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NCT01176006 · ClinicalTrials.gov registry record · Phase 2
Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency
A Phase 2 study of DOCK8 Deficiency, sponsored by National Cancer Institute (NCI).
- Active
- Registry status
- Phase 2
- Development phase
- 70
- Enrollment target
- 1
- Study location
NCT01176006 is a Phase 2 study of DOCK8 Deficiency that is active but no longer recruiting, run by National Cancer Institute (NCI). The registered enrollment target is 70 participants. The trial reports 1 study location across 1 state.
The verdict
NCT01176006, a Phase 2 study of DOCK8 Deficiency, is active but no longer recruiting, sponsored by National Cancer Institute (NCI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 70 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: -DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or certain types of cancers, including blood cancers. A stem cell transplant is a life-saving treatment for this condition. In this study we are evaluating the efficacy and safety of transplant from different donor sources for DOCK8 deficiency. The donors that we are using are matched siblings, matched unrelated donors, and half-matched donors, so called haploidentical related donors, such as mothers or fathers or half-matched siblings. Objectives: -To determine whether transplant of bone marrow cells from different types of donors corrects DOCK8 deficiency. Eligibility: * Donors: Healthy individuals between 2 and 60 years of age who are matched with a recipient. * Recipient: Individuals between 4 and 35 years of age who have confirmed DOCK8 deficiency, have suffered at least one life-threatening infections, or have had certain viral related cancers of cancer and have a stem cell donor. Design: * All participants will be screened with bloodwork, a physical examination and medical history. * DONORS: --Donors who have donate bone marrow cells or blood stem cells will have a sample of blood/bone marrow stored to be compared with the recipients sample after transplant. * RECIPIENTS: * Recipients receiving 10/10 matched related or unrelated donors will receive 4 days of chemotherapy with busulfan and fludarabine to suppress their immune system and prepare them for the transplant. Donors receiving 9/10 matched related or unrelated donors as well as haploidentical related donors will receive 5 days chemotherapy with cyclophosphamide, fludarabine, and busulfan. They will also receive one dose of radiation to suppress their immune system and prepare them for the transplant. * After the initial chemotherapy and radiation (if indicated), recipients will receive the donated stem cells as a single infusio
Conditions Studied
Interventions
- DRUG Busulfan (Busulfex)
- PROCEDURE Reduced-intensity hematopoietic stem cell
- DRUG Fludarabine(Fludara, Berlex Laboratories)
- DRUG Cyclophosphamide(CTX, Cytoxan)
- PROCEDURE Total Body Irradiation (TBI)
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2010-10-05 |
| Est. Completion | 2028-10-31 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01176006
The ClinicalTrials.gov registry entry for NCT01176006 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 70 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Cancer Institute (NCI), which has 3,257 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with DOCK8 Deficiency appearing as the primary indexed condition, and to 5 interventions - of which Busulfan (Busulfex) is the first listed.
NCT01176006 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT01176006 about?
NCT01176006 is a clinical study titled "Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency". Background: -DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or certain types of cancers, including blood cancers. A stem cell transplant is a life-saving treatment for this condition. In this study...
What is the current status of trial NCT01176006?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 70 participants. The study started on 2010-10-05. Estimated completion is 2028-10-31.
What conditions does trial NCT01176006 study?
This clinical trial studies the following conditions: DOCK8 Deficiency.
What interventions are being tested in trial NCT01176006?
The interventions under investigation include: Busulfan (Busulfex) (DRUG), Reduced-intensity hematopoietic stem cell (PROCEDURE), Fludarabine(Fludara, Berlex Laboratories) (DRUG), Cyclophosphamide(CTX, Cytoxan) (DRUG), Total Body Irradiation (TBI) (PROCEDURE).
Who is sponsoring clinical trial NCT01176006?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01176006 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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