Clinical trials: Immune Deficiencies
Amyloid, Systemic Amyloidosis
Detection of Amyloid Deposits in the Wrist by MRI With Mapping and High Resolution Sequences in Systemic Amyloidosis (AMYLOCARP)
This study is based on the hypothesis that MRI could make it possible to non-invasively detect these amyloid deposits at the level of the wrist using parametric sequences known as T1 mapping, in the form of an extension of T1 in…
- Trial name
- AMYLOCARP (NCT05150353)
- Testing
- MRI
- Where
- France
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- GCS Ramsay Santé pour l'Enseignement et la Recherche
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
AL Amyloidosis
A Study of Belantamab Mafodotin in Patients With Relapsed or Refractory AL Amyloidosis
This is an open-label, multicenter, Phase 2 study in subjects with previously treated patients with light chain (AL) amyloidosis in need for therapy. Approximately 35 subjects will receive therapy with belantamab mafodotin.
- Trial name
- EMN27 (NCT04617925)
- Testing
- Belantamab mafodotin
- Where
- 6 sites in France, Germany and 4 more
- Volunteers
- 35 people
- Sponsor
- Stichting European Myeloma Network
Amyloidosis
A Study to Evaluate the Safety of GSK2398852 When Co-administered With GSK2315698 in Patients With Systemic Amyloidosis
The study will be conducted in two parts. The first (Part A) will be an open label single dose escalation part beginning with the proposed starting dose level of GSK2398852 as 5 milligram (mg) [approximately equivalent to 0.1…
- Trial name
- NCT01777243
- Testing
- GSK2398852, GSK2315698
- Where
- 2 sites in United Kingdom
- Volunteers
- 25 people
- Sponsor
- GlaxoSmithKline
Systemic Amyloidosis
A Phase 1 Study of 99mTc-p5+14 in Healthy Volunteers and Patients With AL or ATTR Systemic Amyloidosis
This study will investigate 99mTc-p5+14, an amyloid-reactive synthetic peptide, p5+14, radiolabeled with technetium-99m, as a radiotracer for detecting paamyloid deposits in patients with AL or ATTR-associated systemic…
- Trial name
- NCT05951816
- Testing
- 99mTc-p5+14 is an amyloid reactive peptide labeled with technetium-99m., 99mTc-Pyrophosphate
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- University of Tennessee Graduate School of Medicine
Amyloidosis, Paraproteinemias
Prospective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance Imaging
Cardiac amyloidosis describes a process by which abnormally folded proteins infiltrate the heart tissue. Given the insidious nature of this disease process, diagnosis is often too late for a meaningful intervention.
- Conditions
- Amyloidosis, Paraproteinemias, Cardiomyopathies
- Trial name
- PICA-CMR (NCT02462213)
- Testing
- Amyloidosis, Cardiac Amyloidosis
- Where
- United States
- Sponsor
- University of Cincinnati
COPD (Chronic Obstructive Pulmonary Disease), ALS (Amyotrophic Lateral Sclerosis)
Psilocybin Therapy for Psychological Distress in Palliative Patients
The goal of this clinical trial is to evaluate whether psilocybin therapy can effectively treat depression and psychological distress in adult patients with COPD, ALS, MS, or APD who have at least 6 months life expectancy.
- Conditions
- COPD (Chronic Obstructive Pulmonary Disease), ALS (Amyotrophic Lateral Sclerosis), MS (Multiple Sclerosis), Major Depressive Disorder (MDD)
- Trial name
- PsyPal (NCT06782724)
- Testing
- Psilocybin therapy
- Where
- 4 sites in Czechia, Denmark and 2 more
- Volunteers
- 108 people
- Sponsor
- University Medical Center Groningen
Chronic Myeloproliferative Disorders, Leukemia
Treatment for Chronic Pain in Patients With Advanced Cancer
RATIONALE: Different drug formulations and combinations of drugs may help patients with chronic pain live more comfortably. It is not yet known which regimen is most effective for chronic pain.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00003687
- Testing
- dextromethorphan hydrobromide, morphine sulfate
- Where
- 4 sites in Canada
- Volunteers
- 87 people
- Sponsor
- NCIC Clinical Trials Group
Primary Amyloidosis
Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at…
- Trial name
- NCT01707264
- Testing
- NEOD001
- Where
- 7 sites in United States
- Volunteers
- 69 people
- Sponsor
- Prothena Biosciences Ltd.
AL Amyloidosis
Isatuximab Plus Pomalidomide and Dexamethasone Association for Patients With AL Amyloidosis Not in VGPR or Better After Any Previous Therapy
This phase 2 study ain to evaluate the efficacy of Isatuximab plus Pomalidomide and Dexamethasone (IPd), in patients with AL amyloidosis not in VGPR or better after any previous therapy.
- Trial name
- IsAMYP (NCT05066607)
- Testing
- Isatuximab
- Where
- 15 sites in France
- Volunteers
- 46 people
- Sponsor
- Intergroupe Francophone du Myelome
Pseudobulbar Affect (PBA)
Safety and Efficacy of AVP-923 in PBA Patients With ALS or MS
Objectives of the study are to evaluate the safety, tolerability, and efficacy of two different doses of AVP-923 (capsules containing either 30 mg of dextromethorphan hydrobromide and 10 mg of quinidine sulfate [AVP-923-30] or 20…
- Trial name
- STAR (NCT00573443)
- Testing
- dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg, dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg
- Where
- 62 sites in United States, Argentina and 1 more
- Volunteers
- 326 people
- Sponsor
- Avanir Pharmaceuticals
Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis)
ALS Research Collaborative
The goal of this natural history study is to learn more about the biological and clinical aspects of amyotrophic lateral sclerosis (ALS).
- Conditions
- Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis), ALS With Frontotemporal Dementia (ALS/FTD), Motor Neuron Disease
- Trial name
- ARC (NCT06885918)
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- ALS Therapy Development Institute
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Chronic Myeloproliferative Disorders, Leukemia
Methemoglobinemia in Young Patients With Hematologic Cancer or Aplastic Anemia Treated With Dapsone
RATIONALE: Gathering information about how often methemoglobinemia occurs in young patients receiving dapsone for hematologic cancer or aplastic anemia may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder
- Trial name
- NCT00993694
- Testing
- chemotherapy, dapsone, medical chart review
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- Vanderbilt University
Post-stroke, Parkinson Disease
Evaluation of the Impact of Virtual Park on Training Motivation in Adult Patients
The purpose of this clinical study is to evaluate the feasibility, usability, and motivational impact of VirtualPark, a virtual reality-based dual-task rehabilitation system, in adults with neurological and age-related…
- Conditions
- Post-stroke, Parkinson Disease, Amyotrophic Lateral Sclerosis, Mild Cognitive Impairment (MCI)
- Trial name
- VP_Adults (NCT07616050)
- Testing
- Dual-task training with Virtual Reality, Cycling training
- Where
- 7 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Marta Mondellini
Light Chain Amyloidosis
Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
This study is a multicenter, open label, fixed dose exploratory clinical trial with an expected enrollment of 30 subjects. The main objective is to evaluate the safety of targeted BCMA autologous CART cell injection in the…
- Trial name
- NCT06983951
- Testing
- BCMA CART
- Where
- China
- Sponsor
- Beijing Boren Hospital
Multiple Myeloma
Collection of Samples and Clinical Data From Patients With Amyloid Diseases
RATIONALE: Collecting and storing samples of blood, urine, tissue and data from patients with amyloid diseases to test in the laboratory may help the study of this disease in the future.
- Trial name
- NCT00898235
- Testing
- clonality analysis, polymerase chain reaction, protein analysis
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Boston Medical Center
Amyloidosis; Systemic
Daratumumab Combined With Bortezomib and Dexamethasone in Mayo 04 Stage III Light Chain Amyloidosis
Patients with light chain (AL) amyloidosis who have advanced cardiac damage are at risk of premature mortality. There is ongoing unmet need for effective therapies to rapidly induce deep hematologic response and decrease the…
- Trial name
- NCT04474938
- Testing
- Daratumumab, Bortezomib, Dexamethasone
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Peking Union Medical College Hospital
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
EEG and TMS-based Biomarkers of ALS, MS and FTD
The purpose of this observational study is to improve understanding of the biology of why ALS, MS and FTD have different effects on different people and facilitate better measurement of the disease in future drug testing.
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Multiple Sclerosis
- Trial name
- NCT04918251
- Testing
- 128 electrode electroencephalography (EEG), Transcranial magnetic stimulation (TMS)
- Where
- Ireland
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University of Dublin, Trinity College
Monoclonal Gammopathy of Renal Significance
Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone Therapy for Patients with MIDD
This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed monoclonal immunoglobulin deposition disease treated with daratumumab, bortezomib, cyclophosphamide, and dexamethasone.
- Trial name
- NCT06418477
- Testing
- Dara-CyBorD
- Where
- 3 sites in China
- Volunteers
- 20 people
- Sponsor
- Peking University People's Hospital