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Chemotherapy

Stem Cell Transplant for Job Syndrome

Q: Does the age limit for this experiment extend to people under 20 years old?

This clinical trial is looking for participants who are at least 2 years old, and no more than 60.

Q: Are there any limitations on how many people can join this clinical trial?

That is accurate. According to the information available on clinicaltrials.gov, this study which was created on 10/5/2010, is still looking for participants. In total, they need 90 people from 2 different locations.

Q: To whom does this research opportunity extend?

This study is looking for 90 participants with job syndrome between the ages of 2 and 60. The most important criteria are as follows: applicants must be aged 5-35 years, weigh more than 12 kilograms, have a clinical history of one or more episodes of life-threatening or severely disfiguring infection with opportunistic organisms, including severe recurrent cutaneous and sinopulmonary infections with bacterial or fungal infection, or viral infections with herpes simplex, herpes zoster, Molluscum contagiosum, or human papilloma virus., Left ventricular ejection fraction > 40%, preferably by 2-D echo.

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post transplant

Awards & highlights

Study 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.Check my eligibility

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.See study design

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 below

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I have a donor who is a perfect or near-perfect match, or a half-matched related donor.

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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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year post transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Feasibility

Secondary outcome measures

Safety

Toxicity

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

0 / 8Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor

13,663 Previous Clinical Trials

40,925,815 Total Patients Enrolled

1 Trials studying Dock 8 Deficiency

280 Patients Enrolled for Dock 8 Deficiency

Nirali N Shah, M.D.Principal InvestigatorNational Cancer Institute (NCI)

13 Previous Clinical Trials

2,439 Total Patients Enrolled

Corina E Gonzalez, M.D.Principal InvestigatorNational Cancer Institute (NCI)

Media Library

Busulfan (Busulfex) (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT01176006 — Phase 2

Dock 8 Deficiency Research Study Groups: Group A, Group B, Group C, Group D, Group E

Dock 8 Deficiency Clinical Trial 2023: Busulfan (Busulfex) Highlights & Side Effects. Trial Name: NCT01176006 — Phase 2

Busulfan (Busulfex) (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01176006 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the age limit for this experiment extend to people under 20 years old?

"This clinical trial is looking for participants who are at least 2 years old, and no more than 60."

Answered by AI

Are there any limitations on how many people can join this clinical trial?

"That is accurate. According to the information available on clinicaltrials.gov, this study which was created on 10/5/2010, is still looking for participants. In total, they need 90 people from 2 different locations."

Answered by AI

To whom does this research opportunity extend?

"This study is looking for 90 participants with job syndrome between the ages of 2 and 60. The most important criteria are as follows: applicants must be aged 5-35 years, weigh more than 12 kilograms, have a clinical history of one or more episodes of life-threatening or severely disfiguring infection with opportunistic organisms, including severe recurrent cutaneous and sinopulmonary infections with bacterial or fungal infection, or viral infections with herpes simplex, herpes zoster, Molluscum contagiosum, or human papilloma virus., Left ventricular ejection fraction > 40%, preferably by 2-D echo."

Answered by AI

Are there any open slots for this clinical trial?

"That is accurate. The clinicaltrials.gov website says that this trial, which was first advertised on October 5th 2010, is still recruiting patients. They need 90 individuals from 2 hospitals to take part in the study."

Answered by AI

Does Group B's involvement in this research mark the first time they have been studied?

"964 clinical trials are ongoing for Group B with 168 of them in Phase 3. Although a large concentration of these studies is based in Philadelphia, Pennsylvania, there are 28845 locations running studies for Group B."

Answered by AI

Has Group B's drug been given the green light by the FDA?

"Group B's safety was deemed to be a 2. In clinical trials, a score of 2 means that there is evidence backing the intervention's safety but not its efficacy."

Answered by AI

What type of ailment does Group B typically manage?

"Group B can be used to treat patients with multiple sclerosis, leukemia, myelocytic, acute, and allogeneic hematopoietic stem cell transplant, chronic myelogenous leukemia."

Answered by AI

Recent research and studies

TransplantationJournal

Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency

Freeman, Alexandra F., Nada Yazigi, Nirali N. Shah, David E. Kleiner, Mark Parta, Prescott Atkinson, Theo Heller, et al.. 2019. “Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency”. Transplantation. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/tp.0000000000002649.

clinicaltrials.govStudy

Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

2010. "Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01176006.

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