Clinical trials: SCID
Adenosine Deaminase Severe Combined Immunodeficiency
Registry Study of Revcovi Treatment in Patients With ADA-SCID
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi.
- Trial name
- NCT03878069
- Testing
- elapegademase-lvlr
- Where
- 14 sites in United States
- Volunteers
- 32 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Bronchiectasis, Adenosine Deaminase Deficiency
Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study, bronchiectasis was detected on at least one computed tomography of the lungs…
- Conditions
- Bronchiectasis, Adenosine Deaminase Deficiency, Pulmonary Disease
- Trial name
- NCT05300347
- Testing
- adenosine deaminase enzyme deficiency test
- Where
- Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- TRPHARM
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
This trial is a prospective, non-randomized, open-label, multicentre single-arm phase I/II intervention trial in children up to 24 months of age with RAG1-deficient SCID and an indication for allogeneic hematopoietic stem cell…
- Trial name
- RAG1-SCID (NCT04797260)
- Testing
- Gene therapy
- Where
- 4 sites in Netherlands, Poland and 2 more
- Volunteers
- 10 people
- Sponsor
- Videja B.V.
Intracranial Aneurysm
Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device
The purpose of this study is to assess safety and effectiveness of the p64 Flow Modulation Device.
- Trial name
- NCT02600364
- Where
- 26 sites in Argentina, Belgium and 8 more
- Volunteers
- 450 people
- Sponsor
- Phenox GmbH
Severe Combined Immunodeficiency
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID). The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability…
- Trial name
- NCT02860559
- Testing
- TBX-1400
- Where
- 2 sites in Israel
- Volunteers
- 8 people
- Sponsor
- Taiga Biotechnologies, Inc.
Adenosine Deaminase Deficiency
Investigation of ADA Enzyme Deficiency
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study and were found to be lymphopenic in at least one examination will be included…
- Trial name
- NCT05300334
- Testing
- adenosine deaminase test
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people
- Sponsor
- TRPHARM
Immunologic Deficiency Syndrome, Agammaglobulinemia
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
The objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% caprylate/chromatography (IGIV-C)purified is similar when infused at two different infusion rates.
- Conditions
- Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome
- Trial name
- NCT00220766
- Testing
- Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water
- Where
- 13 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Grifols Therapeutics LLC
Ulcerative Colitis
A Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Moderately to Severely Active Ulcerative Colitis (MK-7240-005)
The purpose of this study is to assess the safety and efficacy of tulisokibart in participants with moderately to severely active Ulcerative Colitis (UC).
- Trial name
- ARTEMIS-UC (NCT04996797)
- Testing
- Tulisokibart, Companion Diagnostic (CDx) Testing
- Where
- 88 sites in United States, Australia and 10 more
- Volunteers
- 178 people
- Sponsor
- Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Adenosine Deaminase Deficiency
Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E
This study was designed as an observational, prospective, epidemiological screening study. Patients who have been admitted to the center and whose lymphopenia and/or Immunoglobulin E elevation has been detected in at least one…
- Trial name
- NCT05300373
- Testing
- ADENOSINE DEAMINASE TEST
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- TRPHARM
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Peanut Allergy
ARTEMIS Peanut Allergy In Children
The purpose of this study is to demonstrate the efficacy and safety of AR101 through oral immunotherapy (OIT) in peanut-allergic children.
- Trial name
- ARTEMIS (NCT03201003)
- Testing
- AR101 powder provided in capsules & sachets
- Where
- 18 sites in France, Germany and 5 more
- Volunteers
- 175 people
- Sponsor
- Aimmune Therapeutics, Inc.
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.
Saccular and Fusiform Aneurysms, Pseudoaneurysm
Targeting Real World Usage In Stroke Treatment
TRUST Registry is an observational, prospective, long-term, post-market surveillance registry of subjects treated with WallabyPhenox flow modulation devices, stent systems, bifurcation aneurysm implants including their HPC…
- Conditions
- Saccular and Fusiform Aneurysms, Pseudoaneurysm, Vascular Dissection, Perforation
- Trial name
- TRUST (NCT07474935)
- Testing
- Aneurysm Treatment with a Neurovascular Flow Diverter, Dissection Treatment with a Neurovascular Flow Diverter, Perforation Treatment with a Neurovascular Flow Diverter
- Where
- 13 sites in France, Germany and 3 more
- Volunteers
- 5,000 people
- Sponsor
- Phenox GmbH
Epilepsy
Study to Evaluate the Safety and Efficacy of USL261 (Intranasal Midazolam) in Patients With Seizure Clusters
The purpose of this study is to examine the safety and effectiveness of USL261 for the outpatient treatment of seizure clusters.
- Trial name
- ARTEMIS1 (NCT01390220)
- Testing
- USL261
- Where
- 90 sites in United States, Australia and 9 more
- Volunteers
- 292 people
- Sponsor
- UCB Biopharma S.P.R.L.
SCID
JSP191 Antibody Targeting Conditioning in SCID Patients
A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation
- Trial name
- NCT02963064
- Testing
- Humanized anti-CD117 Monoclonal Antibody (JSP191)
- Where
- 9 sites in United States
- Volunteers
- 23 people
- Sponsor
- Jasper Therapeutics, Inc.
Mesothelioma, Malignant
Study of the Safety and Efficacy of Amatuximab in Combination With Pemetrexed and Cisplatin in Subjects With Unresectable Malignant Pleural Mesothelioma (MPM)
This study was originally designed as a multicenter, double-blind, randomized, parallel-group study, using a placebo control or amatuximab 5 milligrams per kilogram (mg/kg), administered weekly, designed to evaluate the safety…
- Trial name
- ARTEMIS (NCT02357147)
- Testing
- Amatuximab, Pemetrexed, Cisplatin
- Where
- 49 sites in United States, Australia and 4 more
- Volunteers
- 124 people
- Sponsor
- Morphotek