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11 Primary Immune Deficiency Trials
Power is an online platform that helps thousands of Primary Immune Deficiency patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Cytotoxic T-Lymphocytes for Adenovirus Infections
Columbus, OhioRelated donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with refractory ADV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant.
Funding Source: FDA OOPD
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:1 - 79
Sex:All
Key Eligibility Criteria
Disqualifiers:Acute GVHD, Poor Performance, HIV, Others
Must Not Be Taking:Steroids
20 Participants Needed
Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders
Pittsburgh, PennsylvaniaThe objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 55
Sex:All
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others
100 Participants Needed
Newnorm for Primary Immunodeficiency
Louisville, KentuckyThis trial is testing a new antibody treatment called Newnorm, which is given through an injection under the skin. It aims to help people with primary immunodeficiency diseases, whose immune systems are weak. The treatment provides extra antibodies to help their bodies fight infections.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:Acute Infection, Cancer, Liver Dysfunction, Others
Must Be Taking:Immunoglobulin Therapy
50 Participants Needed
gp91 Grans for Chronic Granulomatous Disease
Bethesda, MarylandBackground:
CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells.
Objective:
To test a procedure in which mRNA is added to a person s blood cells.
Eligibility:
Males aged 18-75 with CGD with a mutation in the gene that makes the protein gp91phox.
Design:
Participants will be screened with:
Medical history
Physical exam
Blood and urine tests
Swab to test for strep throat
Some screening tests will be repeated during the study.
Participants will be admitted to the NIH Clinical Center hospital for at least 7 days. They will have apheresis. For this, a medicine is injected under their skin to prepare their white blood cells for collection. An IV line is placed into an arm vein. Blood goes through the IV line into a machine that divides whole blood into red blood cells, plasma, and white blood cells. The white blood cells are removed, and the rest of the blood is returned to the participant through an IV line in their other arm. The next day, they will get their mRNA-corrected cells via IV. They will be monitored for 3 more days.
After discharge, participants will keep a symptom diary. They will be contacted weekly for one month, and then once a month. They will have a follow-up visit 3 months after the infusion.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 75
Sex:Male
Key Eligibility Criteria
Disqualifiers:Systemic Infections, Kidney Disease, Diabetes, Heart Failure, Others
Must Not Be Taking:High-dose Steroids
25 Participants Needed
Leniolisib for Primary Immunodeficiency
Bethesda, MarylandThis study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of leniolisib.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:12 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Lymphoma, Others
Must Not Be Taking:Immunosuppressants, Corticosteroids, Investigational Drugs, Others
12 Participants Needed
Stem Cell Transplant for Immune Deficiency Syndrome
Bethesda, MarylandBackground:
During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications.
Objective:
To see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them.
Eligibility:
People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors
Design:
Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow.
Participants will be screened with:
Medical history
Physical exam
Blood, urine, and heart tests
CT or PET scans
Before transplant, participants will have dental and eye exams. They will have a bone marrow biopsy. For this, a needle will be inserted through the skin into the pelvis to remove marrow.
Participants will be hospitalized before their transplant. They will have a central catheter put into a vein in their chest or neck. They will get medications through the catheter to prevent complications. Participants will get stem cells through the catheter. They will stay in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool samples. They may need blood transfusions. They may need more scans. They will take more medications.
Participants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the transplant. Then they will have visits once a year for about 5 years
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 69
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Brain Metastases, Uncontrolled Illness, Others
Must Not Be Taking:Investigational Agents
66 Participants Needed
Background:
Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another. Researchers think this treatment can provide a new, healthy immune system to correct T-cell problems in some people.
Objective:
To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with T-cell problems.
Eligibility:
Donors: Healthy people ages 4 and older
Recipients: People the same age with abnormal T-cell function causing health problems
Design:
All participants will be screened with:
* Medical history
* Physical exam
* Blood, heart, and urine tests
Donors will also have an electrocardiogram and chest x-ray. They may have veins tested or a pre-anesthesia test.
Recipients will also have lung tests.
Some participants will have scans and/or bone marrow collected by needle in the hip bones.
Donors will learn about medicines and activities to avoid and repeat some screening tests.
Some donors will stay in the hospital overnight and have bone marrow collected with anesthesia.
Other donors will get shots for several days to stimulate cells. They will have blood removed by plastic tube (IV) in an arm vein. A machine will remove stem cells and return the rest of the blood to the other arm.
Recipients will have:
* More bone marrow and a small fragment of bone removed
* Dental, diet, and social worker consultations
* Scans
* Chemotherapy and antibody therapy for 2 weeks
* Catheter inserted in a chest or neck vein to receive donor stem cells
* A hospital stay for several weeks with more medicines and procedures
* Multiple follow-up visits
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:4+
Sex:All
70 Participants Needed
Bone Marrow Transplant for Immune Deficiency
Bethesda, MarylandBackground:
Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.
Objective:
To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies.
Eligibility:
Donors: Healthy people ages 4 or older
Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant
Design:
Participants will be screened with medical history, physical exam, and blood tests.
Participants will have urine tests, EKG, and chest x-ray.
Donors will have:
Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone.
OR
Blood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm.
Possible vein assessment or pre-anesthesia evaluation
Recipients will have:
Lung test, heart tests, radiology scans, CT scans, and dental exam
Possible tissue biopsies or lumbar puncture
Bone marrow and a small piece of bone removed by needle in the hipbone.
Chemotherapy 1-2 weeks before transplant day
Donor stem cell donation through a catheter put into a vein in the chest or neck
Several-week hospital stay. They will take medications and may need blood transfusions and additional procedures.
After discharge, recipients will:
Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission.
Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Active Psychiatric Disorder, Others
Must Not Be Taking:Investigational Agents
254 Participants Needed
Gene Modified T-cells for Blood Disorders
Washington, District of ColumbiaThis study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:1 - 26
Sex:All
120 Participants Needed
This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 40
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Pregnancy, Others
27 Participants Needed
Cell Depletion using CliniMACS for Primary Immunodeficiencies
Philadelphia, PennsylvaniaThis is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with alpha/beta T and CD19+ depleted peripheral stem cell grafts from unrelated or partially matched related donors. There are two conditioning regimens depending upon patient diagnosis and age.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:< 25
Sex:All
60 Participants Needed
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Frequently Asked Questions
How much do Primary Immune Deficiency clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Primary Immune Deficiency clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Primary Immune Deficiency trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Primary Immune Deficiency is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Primary Immune Deficiency medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Primary Immune Deficiency clinical trials ?
Most recently, we added Leniolisib for Primary Immunodeficiency, gp91 Grans for Chronic Granulomatous Disease and Newnorm for Primary Immunodeficiency to the Power online platform.