Clinical trials: Severe Immune Deficiency
Anal Dysplasia, Human Papillomavirus
The HPV-SAVE Study Team: HPV Screening and Vaccine Evaluation in Men Who Have Sex With Men
Human papillomavirus (HPV) is the most common sexually transmitted infection worldwide. Infection by certain high-risk oncogenic types of HPV (HR-HPV) is the major cause of several cancers in men, notably squamous cell carcinoma…
- Conditions
- Anal Dysplasia, Human Papillomavirus, Anal Cancer
- Trial name
- HPV-SAVE (NCT02503111)
- Testing
- The Hyfrecator ® 2000 Electrosurgical System, Observation Alone
- Where
- 4 sites in Canada
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- University Health Network, Toronto
Hypogammaglobulinemia, Bacterial Infections
A Study to Evaluate Efficacy, Safety, and PK of XEMBIFY®+Standard Medical Treatment (SMT) Compared to Placebo+SMT to Prevent Infections in Participants With HGG and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma, and Non-Hodgkin Lymphoma
The primary purpose of the study is to evaluate whether biweekly administered XEMBIFY® plus Standard Medical Treatment (SMT) over a one-year period will reduce the rate of major bacterial infections per participant per year in…
- Conditions
- Hypogammaglobulinemia, Bacterial Infections, B-cell Chronic Lymphocytic Leukemia, Multiple Myleoma
- Trial name
- EXCELL (NCT05645107)
- Testing
- Xembify
- Where
- 62 sites in United States, Bosnia and Herzegovina and 6 more
- Volunteers
- 386 people
- Sponsor
- Grifols Therapeutics LLC
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Pediatric Patients, Any Type of Severe Combined Immunodeficiency (SCID)
Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell…
- Conditions
- Pediatric Patients, Any Type of Severe Combined Immunodeficiency (SCID), Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Trial name
- HTLP Necker (NCT03879876)
- Testing
- Human T Lymphoid Progenitor (HTLP) injection
- Where
- France
- Volunteers
- 4 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency
Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of…
- Trial name
- ARTEGENE (NCT05071222)
- Testing
- ARTEGENE drug product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Anal Neoplasm
VGX-3100 Delivered Intramuscularly (IM) Followed by Electroporation (EP) for the Treatment of HPV-16 and/or HPV-18 Related Anal or Anal/Peri-Anal, High Grade Squamous Intraepithelial Lesion (HSIL) in Individuals Seronegative for Human Immunodeficiency Virus (HIV)-1/2
This is a phase 2, open-label efficacy study of VGX-3100 administered by intramuscular (IM) injection followed by electroporation (EP) in adult men and women who are human immunodeficiency virus (HIV) negative with histologically…
- Trial name
- NCT03499795
- Testing
- VGX-3100, CELLECTRA™ 5PSP
- Where
- 3 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Inovio Pharmaceuticals
Severe Combined Immunodeficiency
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)
Infants with severe combined immunodeficiency (SCID) have a profound decrease in number and function of immune cells, and therefore remain highly vulnerable to infection. If not corrected this often leads to death.
- Trial name
- NCT03597594
- Testing
- Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Adult B Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia
Ibrutinib in Treating Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma in Patients With HIV Infection
This phase I trial studies the side effects and best dose of ibrutinib in treating B-cell non-Hodgkin lymphoma that has returned or does not respond to treatment in patients with human immunodeficiency virus (HIV) infection.
- Conditions
- Adult B Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia, Cutaneous B-Cell Non-Hodgkin Lymphoma, Extranodal Marginal Zone Lymphoma of Mucosa-Associated Lymphoid Tissue
- Trial name
- NCT02109224
- Testing
- Ibrutinib, Laboratory Biomarker Analysis, Pharmacological Study
- Where
- 7 sites in United States
- Volunteers
- 72 people
- Sponsor
- National Cancer Institute (NCI)
Severe Combined Immunodeficiency, X-Linked
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity.
- Trial name
- NCT03601286
- Testing
- Lentiviral vector transduced CD34+ cells
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Primary Immune-Deficiency (PID) Common Variable Immune Deficiency (CVID)
Severe PID With Lymphoproliferation and Neutropenia
The purpose of this study is to analyse the phenotype in a sub-population of adults with severe primary immunodeficiency with lymphoproliferation and neutropenia and to decipher the possible pathways involved, especially under…
- Trial name
- DICEP (NCT03427593)
- Testing
- FACS analyses, Target Sequencing by NGS ( Next-generation sequencing), Whole Exome Sequencing
- Where
- France
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Marasmus, Kwashiorkor
Mothers Understand And Can do it - Mid Upper Arm Circumference Screening for Malnutrition Performed by Mothers
PRIMARY OBJECTIVE : To determine whether mothers, given minimal group training, are capable of using a MUAC (mid-upper arm circumference) bracelet to screen their children for malnutrition and categorise them into one of three…
- Conditions
- Marasmus, Kwashiorkor, Malnutrition; Degree, Moderate, Malnutrition
- Trial name
- MUAC (NCT01790815)
- Where
- Niger
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Alliance for International Medical Action
HIV Infections
A Phase I Study of Autologous, Activated CD8(+) Lymphocytes Expanded In Vitro and Infused With or Without Recombinant Interleukin-2 to Patients With AIDS or Severe ARC
1) To determine whether it is possible to remove and culture (increase in number and activate) in the laboratory, CD8(+) lymphocytes (white blood cells) from HIV-infected patients receiving zidovudine (AZT); 2) To determine the…
- Trial name
- NCT00000680
- Testing
- Zidovudine, Aldesleukin
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2), Coronavirus Disease 2019 (COVID-19)
A Study to Learn About the Medicine Called Nirmatrelvir Used in Combination With Ritonavir in People With Weakened Immune Systems or at Increased Risk for Poor Outcomes Who Are Hospitalized Due to Severe COVID-19
A Study to Learn About the Medicine Called Nirmatrelvir Used in Combination With Ritonavir in People with Weakened Immune Systems or at Increased Risk for Poor Outcomes who are Hospitalized Due to Severe COVID-19
- Conditions
- Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2), Coronavirus Disease 2019 (COVID-19), Immunocompromised, Hospitalization
- Trial name
- EPIC-HOS (NCT05545319)
- Testing
- Nirmatrelvir, Ritonavir
- Where
- 11 sites in United States, Bulgaria
- Sponsor
- Pfizer
Schizophrenia, HIV Infections
The Quality of Life of Patients With Severe Illnesses
Lately researchers and clinicians have emphasized the health related quality of life. Several reasons account for this trend. First of all, the advanced medical practices have contributed to the increased life expectancy.
- Trial name
- NCT00173329
- Where
- Taiwan
- Sponsor
- National Taiwan University Hospital
Adenosine Deaminase Deficiency
Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach
Adenosine deaminase 2 deficiency (DADA2), caused by autosomal recessive mutations in the ADA2 gene (OMIM: 607575), is an autoinflammatory disease characterized by early-onset inflammatory vasculopathy, stroke, and…
- Trial name
- DADA2 (NCT07882680)
- Testing
- bone marrow aspiration and biopsy
- Where
- Italy
- Volunteers
- 29 people
- Sponsor
- Istituto Giannina Gaslini
Human Papilloma Virus, Human Immunodeficiency Virus
Use of ACU-D1 in HPV Associated Vulvar and Perianal Lesions in People With HIV
The goal of this study is to test the maximum tolerated dose of ACU-D1 in HIV-positive people with HPV-associated vulvar and perianal lesions.
- Conditions
- Human Papilloma Virus, Human Immunodeficiency Virus, Anal Intraepithelial Neoplasia, High-Grade Squamous Intraepithelial Lesions
- Trial name
- NCT06233331
- Testing
- Dose Level 1 ACU-D1 ointment, Dose Level 2 ACU-D1 ointment, Dose Level 3 ACU-D1 ointment
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Emory University
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the…
- Trial name
- NCT03645460
- Testing
- Direct intravenous injection of ivlv-ADA lentiviral vector
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Severe Combined Immunodeficiency, Malignancy, Hematologic
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA requires for all participants in gene transfer protocols and assures that adequate followup can be maintained for a wide variety of…
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm
- Trial name
- NCT00695279
- Testing
- Venipuncture
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- St. Jude Children's Research Hospital
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
Severe Combined Immunodeficiency Disease, X-linked
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system.
- Trial name
- LVXSCID-ND (NCT01512888)
- Testing
- CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
- Where
- 3 sites in United States
- Volunteers
- 28 people
- Sponsor
- St. Jude Children's Research Hospital