Clinical trials: Primary Immune Deficiency Disorders
Severe Combined Immunodeficiency
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells…
- Trial name
- NCT00028236
- Testing
- Gene-Transduced Autologous CD34+ Stem Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Care Appointment Keeping
Intervention to Retain HIV-positive Patients in Medical Care
PROJECT OVERVIEW This research is a collaboration between the Centers for Disease Control and Prevention (CDC), the Health Resources and Services Administration (HRSA), and six university-affiliated HIV clinics in the United…
- Trial name
- Phase II (NCT01537367)
- Testing
- Enhanced contact plus behavioral skills, Enhanced contact only, Standard of Care
- Where
- 6 sites in United States
- Volunteers
- 1,838 people
- Sponsor
- Centers for Disease Control and Prevention
Ataxia Telangiectasia (AT)
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Ataxia Telangiectasia (AT) is an autosomal recessive inherited condition caused by mutations in the ATM gene1. Patients suffer from neuro-degenerative problems, usually commencing in the second year of life, and affecting…
- Trial name
- NCT00656409
- Testing
- Conjugated pneumococcal vaccine (Prevenar)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institute of Child Health
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
Hereditary Angioedema (HAE)
EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
- Trial name
- NCT01826916
- Testing
- DX-88 (ecallantide)
- Volunteers
- 77 people
- Sponsor
- Shire
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Hereditary Angioedema (HAE)
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in…
- Trial name
- NCT01095510
- Testing
- CINRYZE
- Where
- 11 sites in United States, Germany and 1 more
- Volunteers
- 9 people
- Sponsor
- Shire
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
Primary IgA Nephropathy
Integrative Medicine of IgA Nephropathy
The present study was designed to identify the efficacy and safety of Integrative Medicine by joint oral steroid medicine on liver-kidney yin deficiency, severe IgA nephropathy.
- Trial name
- NCT02712697
- Testing
- WM (Shentong Granules), Hormone (prednisone)
- Where
- China
- Volunteers
- 140 people
- Sponsor
- Shanghai University of Traditional Chinese Medicine
Hereditary Angioedema
A Study of STAR-0215 in Healthy Adult Participants
This is a first-in-human, single-center, randomized, double-blind, placebo-controlled, single ascending dose study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of a single…
- Trial name
- NCT05477160
- Testing
- STAR-0215 (SC), STAR-0215 (IV)
- Where
- United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Astria Therapeutics, Inc.
Angioedema
Study of Heparin Prophylaxis of Hereditary Angioedema Exacerbations
OBJECTIVES: I. Determine the safety and efficacy of inhaled and subcutaneously administered heparin in the treatment of hereditary angioedema.
- Trial name
- NCT00004694
- Testing
- heparin
- Volunteers
- 24 people
- Sponsor
- University of Iowa
Primary Immunodeficiency Diseases
A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam.
- Trial name
- NCT01012323
- Testing
- NewGam
- Where
- 7 sites in United States
- Volunteers
- 51 people
- Sponsor
- Octapharma
Primary Immunodeficiency Disease
Clinical and Laboratory Online Patient- and Research Database for Primary Immunodeficiencies in Switzerland
A Swiss national, multi-centre, online patient and research database will be created, using the existing ESID database server system.
- Trial name
- NCT01150240
- Where
- 6 sites in Switzerland
- Volunteers
- 800 people
- Sponsor
- University of Zurich
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Primary Immunodeficiency
Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra) in Subjects With Primary Immunodeficiency (PID)
This multicenter, open-label, parallel-arm, non-randomized study is designed to evaluate safety and tolerability of higher infusion parameters of IgPro20 in subjects with primary immunodeficiency (PID).
- Trial name
- NCT03033745
- Testing
- IgPro20
- Where
- 12 sites in United States, Canada
- Volunteers
- 49 people
- Sponsor
- CSL Behring
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Cannabinoid Hyperemesis Syndrome
Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department
Patients diagnosed with cannabinoid hyperemesis syndrome that are safe for discharge from the emergency department will be sent an outpatient prescription for haloperidol to be used as needed at home.
- Trial name
- NCT07726069
- Testing
- Haloperidol
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
The main aim of this study is to compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab.
- Trial name
- NCT05469789
- Testing
- No Intervention
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Takeda
Human Immunodeficiency Virus, Alcohol-Related Disorders
Project Khanya: Peer-Delivered Intervention to Improve HIV Medication Adherence and Substance Use in South Africa
The purpose of this study is to evaluate a stepped care behavioral intervention for HIV medication adherence and substance use ("Khanya") integrated into an HIV primary care setting in South Africa.
- Conditions
- Human Immunodeficiency Virus, Alcohol-Related Disorders, Drug Use, Substance Use
- Trial name
- NCT05933226
- Testing
- Khanya
- Where
- South Africa
- Volunteers
- 160 people
- Sponsor
- University of Maryland, College Park
Primary Immunodeficiencies (PID), Secondary Immunodeficiencies (SID)
Assessing Patient Preference for Infusion Systems
This crossover study evaluates patient experiences and preferences between mechanical and electrical infusion pumps for subcutaneous immunoglobulin (SCIg) therapy.
- Trial name
- NCT07189013
- Testing
- Mechanical infusion pump
- Where
- United Kingdom
- Volunteers
- 52 people
- Sponsor
- KORU Medical Systems, Inc.