Blood cell harvest could fuel future immune disease cures
NCT ID NCT00001405
First seen Jun 25, 2026 · Last updated Sep 17, 2026 · Updated 23 times
Summary
This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advance treatments, not to directly treat participants.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Blood stem cells and immune cells collected via apheresis or bone marrow harvest
- What this could lead to
- If successful, this could help create new gene therapies or cell treatments for people with inherited immune disorders.
- What could go wrong
- This is a collection study, not a treatment trial. The cells are used for lab research or other approved protocols, so direct benefits to participants are unlikely.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
About 850 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Feb 1994
- Lead sponsor
-
A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Patients with PID or other blood disorders Healthy adult individuals volunteering for research donation
- Ages
-
18 to 70 years
- Sex
-
Anyone
- Healthy volunteers
-
Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* ELIGIBILITY CRITERIA: Patients (Patients with a genetically defined PID or other blood disorder or clinical history consistent with PID or other blood disorder) 1. Patients will have a genetically defined PID or have a clinical history of recurrent infections or other problems suggestive of PID or other blood disorder, must be 18-70 years of age, 2. Some patients may have active bacterial or fungal infection at the time of study entry. 3. Preserved renal function (creatinine less than or equal to 2.5 mg/dL; less than or equal to 3+ proteinuria); preserved hepatic function (bilirubin less than or equal to 2.0 mg/dl); preserved hematologic function (WBC greater than or equal to1000/mm\^3;granulocytes greater than or equal to 500/mm\^3; platelets greater than or equal to 100,000; hematocrit greater than or equal to 25). Of note, patients with PID often have associated chronic thrombocytopenia. Patients with stable chronic thrombocytopenia will be eligible for collection, at the investigator s discretion, with the caveat that patients with platelet count \<40,000 the day prior to collection will be transfused with platelets on the morning of collection. Platelets may also be given to these patients following the collection if medically indicated.. 4. Patients of childbearing potential may be entered if using effective contraception and having a negative serum or urine pregnancy test within one week of beginning G-CSF administration. 5. Patients may remain on their regimen of prophylactic treatments as deemed necessary by the investigator. 6. Willingness to allow blood cell samples to be stored 7. Willingness to allow blood and/or bone marrow samples to be modified to iPS cells Healthy Adult Volunteers 1. Healthy adults aged 18-65 without active current infection or history of recurrent infection, 2. Weighs at least 50kg. 3. Normal renal function (creatinine less than or equal to 1.5 mg/dL; less than or equal to 1+ proteinuria); normal hepatic function (bilirubin less than or equal to 1.5 mg/dL); normal hematologic function (WBC greater than or equal to 2500/mm\^3; granulocytes greater than or equal to 1200/mm\^3; platelets greater than or equal to 120,000; hematocrit greater than or equal to 38). 4. Normal female volunteers of childbearing potential may be entered if using effective contraception and having a negative serum or urine pregnancy test within one week of beginning GCSF administration. 5. Willingness to allow blood cell samples to be stored 6. Willingness to allow blood and/or bone marrow samples to be modified to iPS cells For PBMCs and grans collections, adult subjects with known genetic mutations may participate as healthy volunteers for research purposes as long as the other criteria listed above are fulfilled. EXCLUSIONS: Patients 1. Patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm Hg from the stable patient s baseline measurement) or requiring mechanical respiratory assistance are excluded. 2. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test are excluded. 3. Must be negative by routine blood donor eligibility testing criteria including tests for syphilis (RPR) and TTV Donor Transplant Panel testing (list is modified periodically, but may include hepatitis B and C, HIV and HTLV, T. cruzi). This does not apply to leukapheresis patients, as these tests are not required by DTM. 1. XSCID patients do not make antibodies and false positives may occur because they receive periodic infusions of pooled donations of IVIG. We have observed positive anti-HBc testing in these patients. If this occurs, more specific DNA or antigen testing will be done and must be negative. 2. Patients with CGD and other patients with autoimmunity as part of their PID phenotype may have false positive antibody tests and if this occurs more specific DNA or antigen testing will be done and must be negative. 3. Autologous HSC Transplant patients - may be positive for Hepatitis B and C if the investigator deems it necessary to be collected and used as a safety back-up Healthy Volunteers 1. Active bacterial, fungal or viral infection as evidenced by history, physical exam (temperature \>38 degress C), or WBC \>9000 are excluded. 2. Females who are pregnant or lactating as determined by history and/or pregnancy test are excluded. 3. Must be negative by routine blood donor eligibility testing criteria, including tests for syphilis (RPR) and TTV Recipient Transplant Panel (list is modified periodically, but may include hepatitis B and C, HIV and HTLV, T. cruzi) This does not apply to leukaphersis patients, as these tests are not required by DTM policy. 4. Someone without peripheral venous access in arm veins adequate for apheresis (healthy volunteers only). 5. If in the opinion of the investigator participation in this study places the healthy adult volunteer at undue risk. Patients being considered for clinical scale bone marrow harvesting 1. Who are unable to lie prone during the bone marrow harvesting procedure. 2. Who are unable to tolerate general anesthesia during the bone marrow harvesting procedure.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Blood disorder are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- How does COVID-19 hit people born with weak immune systems?
- Can tiny tablets replace protein powder for kids with PKU?
- Could a stool sample replace a spinal tap? new study tests Non-Invasive infection detection
- New drug could help boys with duchenne walk and move longer
- Massive blood disorder registry aims to transform care
- Groundbreaking gene therapy aims to fix immune system in rare disease