Clinical trials: XLP
NLRC4-MAS, XIAP Deficiency
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, leucine-rich repeat and…
- Trial name
- NCT03113760
- Testing
- Tadekinig alfa, 0.9% sodium chloride
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- AB2 Bio Ltd.
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
- 20 people
- Sponsor
- Washington University School of Medicine
Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation
The main objective of this study is to generate diagnosis and therapeutic-decision tools through the identification of molecular causes of PIDs with autoimmunity/inflammation and the variability in disease outcome at the…
- Conditions
- Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia, Autoimmune Diseases, Autoimmune Anemia
- Trial name
- ATRACTion (NCT04902807)
- Testing
- Collection of samples
- Where
- France
- Volunteers
- 325 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)
A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2.
- Trial name
- NCT06309823
- Testing
- MAS825
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
Erythropoietic Protoporphyria, EPP
Oral Iron for Erythropoietic Protoporphyrias
In the medical literature there are conflicting reports on whether iron improves symptoms in patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve EPP symptoms.
- Conditions
- Erythropoietic Protoporphyria, EPP, X-linked Protoporphyria, XLP
- Trial name
- EPP (NCT02979249)
- Testing
- Oral Iron
- Where
- 6 sites in United States
- Volunteers
- 16 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Erythropoietic Protoporphyria, EPP
Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
The initial objective of this protocol is to assemble a well-documented group of patients with confirmed diagnoses of the erythropoietic protoporphyrias, including autosomal recessive Erythropoietic Protoporphyria (EPP) and…
- Conditions
- Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP
- Trial name
- NCT01688895
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
EPP, XLP
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and…
- Trial name
- NCT04402489
- Testing
- MT-7117 Low Dose, MT-7117 High Dose
- Where
- 31 sites in United States, Australia and 8 more
- Volunteers
- 184 people
- Sponsor
- Tanabe Pharma America, Inc.
Benign Lymphoproliferative Disorder
Study of Autoimmune Lymphoproliferative Syndrome (ALPS)
The purpose of the protocol is to allow for patients, and relatives of patients, who may have the newly described autoimmune lymphoproliferative syndrome, to be evaluated at the NIH Clinical Center.
- Trial name
- NCT00001350
- Where
- United States
- Volunteers
- 1,200 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
XIAP Deficiency, NLRC4-MAS
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency As Open Label Extension
This is an open-label extension study for patients previously enrolled in the AB2 Bio Ltd. ongoing Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953).
- Trial name
- NCT03512314
- Testing
- Tadekinig alfa
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 11 people
- Sponsor
- AB2 Bio Ltd.
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II.
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease
- Trial name
- NCT00006054
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
XIAP Deficiency
Quercetin in Patients With XIAP (X-linked Inhibitor of Apoptosis) Deficiency
The purpose of the study is to evaluate safety and tolerability of oral quercetin in reducing inflammation in male patients with XIAP deficiency.
- Trial name
- NCT07433621
- Testing
- Quercetin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma
T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts
Subjects are being asked to participate in this study because treatment of their disease requires them to receive a stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma, Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Trial name
- MOHEL (NCT00368355)
- Testing
- Ara-C, Cyclophosphamide, Campath-1H
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Baylor College of Medicine
Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma
CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene
Patients are being asked to participate in this study because they will be receiving a stem cell transplant as treatment for their disease.
- Conditions
- Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome, Chronic Myeloid Leukemia
- Trial name
- CASPALLO (NCT00710892)
- Testing
- Allodepleted T Cells
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Lymphoproliferative Syndrome, Autoimmune, Lymphoma
Retrospective Observational Study of Italian Cohort of Lymphoma Patients (RETRO-LYMPH)
observational, non-interventional, retrospective, single-center study focusing on patients with lymphoma / lymphoproliferative syndrome between January 1980 and September 2021
- Trial name
- RETRO-LYMPH (NCT06781437)
- Where
- Italy
- Volunteers
- 5,500 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Study of Bitopertin in Participants With EPP or XLP (APOLLO)
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older.
- Trial name
- APOLLO (NCT06910358)
- Testing
- DISC-1459
- Where
- 27 sites in United States, Australia and 10 more
- Volunteers
- 183 people
- Sponsor
- Disc Medicine, Inc
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
- Trial name
- INSPIRE (NCT06144840)
- Testing
- Dersimelagon
- Where
- 35 sites in United States, Australia and 9 more
- Volunteers
- 165 people
- Sponsor
- Tanabe Pharma America, Inc.