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Chemotherapy
Stem Cell Transplant for Job Syndrome
Phase 2
Recruiting
Led By Nirali N Shah, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Available 10/10 or 9/10 HLA-matched related or unrelated donor or a haploidentical related donor.
Creatinine: Subjects: less than or equal to 2.0 mg/dl or creatinine clearance greater than or equal to 30 ml/min/1.73 m^2. Pediatric subjects (<18 years old): Creatinine less than or equal to 1.5 mg/dl or a creatinine clearance of greater than or equal to 30 mL/min/1.73 m^2.
Must not have
Sexually active individuals capable of becoming pregnant who are unable or unwilling to use effective form(s) of contraception during time enrolled on study and for 1 year post-transplant. Effective forms of contraception include one or more of the following: intrauterine device (IUD), hormonal (birth control pills, injections, or implants), tubal ligation/hysterectomy, partner s vasectomy, barrier methods, (condom, diaphragm, or cervical cap), or abstinence. Males on the protocol must use an effective form of contraception at study entry, and for one year post-transplant. The effects of transplant, the radiation, and the medications used after transplant may be harmful to a fetus.
No available 10/10 or 9/10 HLA-matched related or unrelated donor or haploidentical related donor.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post transplant
Awards & highlights
No Placebo-Only Group
Summary
This trial is evaluating the efficacy and safety of transplant from different donor sources for DOCK8 deficiency.
Who is the study for?
This trial is for individuals aged 4-35 with confirmed DOCK8 deficiency, who've had life-threatening infections or viral-related cancers and have a suitable stem cell donor. Donors must be healthy, matched to the recipient, and aged 2-60. Participants need functioning hearts (with specific ejection fraction criteria), adequate kidney function, normal liver tests, and must not be pregnant or breastfeeding.
What is being tested?
The study tests whether bone marrow cells from different donors can treat DOCK8 deficiency. Recipients will undergo chemotherapy with busulfan and fludarabine (or cyclophosphamide added for certain matches) plus radiation in some cases to prepare for transplant. Post-transplant care includes more chemo and drugs to prevent graft versus host disease.
What are the potential side effects?
Potential side effects include reactions to chemotherapy such as nausea, fatigue, hair loss; risks from radiation like skin irritation; complications from stem cell infusion; immune system suppression leading to infection risk; graft versus host disease where donor cells attack the patient's body.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have a donor who is a perfect or near-perfect match, or a half-matched related donor.
Select...
My kidney function, measured by creatinine levels, is within the required range.
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I am between 4 and 35 years old.
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I (or my caregiver) can communicate in English or Spanish.
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I have had severe infections due to weak immunity.
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I have specific genetic mutations in the DOCK8 gene.
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I am over 18 and had a transplant for DOCK8 deficiency.
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I weigh at least 12 kilograms.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am using effective birth control or abstaining from sex for 1 year post-transplant.
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I don't have a closely matched donor for a transplant.
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I am HIV positive.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year post transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post transplant
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Feasibility
Secondary study objectives
Safety
Toxicity
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
5Treatment groups
Experimental Treatment
Active Control
Group I: Group DExperimental Treatment1 Intervention
Family Interview (closed)Participation in research interview
Group II: Group CExperimental Treatment3 Interventions
Donor (closed)
Group III: Group AActive Control3 Interventions
10/10 HLA Matched Related or Unrelated Donor Transplant
Group IV: Group BActive Control5 Interventions
9/10 HLA Matched Related or Unrelated Donor Transplant
Group V: Group EActive Control1 Intervention
Patient and caregiver psychosocial and QOL assessments during HSCTParticipation in interview and questionnaires
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,928 Previous Clinical Trials
41,018,031 Total Patients Enrolled
1 Trials studying Dock 8 Deficiency
272 Patients Enrolled for Dock 8 Deficiency
Nirali N Shah, M.D.Principal InvestigatorNational Cancer Institute (NCI)
13 Previous Clinical Trials
2,449 Total Patients Enrolled
Corina E Gonzalez, M.D.Principal InvestigatorNational Cancer Institute (NCI)
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a donor who is a perfect or near-perfect match, or a half-matched related donor.My cancer is in remission, or I have a virus-related cancer.My child doesn't need extra oxygen and can breathe and play without getting too tired or short of breath.I have cancer in another part of my body not related to blood, except if it's virus-caused.I am using effective birth control or abstaining from sex for 1 year post-transplant.My cancer has spread to my brain, except if it's caused by a virus where the transplant might help.I don't have a closely matched donor for a transplant.I am 8 years or older and need a transplant for DOCK8 deficiency.I am a caregiver of a child aged 4-25 with DOCK8 deficiency undergoing transplantation.I and my caregiver can understand and complete surveys and interviews.My family member can donate if they carry one DOCK8 gene mutation and match my HLA type.My kidney function, measured by creatinine levels, is within the required range.You have a mental health condition that may affect your ability to follow the transplant instructions or understand the study information.I am between 4 and 35 years old.I have chronic active hepatitis B but may still be eligible for the trial after a risk-benefit discussion.I (or my caregiver) can communicate in English or Spanish.I have had severe infections due to weak immunity.I have specific genetic mutations in the DOCK8 gene.I am over 18 and had a transplant for DOCK8 deficiency.I can understand and agree to the study's terms by signing a consent form.I weigh at least 12 kilograms.I am HIV positive.
Research Study Groups:
This trial has the following groups:- Group 1: Group A
- Group 2: Group B
- Group 3: Group C
- Group 4: Group D
- Group 5: Group E
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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