procedures and is available for protocol visits for the duration of the study when possible.
- Patients who have a CRP of greater than 100 but otherwise meet will be enrolled on the high-risk arm.
- CRP will be assessed no more than 7 weeks and no less than 6 weeks prior to anticipated transplant to determine on which arm the patient will be treated.
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Ejection fraction of less than 30% by echocardiography.
- Forced expiratory volume (FEV1%) of less than 35% and/or an adjusted diffusing capacity of lung of carbon monoxide (adj DLCO) of less than 30%.
- Transaminases \>5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
- Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT treatment unlikely, and/or to make regulatorily and legally effective informed consent impossible.
- Major anticipated illness or organ failure incompatible with survival from allogeneic peripheral blood stem cell (AlloPBSC) transplant.
- Pregnant or lactating.
- Uncontrolled seizure disorder per principal investigator (PI) discretion.
- Individuals older than 65 years are excluded. It is known from standard transplantation that these individuals have a higher risk of morbidity and mortality related to transplantation. Given the investigational nature...
- Active TB infection.
- Any condition or circumstance that the PI feels would create difficulty in maintaining compliance with the requirements of this protocol.
- Individuals who are not willing to submit their information as part of the alemtuzumab (Campath [Registered]) Distribution Program application or participants whom the Distribution Program committee has determined are...
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 all sexes, 4 years to 65 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.