Clinical trials: Primary Immune Deficiency
Primary Immunodeficiency Disease
Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID
This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)
- Trial name
- NCT02810444
- Testing
- IgG Next Generation (BT595)
- Where
- 19 sites in United States, Germany and 3 more
- Volunteers
- 81 people
- Sponsor
- Biotest
Hereditary Angioedema, HAE
Oral Berotralstat Expanded Access Program
This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- NCT04428632
- Testing
- Berotralstat
- Sponsor
- BioCryst 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)
Primary Immunodeficiency Diseases (PID)
Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens and treatment administration…
- Trial name
- NCT02593188
- Testing
- HYQVIA
- Where
- 30 sites in United States
- Volunteers
- 264 people
- Sponsor
- Baxalta now part of Shire
Wiskott-Aldrich Syndrome
Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
Wiskott-Aldrich syndrome (WAS) is a rare X-linked congenital immune-deficiency syndrome and hematopoietic stem cell transplantation (HSCT) has become a curative modality.
- Trial name
- NCT00885833
- Testing
- Fludarabine, Busulfan, Thymoglobulin
- Where
- South Korea
- Volunteers
- 5 people
- Sponsor
- The Korean Society of Pediatric Hematology Oncology
Opioid Use Disorder, HIV Infections
Buprenorphine Integration Research and Community Health
The goal of this clinical trial is to evaluate an intervention strategy in introducing screen/evaluate/treat (SET) procedures for HIV/ hepatitis C/ and Opioid Use Disorder in Primary Care Clinics in West Virginia.
- Conditions
- Opioid Use Disorder, HIV Infections, Hepatitis C
- Trial name
- BIRCH (NCT05668780)
- Testing
- NIATx Coaching
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Yale University
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
HIV/AIDS
Using Open Contest and Neuro-influence Experiment to Develop and Evaluate PrEP Promotion Messages for High Risk Men
The aim of this study is to examine the utility of neuroimaging technique to evaluate pre-exposure prophylaxis (PrEP) promotion messages for men who have sex with men (MSM) at risk of HIV in Baltimore.
- Trial name
- NCT04166851
- Testing
- PrEP campaign messages developed via open contest, PrEP campaign messages developed via social marketing approach
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
HIV Infection
The Effect of Raltegravir on HIV Decay During Primary and Chronic Infection
The purpose of this study is to measure the decay characteristics of HIV in the blood of patients after taking a combination of anti-HIV drugs, which includes a new class of anti-HIV drug, an integrase inhibitor.
- Trial name
- PINT (NCT00641641)
- Testing
- Tenofovir + emtricitabine + raltegravir.
- Where
- 4 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Kirby Institute
Severe Combined Immunodeficiency
Clinical Characteristics and Genetic Profiles of Severe Combined Immunodeficiency in China
Severe combined immunodeficiency (SCID), a rare primary immunodeficiency dieases (PID), is poorly characterized in mainland China.
- Trial name
- NCT02231983
- Testing
- gene sequencing
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shanghai Children's Medical Center
HIV Infection
TMC125-C223: TMC125 in HIV-1 Infected Subjects
The purpose of this study is to investigate the ability of TMC125, a Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), to lower the amount of virus in your blood when administered twice daily for 48 weeks.
- Trial name
- NCT00081978
- Testing
- TMC125
- Volunteers
- 211 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Funding Source--FDA OOPD. Orphan Product Grant Number--1R01FD004091-01A1 Context: Wiskott-Aldrich syndrome (WAS) is a fatal, devastating disease with ill-defined treatment modalities, which affects young boys.
- Trial name
- WAS (NCT00774358)
- Testing
- Interleukin-2
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Soma Jyonouchi
Hereditary Angioedema (HAE)
Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers
This is a single-dose study to evaluate the pharmacokinetics, safety, and tolerability of icatibant administered to adult Japanese subjects.
- Trial name
- NCT02045264
- Testing
- Icatibant (30 mg)
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Shire
Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with…
- Conditions
- Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation, Graft Failure
- Trial name
- NCT03019809
- Testing
- G-CSF for Conditioning before HSCT., Plerixafor for Conditioning before HSCT.
- Where
- Russia
- Volunteers
- 30 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Asthma, Atrial Fibrillation
The Living With a Long-Term Condition Study
Psychological distress (anxiety and depression) is common in and experienced differently by people living with long-term health conditions (LTCs).
- Conditions
- Asthma, Atrial Fibrillation, Cancer, Cerebrovascular Disorders
- Trial name
- LTC (NCT06072287)
- Testing
- Living with LTC Questionnaire
- Where
- United Kingdom
- Volunteers
- 600 people
- Sponsor
- King's College London
Refractory Viral Infections
Multivirus-specific Cytotoxic T Lymphocytes (mCTL)
PIDD represent an expanding group of genetic disorders that compromise immunity against bacteria, viruses, and fungi. The most severe forms of PIDD cause profound susceptibility to opportunistic infections due to impaired or…
- Trial name
- NCT02510404
- Testing
- mCTLs
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Catherine Bollard
Hereditary Angioedema (HAE)
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single subcutaneous (SC) administration of icatibant in…
- Trial name
- NCT01386658
- Testing
- icatibant
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Shire
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
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
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