Clinical trials: Primary Immune Deficiency Disorders
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Leukocyte-Adhesion Deficiency Syndrome
Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency
This study will investigate the safety and effectiveness of a modified stem cell transplant procedure for treating leukocyte adhesion deficiency (LAD).
- Trial name
- NCT00031005
- Testing
- modified stem cell transplant
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
X-linked Severe Combined Immunodeficiency
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
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
- NCT01175239
- Testing
- Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
Primary Immune Deficiency Disorders (PIDD)
Open Label, Phase III Study of NABI-IGIV 10% [Immune Globulin Intravenous(Human), 10%] In Subjects With Primary Immune Deficiency Disorders (PIDD)
The purpose of this study is to determine if NABI-IGIV (10%) [Immune Globulin Intravenous (Human), 10%] is safe and effective in preventing serious bacterial infections (SBIs) in the treatment of patients with primary immune…
- Trial name
- NCT00538915
- Testing
- Nabi-IGIV 10% [Immune Globulin Intravenous (Human). 10%]
- Where
- 17 sites in United States
- Volunteers
- 63 people
- Sponsor
- ADMA Biologics, Inc.
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
HIV
Leukapheresis Procedures to Obtain Plasma and Lymphocytes for Research Studies on Antiretroviral Naive HIV-1 Research Participants
There is evidence that early and aggressive treatment with antiretroviral drugs can prevent the loss of immune cell function that accompanies HIV infection.
- Trial name
- NCT00039689
- Where
- United States
- Volunteers
- 750 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Wiskott-Aldrich Syndrome
Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding.
- Trial name
- NCT02064933
- Where
- 43 sites in United States, Canada
- Volunteers
- 305 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
- Trial name
- KONFIDENT-KID (NCT06467084)
- Testing
- KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Chlamydia Infections, Gonorrhea
Extraordinarily Fun Training Project in Compulsory Secondary Education - Sexually Transmitted Infections
Project FEDE-ITS will improve the STI knowledge and its treatment, of adolescents in the 1st and 2nd year of compulsory secondary education in the intervention group compared to compared to the control group, and will modify the…
- Conditions
- Chlamydia Infections, Gonorrhea, Syphilis Infection, Human Immunodeficiency Virus (HIV)
- Trial name
- FEDE-ITS (NCT05727033)
- Testing
- Participatory teaching group
- Where
- 4 sites in Spain
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- Fundacio d'Investigacio en Atencio Primaria Jordi Gol i Gurina
Hereditary Angioedema, HAE
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in…
- Trial name
- APeX-J (NCT03873116)
- Testing
- BCX7353 capsules
- Where
- 10 sites in Japan
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals
Severe Combined Immunodeficiency Syndrome
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene.
- Trial name
- NCT00018018
- Testing
- CD34+ cells transduced with ADA retrovir
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Lymphoma, Leukemia
Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation
RATIONALE: Following stem cell transplantation, a major risk is graft-versus-host disease (GVHD). This occurs when donor immune cells that have been infused recognise the host's cells as 'foreign' and attack these cells.
- Conditions
- Lymphoma, Leukemia, Myeloma, Myelodysplastic Syndromes
- Trial name
- ToTem (NCT03836690)
- Testing
- CD62L- Tem
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University College, London
HIV Infections, Men
Improving HIV Testing, Linkage, and Retention in Care for Men Through U=U Messaging
This study will evaluate the impact of U=U messaging and counseling on gaps in the HIV care cascade for men, including testing uptake and ART initiation (Aim 1), achieving viral suppression and retention in care (Aim 2) in two…
- Conditions
- HIV Infections, Men, Treatment Adherence
- Trial name
- NCT05602376
- Testing
- U=U testing messaging scripts, U=U adherence messaging scripts
- Where
- 2 sites in South Africa
- Volunteers
- 3,642 people, healthy volunteers welcome
- Sponsor
- Desmond Tutu HIV Foundation
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Hereditary Angioedema
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab.
- Trial name
- SPRING (NCT04070326)
- Testing
- Lanadelumab
- Where
- 17 sites in United States, Canada and 3 more
- Volunteers
- 21 people
- Sponsor
- Shire
Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm
Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study
This phase Ib trial investigates the side effects of the combination of nivolumab and ipilimumab, and to see how well they work in treating patients with cancers that have come back (relapsed) or does not respond to treatment…
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Lynch Syndrome, Recurrent Lymphoma
- Trial name
- NCT04500548
- Testing
- Biospecimen Collection, Ipilimumab, Nivolumab
- Where
- 9 sites in United States, Canada
- Sponsor
- National Cancer Institute (NCI)
AIDS-related Non-Hodgkin Lymphoma, AIDS-related Plasmablastic Lymphoma
Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma
This pilot clinical trial studies gene therapy after frontline chemotherapy in treating patients with acquired immune deficiency syndrome (AIDS)-related non-Hodgkin lymphoma (NHL).
- Conditions
- AIDS-related Non-Hodgkin Lymphoma, AIDS-related Plasmablastic Lymphoma, AIDS-related Primary Effusion Lymphoma, HIV Infection
- Trial name
- NCT01961063
- Testing
- busulfan, lentivirus vector rHIV7-shI-TAR-CCR5RZ-transduced hematopoietic progenitor cells, pharmacological study
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- City of Hope Medical Center
Primary Immunodeficiency Disorders
Evaluation of Efficacy and Tolerability of Hizentra®
The purpose of this study is to measure the changes in the Treatment Satisfaction Questionnaire for Medication in the areas of effectiveness, side effects, and convenience of administration of each medication in Primary…
- Trial name
- NCT01354587
- Testing
- Hizentra
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of South Florida