Clinical trials: Primary Immune Deficiency Disorders
SCID, X-Linked
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect.
- Trial name
- NCT03217617
- Testing
- Direct intravenous injection of ivlv-X1 lentiviral vector
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Primary Immunodeficiency Diseases
Evaluation of an IgG Deficiency Rapid Screening Test: A Performance Study With Primary Immunodeficiency (PID) Patients in Tunisia
To evaluate the usability and utility of the device, % agreement between the PID-RDT and the referent assay (serum/plasma), and % agreement between capillary blood and venous blood samples using the PID-RDT within confirmed PID…
- Trial name
- NCT05621876
- Testing
- IgG deficiency rapid screening test
- Where
- Tunisia
- Volunteers
- 50 people
- Sponsor
- PATH
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
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
Immunoproliferative Disorder
Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome
The primary goal of this Phase I/II study is to assess the immune response and safety of recombinant human CD40 ligand (rhuCD40L) in patients with X-linked hyper IgM syndrome (XHIM).
- Trial name
- NCT00001145
- Testing
- Bacteriophage, rhuCD40L, KLH
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immunodeficiencies, Immune Dysregulation Syndromes
Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies
This 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…
- Trial name
- NCT02990819
- Testing
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Philadelphia
Primary Immunodeficiency, PID
Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals…
- Trial name
- BOLT-BMT (NCT03330795)
- Testing
- CD3/CD19 neg allogeneic BMT
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Sunitinib Malate in Treating HIV-Positive Patients With Cancer Receiving Antiretroviral Therapy
This phase I trial studies the side effects and the best dose of sunitinib malate in treating human immunodeficiency virus (HIV)-positive patients with cancer receiving antiretroviral therapy.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT00890747
- Testing
- sunitinib malate, pharmacological study, laboratory biomarker analysis
- Where
- 11 sites in United States
- Volunteers
- 42 people
- Sponsor
- National Cancer Institute (NCI)
Primary Immunodeficiency Diseases (PID)
A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID)
The main aim of the study is to check how much TAK-771 stays in their blood over time, side effect from the study treatment or TAK-771, how much TAK-771 participants can receive without getting side effects from it, and if…
- Trial name
- NCT05150340
- Testing
- TAK-771
- Where
- 12 sites in Japan
- Volunteers
- 16 people
- Sponsor
- Takeda
Melanoma, Non-small Cell Lung Cancer
A Study of CDX-1140 (CD40) as Monotherapy or in Combination in Patients With Advanced Malignancies
This is a study to determine the maximum tolerated dose (MTD) for CDX-1140 (CD40 antibody), either alone or in combination with CDX-301 (FLT3L), pembrolizumab, or chemotherapy and to further evaluate its tolerability and efficacy…
- Conditions
- Melanoma, Non-small Cell Lung Cancer, Breast Cancer, Gastric Cancer
- Trial name
- NCT03329950
- Testing
- CDX-1140, CDX-301, pembrolizumab
- Where
- 11 sites in United States
- Volunteers
- 132 people
- Sponsor
- Celldex Therapeutics
Toxoplasmosis, Cerebral, HIV Infections
Primary Prophylaxis of Cerebral Toxoplasmosis in HIV-Infected Patients
To evaluate the effectiveness of pyrimethamine (given with leucovorin calcium versus placebo (an inactive substance) for the primary prophylaxis (prevention) of cerebral toxoplasmosis in HIV-infected patients.
- Trial name
- NCT00000643
- Testing
- Pyrimethamine, Leucovorin calcium
- Where
- 24 sites in United States
- Volunteers
- 150 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hiv
Pharmacokinetics and Efficacy of Multiple Dosing of LP-98 for Injection in HIV-infected Patients
A Randomized, Double-Blind, Parallel-Group, Exploratory Clinical Study to Evaluate the Safety, Pharmacodynamic Effects, and Pharmacokinetic Characteristics of Multiple Subcutaneous Injections of LP-98 in Treatment-Naive…
- Trial name
- NCT06560489
- Testing
- Subjects will be randomly assigned to receive 1.25 mg, 2.5 mg, 5 mg, or 10 mg of LP-98.
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Shanxi Kangbao Biological Product Co., Ltd.
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Hereditary Angioedema, HAE
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE).
- Trial name
- OPuS-2 (NCT02303626)
- Testing
- BCX4161
- Where
- 40 sites in United States, Belgium and 6 more
- Volunteers
- 110 people
- Sponsor
- BioCryst Pharmaceuticals
Hyper-IgE Syndrome, Job's Syndrome
Omalizumab to Treat Hyper-IgE (Job's) Syndrome
This study will evaluate the safety and effectiveness of a laboratory-made antibody called omalizumab in patients with Job's syndrome, or hyper-IgE syndrome (HIES).
- Trial name
- NCT00260702
- Testing
- Omalizumab (Xolair)
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Human Immunodeficiency Virus
Raltegravir (Isentress/MK-0518) and HIV-1 Infected CD4 Cells During Acute/Early HIV-1
This is an investigator-initiated, two-year, randomized, controlled, single-center, open-label, pilot study comparing 3-drug highly active antiretroviral therapy (HAART) to 3-drug HAART plus raltegravir for persons with acute and…
- Trial name
- UW PIC 330 (NCT00781287)
- Testing
- 3-drug anti-HIV therapy, Raltegravir
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Washington
Primary Immunodeficiency (PID)
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)
This study is a continuation of the study ZLB06_001CR with the objective of assessing efficacy, tolerability, safety of IgPro, as well as long-term health-related quality of life in patients with PID.
- Trial name
- NCT00751621
- Testing
- IgPro20
- Where
- 13 sites in France, Germany and 6 more
- Volunteers
- 40 people
- Sponsor
- CSL Behring
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 Immune Deficiency Disorder
Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).
- Trial name
- NCT01458171
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 23 people
- Sponsor
- CSL Behring
HIV Infections
Effectiveness of Adding Interleukin-2 to Anti-HIV Drugs in Patients Recently Infected With HIV
The purpose of this study is to see whether taking interleukin-2 (IL-2) and other anti-HIV drugs affects the course of HIV disease in patients with primary HIV infection (the time period that immediately follows infection with…
- Trial name
- NCT00006441
- Testing
- Lamivudine/Zidovudine, Nelfinavir mesylate, Aldesleukin
- Where
- 2 sites in United States
- Volunteers
- 398 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)