Primary T-cell Immunodeficiency DisordersNCT03188419
Breadth of Donor Options for People With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide
An observational study of Primary T-cell Immunodeficiency Disorders and Common Variable Immunodeficiency, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: People who have certain immune system diseases often need a procedure called allo HSCT. This is short for allogeneic hematopoietic stem cell transplant. This might cure people with these diseases. Many people who need allo HSCT need donors who are relatives with similar genes. Read more about this study
- Who is it for?
- Ages 4 to 85 years
- Conditions the study is for
- Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency
- How many people
- 161 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jun 2017to 20 Feb 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 85 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Retrospective chart review of patients who with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT)at the NIH Clinical Center.
- Data Analysis Only
Trial locations and contacts
1 site in 1 country.
United States1 site
National Cancer Institute (NCI)
Bethesda, Maryland, 20892
About this study
Official title: Retrospective Study of the Breadth of Donor Options for Patients With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide
Background:
People who have certain immune system diseases often need a procedure called allo HSCT. This is short for allogeneic hematopoietic stem cell transplant. This might cure people with these diseases. Many people who need allo HSCT need donors who are relatives with similar genes. But the disease may also affect those in the donor pool. This may mean there are fewer options for people with inherited diseases. Researchers want to collect data on how transplant candidates and their donors are found.
Read more
Objective:
To find out how genetic diseases and the ways they are inherited affect the breadth of options for allo HSCT donors.
Eligibility:
Records from studies that have already been done. These will be for people ages 4 and older who were evaluated for allo HSCT or to be donors.
Design:
Participants already signed a consent form for their records to be shared.
Researchers will study the participant data.
Data will be stored in an electronic system. Researchers will use passwords to protect the data.
Read the detailed description
This protocol is a retrospective review of donor search results for patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT).
The study will involve collecting information related to the donor search for transplant candidates and their prospective donors, using records in CRIS, Crimson, the HLA lab, and records in the transplant coordinator office (such as shipping logs of HLA typing kits). The study will not involve the use of specimens or participant contact.
The participants whose records will be reviewed will be those who were evaluated for allo HSCT or donation on an NIH primary immunodeficiency transplant protocol at a time when haplo donors were eligible (March 30, 2012 to present for GATA2, January 1, 2015 for CGD, October 6, 2015 to present for 16-C-0003, and May 21, 2014 to present for DOCK8).
The Principal Investigators on the included protocols have granted permission to conduct this study and have verified that none of the original protocols or informed consent documents preclude such a review of clinical data.
- Keywords
- Prospective Donors, Immunodeficiency
- Medical subject headings
- T cell immunodeficiency primary, Common Variable Immunodeficiency, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Retrospective chart review of patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2017
- Study start14 Jun 2017
- Primary completion29 Dec 2019
- Study completion20 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary T-cell Immunodeficiency DisordersStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 161 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Jennifer A Kanakry, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03188419
- Sponsor's protocol ID
- 999917104
- Other IDs
- 17-C-N104
- Status last verified
- Feb 2020
- First submitted
- 14 Jun 2017
- Last updated
- 21 Feb 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Hsieh MM, Kang EM, Fitzhugh CD, Link MB, Bolan CD, Kurlander R, Childs RW, Rodgers GP, Powell JD, Tisdale JF. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009 Dec 10;361(24):2309-17. doi: 10.1056/NEJMoa0904971.
PubMed 20007560Gragert L, Eapen M, Williams E, Freeman J, Spellman S, Baitty R, Hartzman R, Rizzo JD, Horowitz M, Confer D, Maiers M. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014 Jul 24;371(4):339-48. doi: 10.1056/NEJMsa1311707.
PubMed 25054717
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Study of the Breadth of Donor Options for Patients With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide
- ClinicalTrials.gov ID
- NCT03188419
- Acronym / sponsor ID
- 999917104
- Keywords
- Prospective Donors, Immunodeficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 161 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 14 Jun 2017 (actual)
- Primary completion
- 29 Dec 2019 (actual)
- Study completion
- 20 Feb 2020 (actual)
- First posted
- 15 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
People who have certain immune system diseases often need a procedure called allo HSCT. This is short for allogeneic hematopoietic stem cell transplant. This might cure people with these diseases. Many people who need allo HSCT need donors who are relatives with similar genes. But the disease may also affect those in the donor pool. This may mean there are fewer options for people with inherited diseases. Researchers want to collect data on how transplant candidates and their donors are found.
Objective:
To find out how genetic diseases and the ways they are inherited affect the breadth of options for allo HSCT donors.
Eligibility:
Records from studies that have already been done. These will be for people ages 4 and older who were evaluated for allo HSCT or to be donors.
Design:
Participants already signed a consent form for their records to be shared.
Researchers will study the participant data.
Data will be stored in an electronic system. Researchers will use passwords to protect the data.
Detailed description
This protocol is a retrospective review of donor search results for patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT).
The study will involve collecting information related to the donor search for transplant candidates and their prospective donors, using records in CRIS, Crimson, the HLA lab, and records in the transplant coordinator office (such as shipping logs of HLA typing kits). The study will not involve the use of specimens or participant contact.
The participants whose records will be reviewed will be those who were evaluated for allo HSCT or donation on an NIH primary immunodeficiency transplant protocol at a time when haplo donors were eligible (March 30, 2012 to present for GATA2, January 1, 2015 for CGD, October 6, 2015 to present for 16-C-0003, and May 21, 2014 to present for DOCK8).
The Principal Investigators on the included protocols have granted permission to conduct this study and have verified that none of the original protocols or informed consent documents preclude such a review of clinical data.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Retrospective chart review of patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT) |
Who may qualify
- Ages
- 4 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Retrospective chart review of patients who with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT)at the NIH Clinical Center.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
Time frame: 1 year
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2020.
- Background: PubMed 20007560Hsieh MM, Kang EM, Fitzhugh CD, Link MB, Bolan CD, Kurlander R, Childs RW, Rodgers GP, Powell JD, Tisdale JF. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009 Dec 10;361(24):2309-17. doi: 10.1056/NEJMoa0904971.
- Background: PubMed 25054717Gragert L, Eapen M, Williams E, Freeman J, Spellman S, Baitty R, Hartzman R, Rizzo JD, Horowitz M, Confer D, Maiers M. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014 Jul 24;371(4):339-48. doi: 10.1056/NEJMsa1311707.
Related trials for Primary T-cell Immunodeficiency Disorders
RecruitingPhase 2
Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency
Not yet recruitingEarly Phase 1
25BT056: Basket Study of Viral Cytotoxic T Lymphocytes
EBV Viremia, CMV Viremia
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Active, not recruiting
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Active, not recruitingPhase 2
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer A Kanakry, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary T-cell Immunodeficiency Disorders and Common Variable Immunodeficiency, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03188419, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2020; AzTrials last refreshed its copy on 7 Nov 2026.