Clinical trials: Primary Immune Deficiency
Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm
Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study
This phase Ib trial investigates the side effects of the combination of nivolumab and ipilimumab, and to see how well they work in treating patients with cancers that have come back (relapsed) or does not respond to treatment…
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Lynch Syndrome, Recurrent Lymphoma
- Trial name
- NCT04500548
- Testing
- Biospecimen Collection, Ipilimumab, Nivolumab
- Where
- 9 sites in United States, Canada
- Sponsor
- National Cancer Institute (NCI)
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
Primary Immunodeficiency Disease
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study.
- Trial name
- NCT04842643
- Testing
- IGSC 20% infusion
- Where
- 8 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Hereditary Angioedema
Assessment of the State of Health, Quality of Life and Expectations of Patients With Hereditary Angioedema
8 years after the establishment of the therapeutic education program and 14 years after the creation of the National Reference Center for Angioedemas (CREAK), it is necessary to make an inventory in 2021 on the disease…
- Trial name
- BESQAOH (NCT04888650)
- Testing
- questionnaire
- Where
- France
- Volunteers
- 314 people
- Sponsor
- University Hospital, Grenoble
Cytomegalovirus Infections, Primary Immune Deficiency Disorder
Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV)
CMV cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults (CAYA) with refractory cytomegalovirus (CMV) infection post…
- Trial name
- NCT03266640
- Testing
- viral specific cytotoxic t-lymphocytes
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
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
Primary Immunodeficiency Diseases (PID)
A Study to Evaluate the Long-term Safety of TAK-771 in Japanese Primary Immunodeficiency Disease (PID) Participants
The main aim of the study is to check side effect from the study treatment with TAK-771 in long term. Participants can have taken part in the previous study TAK-771-3004 (NCT05150340).
- Trial name
- NCT05513586
- Testing
- TAK-771
- Where
- 9 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Takeda
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
Campylobacter Infections
Recurrent Campylobacter Bacteraemia in Immunocompromised Patients
Some rare cases of recurrent Campylobacter bacteraemia (RCB) exist with relapses months to years after an effective treatment and a negativation of all bacterial samples.
- Trial name
- CABARET (NCT06432777)
- Testing
- Characteristics of patients and Campylobacter bacteraemia episodes
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
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 Immunodeficiency Disorders (PIDs)
Leniolisib for Immune Dysregulation in PIDs
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib.
- Trial name
- NCT06549114
- Testing
- Leniolisib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Pharming Technologies B.V.
Hereditary Angioedema
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
- Trial name
- KONFIDENT-KID (NCT06467084)
- Testing
- KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Ciliary Dyskinesia, Kartagener Syndrome
Characterizing the Upper Airway Manifestations in Primary Ciliary Dyskinesia and Primary Immunodeficiencies
Though common, morbidities related to upper airway disease in primary ciliary dyskinesia (PCD) and primary immunodeficiencies (PID) have not been fully characterized.
- Conditions
- Primary Ciliary Dyskinesia, Kartagener Syndrome, Primary Immune Deficiency
- Trial name
- NCT04919018
- Where
- 4 sites in United States, Canada
- Volunteers
- 159 people
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
HIV/AIDS and Infections, Drug Abuse
Preventing HIV/STI in Urban Adolescents Via an mHealth Primary Care Intervention
The study evaluates the preliminary efficacy of an innovative mobile-health (mHealth) intervention (hereon referred to as S4E) to improve human immunodeficiency virus and sexually transmitted infection testing and reduce HIV/STI…
- Conditions
- HIV/AIDS and Infections, Drug Abuse, Adolescent Behavior, Drug Usage
- Trial name
- NCT03368456
- Testing
- S4E, Usual Care
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Michigan
PIDs Linked to PI3K
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE…
- Trial name
- NCT06990529
- Testing
- Leniolisib
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 12 people
- Sponsor
- Pharming Technologies B.V.
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency disorders. II. Explore the mutations within each syndrome to better understand the genetics of these disorders. III.
- Conditions
- X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
- Trial name
- NCT00004341
- Where
- United States
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)