Clinical trials: Primary Immune Deficiency Disorders
Immunological Disease, Autoimmune Diseases
Biologics And Clinical Immunology Cohort at Sinai
The study team plans to establish a bioregistry of patients receiving biologic therapy as part of their standard treatment at the Mount Sinai Therapeutic Infusion Center and affiliated practices.
- Conditions
- Immunological Disease, Autoimmune Diseases, Primary Immune Deficiency
- Trial name
- BACICS (NCT04784364)
- Where
- United States
- Volunteers
- 800 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Primary Immune Deficiency, Exercise Capacity
Evaluation of Adult Patients With Immunodeficiency Within the Scope of the ICF
Primary immunodeficiency is a clinically, immunologically, and genetically heterogeneous group of diseases that occur as a result of quantitative and/or qualitative deficiency of one or more cellular and molecular components…
- Conditions
- Primary Immune Deficiency, Exercise Capacity, Physical Activity, Quality of Life
- Trial name
- NCT06014463
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
HIV Infections
Anti-HIV Drugs for Treating Infants Who Acquired HIV Infection at Birth
The purpose of this study is to compare the effects of anti-HIV drug courses of different lengths in infants who became HIV infected at birth.
- Trial name
- NCT00102960
- Testing
- Abacavir sulfate, Didanosine, Efavirenz
- Volunteers
- 377 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immune Deficiency Disorder
A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin Products
This is a safety study to evaluate the risk of low blood pressure in subjects with Primary Immune Deficiency disorder (PID) treated with Bivigam™ or another commercial product under real world conditions.
- Trial name
- NCT03037359
- Testing
- Bivigam, Other
- Where
- 17 sites in United States
- Volunteers
- 200 people
- Sponsor
- ADMA Biologics, Inc.
Primary Immunodeficiency Disease
Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients
The purpose of this study was to assess efficacy and safety of Kedrion Immunoglobulin 10% (KIg10) in participants with Primary Immunodeficiency (PID).
- Trial name
- CARES10 (NCT03961009)
- Testing
- Kedrion IVIG 10%
- Where
- 11 sites in United States
- Volunteers
- 47 people
- Sponsor
- Kedrion S.p.A.
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Coronary Artery Calcification, HIV Infections
French CAC-HIV Cohort Study
Clinical study: Methods: observational transversal two-arm cohort study including adults living with HIV (PLHIV) and HIV negative subjects (HIV-) at intermediate cardiovascular risk.
- Trial name
- CAC-HIV (NCT05563116)
- Testing
- Computed tomography coronary artery calcium (CAC) score (part of routine workup)
- Volunteers
- 689 people
- Sponsor
- Franck Boccara
Primary Immunodeficiency Diseases (PID)
Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies
In the prospective part of the study, patients diagnosed with PID will receive replacement therapy with Cutaquig for at least 6 months and will be randomised with the help of physicians of the Russian PID Registry.
- Trial name
- NCT05986734
- Testing
- Cutaquig
- Where
- Russia
- Volunteers
- 100 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Primary Antibody Deficiencies
Early Detection of Primary Antibody Deficiencies in Primary Care Facilities by an Algorithm Driven Selection of Serologic Testing in Individuals at Risk.
Rationale: Primary antibody deficiencies (PAD) encompass a group of rare heterogeneous diseases. The clinical presentation may vary widely, including infectious and autoimmune symptoms and increased risk of malignancy.
- Trial name
- GP-PAD II (NCT05310604)
- Testing
- Blood withdrawal
- Where
- Netherlands
- Volunteers
- 104 people
- Sponsor
- UMC Utrecht
Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer)
An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
This is a Phase Ib/II clinical study. The Phase Ib dose-escalation study aims to evaluate and determine the recommended Phase II dose (RP2D) of TCC1727 in combination with benmelstobart /olaparib /topotecanfor patients with…
- Conditions
- Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer), Gastric (Stomach) Cancer, Endometrial Cancer
- Trial name
- NCT07371663
- Testing
- TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg
- Where
- 3 sites in China
- Volunteers
- 266 people
- Sponsor
- Beijing Tide Pharmaceutical Co., Ltd
Primary Immunodeficiency Diseases (PID)
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%).
- Trial name
- NCT04346108
- Testing
- Immune Globulin Intravenous (IGIV), Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
- Where
- 8 sites in Japan
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Primary Immune Deficiency
Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study)
The purpose of this study is to assess the long-term efficacy, tolerability, and safety of IgPro20 in subjects with primary immunodeficiency (PID) as an extension to the preceding follow-up study ZLB07_001CR (NCT01458171).
- Trial name
- NCT01461018
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Wiskott-Aldrich Syndrome
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HLA-immune system type) matched…
- Trial name
- NCT00160355
- Testing
- Hematopoietic stem cell transplantation, Miltenyi CliniMACS selection system, Fludarabine, Melphalan, Thiotepa
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Hereditary Angioedema (HAE)
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
- Trial name
- NCT05460325
- Testing
- Lanadelumab
- Where
- 4 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Human Immunodeficiency Virus
HIV Prevention in the Primary Care Setting
In the third decade of the HIV pandemic, what was once a uniformly and rapidly fatal disease has been transformed into a chronic illness by advances in the understanding of HIV pathogenesis and therapeutics.
- Trial name
- NCT00381524
- Testing
- Increase condom use
- Where
- United States
- Volunteers
- 234 people
- Sponsor
- University of Alabama at Birmingham
Wiskott-Aldrich Syndrome
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with…
- Trial name
- NCT04350164
- Testing
- Romiplostim
- Where
- Russia
- Volunteers
- 67 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Human Placental-Derived Stem Cell Transplantation
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease
- Trial name
- HPDSC (NCT01586455)
- Testing
- Human Placental Derived Stem Cell
- Where
- 3 sites in United States
- Volunteers
- 43 people
- Sponsor
- New York Medical College
Ataxia Telangiectasia, Leukemia
Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability.
- Conditions
- Ataxia Telangiectasia, Leukemia, Lymphoma
- Trial name
- NCT04037189
- Where
- Israel
- Volunteers
- 250 people
- Sponsor
- Rabin Medical Center