Clinical trials: Primary Immune Deficiency Disorders
Primary Immunodeficiency (PID)
Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or Pumps
The purpose of this study is to compare satisfaction (LQI questionnaire, factor I: treatment interference) in PID patients receiving subcutaneous injections of Gammanorm® 165 mg/mL according to the delivery device.
- Trial name
- NCT02180763
- Testing
- Gammanorm
- Where
- 8 sites in France
- Volunteers
- 31 people
- Sponsor
- Octapharma
X-linked Severe Combined Immunodeficiency
Gene Therapy for X-linked Severe Combined Immunodeficiency
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys.
- Trial name
- SCID2 (NCT01410019)
- Testing
- Gene transfer
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Organic, Including Symptomatic, Mental Disorders, Substance Use
Exploring the Comorbidity Between Mental Disorders and General Medical Conditions
Mental disorders have been shown to be associated with a number of general medical conditions (also referred to as somatic or physical conditions).
- Conditions
- Organic, Including Symptomatic, Mental Disorders, Substance Use, Schizophrenia, Mood [Affective] Disorders
- Trial name
- COMO-GMC (NCT03847753)
- Testing
- Organic Disorders, Substance Use Disorders, Schizophrenia Disorders
- Volunteers
- 5,940,299 people
- Sponsor
- University of Aarhus
Primary Immunodeficiency Diseases (PID)
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins
Primary immunodeficiency disease (or PIDD) is a group of conditions in which the immune system does not work properly. Some people with PIDD do not make enough antibodies.
- Trial name
- NCT07794735
- Testing
- TAK-664
- Volunteers
- 9 people
- Sponsor
- Takeda
Nervous System Disease, Immune System Disease
Detection and Characterization of Neurologic Manifestations of Inborn and Acquired Errors of Immunity
Background: Immune system and nervous system have significant interaction so that People with immunity diseases can have complications that affect the nervous system and people with some neurological disease may have defects in…
- Trial name
- NCT06169150
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency Diseases (PID)
Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects
The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary…
- Trial name
- NCT03277313
- Testing
- HYQVIA
- Where
- 19 sites in United States
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema (HAE)
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE).
- Trial name
- APeX-1 (NCT02870972)
- Testing
- BCX7353
- Where
- 24 sites in Australia, Austria and 9 more
- Volunteers
- 75 people
- Sponsor
- BioCryst Pharmaceuticals
Human Immunodeficiency Virus, Substance Use
Harm Reduction in HIV Primary Care for PLWH Who Use Drugs
People living with HIV (PLWH) who use drugs experience significant health disparities including lower rates of retention in HIV care and higher rates of unsuppressed viral load, resulting in secondary infections and increased…
- Conditions
- Human Immunodeficiency Virus, Substance Use, Stigma, Social
- Trial name
- NCT05404750
- Testing
- Harm Reduction
- Where
- 3 sites in United States
- Volunteers
- 641 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
HIV Infections
Development and Implementation of an HIV-testing Intervention for Primary Care in Belgium
An innovative HIV-testing strategy is developed based on mathematical modeling results on undiagnosed HIV and being implemented in primary care settings in Belgian's Flemish region.
- Trial name
- NCT04056156
- Testing
- HIV-screening advice, HIV-testing advice plus group-level training
- Where
- Belgium
- Volunteers
- 6,211 people
- Sponsor
- Institute of Tropical Medicine, Belgium
HIV Infections, Hypertension
ENHANCE-EvideNce Led Co-created HeAlth Systems interventioNs for MLTCs CarE
The goal of this study is to determine the effect of the ENHANCE intervention in improving clinical outcomes and evaluating the effects of the intervention on implementation processes and outcomes.
- Conditions
- HIV Infections, Hypertension, Diabetes Mellitus, Asthma
- Trial name
- ENHANCE (NCT06248190)
- Testing
- ENHANCE intervention (health systems)
- Where
- 32 sites in South Africa
- Volunteers
- 1,837 people
- Sponsor
- University of KwaZulu
Hereditary Angioedema
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
The objectives of the study are to: 1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema 2.
- Trial name
- NCT01095497
- Testing
- CINRYZE
- Where
- 7 sites in United States
- Volunteers
- 26 people
- Sponsor
- Shire
IgM and IgG in Blood
Immune Response Following COVID-19 Vaccination
Few trials have reported the safety and efficacy of the COVID-19 vaccines. However, these trials were mostly focused on post-vaccination adverse events and short-term antibody detection with none monitoring the presence of…
- Trial name
- IFVAC (NCT04736524)
- Testing
- Rapid antibody test
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Texas Cardiac Arrhythmia Research Foundation
Ataxia Telangiectasia
Baclofen Treatment of Ataxia Telangiectasia
This research is being done to find out if Baclofen, a medicine that is often used for the treatment of abnormal stiffness, might also be useful to treat some of the neurologic problems caused by ataxia telangiectasia (A-T).
- Trial name
- NCT00640003
- Testing
- Baclofen
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Johns Hopkins University
Primary Immune Deficiency Disorder
Octagam 5% Versus Comparator Post Marketing Trial
Study Design: This is a 2 arm non-interventional trial that will compare the occurrence of adverse drug reactions between Octagam 5% and other marketed IVIG infusion treatments.
- Trial name
- NCT01859754
- Testing
- Octagam 5%, Other marketed IVIG product
- Where
- 27 sites in United States
- Volunteers
- 623 people
- Sponsor
- Octapharma
Sphingolipidoses, Enzyme Deficiency
SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)
This protocol aims to gather information about sphingosine phosphate lyase insufficiency syndrome (SPLIS), also known as NPHS14, and to create a SPLIS patient registry.
- Trial name
- SPLIS-OSPRI (NCT04885179)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Primary Immune Deficiency Diseases
Primitive Immunodeficiency, Intrauterine Devices and Menstrual Hygiene Products
Primary immune deficiency diseases (PIDDs) constitute a large group of genetic disorders of the immune system which associates to varying degrees an increased susceptibility to infections and immuno-pathological manifestations…
- Trial name
- FEMIDIP (NCT06519734)
- Testing
- Questionnaire
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Severe Combined Immunodeficiency, Malignancy, Hematologic
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA requires for all participants in gene transfer protocols and assures that adequate followup can be maintained for a wide variety of…
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm
- Trial name
- NCT00695279
- Testing
- Venipuncture
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- St. Jude Children's Research Hospital
Immunologic Deficiency Syndrome
A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency
This study will compare the blood level of Gamunex in patients. Patients will take it as an injection under the skin or in a vein. The study will compare how safe and tolerable the two methods are in the patients.
- Trial name
- NCT00389324
- Testing
- Immune Globulin Intravenous (Human)
- Where
- 8 sites in United States, Canada
- Volunteers
- 35 people
- Sponsor
- Grifols Therapeutics LLC
Leukocyte Adhesion Deficiency Syndrome
Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
This study will evaluate the safety and effectiveness of the drug, interferon gamma, in treating leukocyte adhesion deficiency type I (LAD I).
- Trial name
- NCT00001905
- Testing
- Interferon gamma
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)