Clinical trials: Primary Immune Deficiency Disorders
Hiv
Identifying Efficient Linkage Strategies for Men
HIV self-testing (HIVST) has been found to be a highly acceptable approach for men to learn of their HIV status and has resulted in increased testing uptake.
- Trial name
- IDEaL (NCT05137210)
- Testing
- Male Counseling and Facility Navigation, Home-Based ART Initiation and Facility Navigation, Stepped Intervention
- Where
- Malawi
- Volunteers
- 569 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
X-Linked Combined Immunodeficiency Diseases
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
The purpose of this study is to evaluate the safety and effectiveness of lentiviral gene transfer treatment at restoring immune function to participants with X-linked severe combined immunodeficiency (XSCID) who are 2 to 40 years…
- Trial name
- LVXSCID-OC (NCT03315078)
- Testing
- CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Blood Coagulation Disorders
Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles
Congenital bleeding disorders characterized by abnormal platelet granules include Gray Platelet syndrome (GPS; defective alpha-granules), Hermansky-Pudlak syndrome (HPS; defective delta-granules), and combined alpha delta-storage…
- Trial name
- NCT00086476
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
MDS, Anemia, Aplastic
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that…
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure
- Trial name
- NCT00692926
- Testing
- ALDHbr Umbilical Cord Blood Cells
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Joanne Kurtzberg, MD
Hereditary Angioedema (HAE)
A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy Subjects
The purpose of this study is to assess the safety and tolerability of the study drug (DX-2930) administered subcutaneously in healthy subjects. Subjects will be randomized to receive DX-2930 or placebo.
- Trial name
- NCT01923207
- Testing
- DX-2930
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Shire
Primary Antibody Deficiency Disorders
Exercise Intervention and Immunodeficiency
The purpose of this study is to assess if a 12-week exercise program is safe, practical, and acceptable for adults with primary antibody deficiencies.
- Trial name
- NCT07698197
- Testing
- App-based exercise intervention
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Yale University
HIV
HIV Clinic-Based Intervention to Improve ART Adherence and Prevent HIV Transmission
This research, co-sponsored by the Centers for Disease Control and Prevention and the National Institute of Mental health, is conducted at six HIV clinics in the U.S.
- Trial name
- APTcare (NCT02044484)
- Testing
- Multi-component intervention
- Where
- 6 sites in United States
- Volunteers
- 2,794 people
- Sponsor
- Centers for Disease Control and Prevention
Primary Immunodeficiency
Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies
Background: The immune system helps the body fight infections. Primary immunodeficiency disorders (PIDs) are diseases that make it easier for people to get sick. Many PIDs are inherited.
- Trial name
- NCT03610802
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 3,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immune Deficiency
Bronchial Inflammation in Patients With Severe Immune Deficiency Under Immunoglobulin Treatment
The purpose of the clinical trial is to gain a more substantial understanding of bronchial inflammation in patients with severe primary immundeficiency under immunoglobulin therapy.
- Trial name
- NCT01359384
- Where
- Germany
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Johann Wolfgang Goethe University Hospital
Hereditary Angioedema (HAE)
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single subcutaneous (SC) administration of icatibant in…
- Trial name
- NCT01386658
- Testing
- icatibant
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Shire
HIV, AIDS
Project RETAIN: Providing Integrated Care for HIV-Infected Crack Cocaine Users
This study will evaluate the efficacy of an integrated "Retention Clinic" in achieving virologic suppression among HIV-infected cocaine (including crack) users by using a two-group randomized, prospective trial.
- Conditions
- HIV, AIDS, Substance Abuse
- Trial name
- NCT01614106
- Testing
- Retention Clinic
- Where
- 2 sites in United States
- Volunteers
- 360 people, healthy volunteers welcome
- Sponsor
- Columbia University
Hereditary Angioedema (HAE)
Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
- Trial name
- NCT01059526
- Testing
- ecallantide
- Where
- 40 sites in United States
- Volunteers
- 81 people
- Sponsor
- Shire
Refractory Viral Infections
Multivirus-specific Cytotoxic T Lymphocytes (mCTL)
PIDD represent an expanding group of genetic disorders that compromise immunity against bacteria, viruses, and fungi. The most severe forms of PIDD cause profound susceptibility to opportunistic infections due to impaired or…
- Trial name
- NCT02510404
- Testing
- mCTLs
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Catherine Bollard
Primary Ciliary Dyskinesia, Primary Immune Deficiency
Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults
The investigators will utilize a systematic approach for the diagnostic evaluation of patients to identify characteristics which may distinguish between Primary Immunodeficiency (PID) disorders versus Primary Ciliary Dyskinesia…
- Conditions
- Primary Ciliary Dyskinesia, Primary Immune Deficiency, Kartagener Syndrome
- Trial name
- NCT04702243
- Testing
- Genetic Testing for PCD or PID, Unaffected Family Member Genetic Testing
- Where
- 8 sites in United States, Canada
- Volunteers
- 436 people
- Sponsor
- University of North Carolina, Chapel Hill
Primary or Secondary Immune Deficiency Including Immunosuppressive Therapy, Chemotherapy, HIV Infection
Performance of Clinical Metagenomics in Stool and Urine Samples for Unexplained Diseases Diagnostic and Emerging Diseases Surveillance in Immunocompromised Patients
The study is based on the hypothesis that the concomitant use of mNGS in non-invasive samples (stool, urine) could improve the rate of detected pathogens in immunodeficient patients compared with mNGS performed in an invasive…
- Trial name
- SENTINEL (NCT07603453)
- Testing
- Metagenomics
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hereditary Angioedema
A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161
The purpose of the study is to compare how the body takes up and then eliminates the study drug BCX4161 when it is taken orally as a hard gelatin capsule and as a soft gelatin capsule.
- Trial name
- NCT02125162
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Primary Immunodeficiency Disease
Pediatric Primary Immunodeficiency Disease (PID) in China
This study is a prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children.
- Trial name
- NCT03252548
- Volunteers
- 400 people
- Sponsor
- Beijing Children's Hospital
Primary Immune Deficiency
Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.
- Trial name
- NCT00719680
- Testing
- IgPro20
- Where
- 4 sites in United States
- Volunteers
- 21 people
- Sponsor
- CSL Behring
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
This 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…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals