Clinical trials: Immune Deficiencies
AL Amyloidosis
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability.
- Trial name
- OLE (NCT02613182)
- Testing
- NEOD001
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Prothena Biosciences Ltd.
Multiple Myeloma, Amyloidosis
APG-2575 in Combination With Novel Therapeutic Regimens in Subjects With Relapsed or Refractory Multiple Myeloma
This is a Phase Ib/II, open-label, multi-center study evaluating the safety, tolerability, efficacy, and PK/ Pharmacodynamics of APG-2575 in combination with Pd/DRd in patients with relapsed/refractory (RR) multiple myeloma (MM).
- Trial name
- NCT04942067
- Testing
- APG-2575+ Pd, APG-2575 + DRd
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Ascentage Pharma Group Inc.
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Multiple Myeloma, Solitary Plasmacytoma
MargheRITA (Remote Intelligence for Therapeutic Adherence)
It is essential to improve clinical efficiency and management of hematological and oncological patients treated on an outpatient basis.
- Conditions
- Multiple Myeloma, Solitary Plasmacytoma, Amyloidosis, Chronic Myeloid Leukemia
- Trial name
- MargheRITA (NCT05260203)
- Testing
- RITA (App)
- Where
- Italy
- Volunteers
- 124 people
- Sponsor
- Advice Pharma Group srl
Amyloidosis
Phase 1 Study of GSK2315698 in Healthy Japanese Subjects
This is the first study in which GSK2315698 will be administered in Japanese population. The primary objective of the study is to investigate safety and tolerability, pharmacokinetics, and pharmacodynamics after single…
- Trial name
- NCT02953808
- Testing
- GSK2315698
- Where
- Japan
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS)
PRISM Registry: Pseudobulbar Affect Registry Series
PBA is a neurologic condition that is estimated to impact over a million patients and their families in the United States. PBA occurs secondary to an otherwise unrelated neurologic disease or injury, and manifests as involuntary…
- Conditions
- Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Parkinson's Disease
- Trial name
- NCT01366027
- Where
- United States
- Volunteers
- 5,290 people
- Sponsor
- Avanir Pharmaceuticals
Primary Systemic (AL) Amyloidosis
The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis.
- Trial name
- VITAL (NCT02312206)
- Testing
- NEOD001
- Where
- 79 sites in United States, Australia and 13 more
- Volunteers
- 260 people
- Sponsor
- Prothena Biosciences Ltd.
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
Multiple Myeloma, Immunoglobulin Light-chain Amyloidosis
A Study to Investigate the Relationship Between Duration of Treatment and Response in Patients With Multiple Myeloma (MM) or Systemic AL Amyloidosis Treated in Real-life Practice
The study will provide information on outcomes in people with multiple myeloma, or systemic AL amyloidosis, or both, under standard care. AL is short for amyloid light-chain.
- Trial name
- DOrianT (NCT04659798)
- Where
- 14 sites in Spain
- Volunteers
- 240 people
- Sponsor
- Takeda
Anemia, Iron-Deficiency, Kidney Failure, Chronic
Effect of Ferrlecit® Versus Oral Iron on Iron Deficient Chronic Kidney Disease (CKD) Patients Receiving Erythropoietic Agents
This study compares the effect of Ferrlecit® (a form of intravenous iron) to ferrous sulfate (a form of oral iron) in treating anemia and iron deficiency in chronic kidney disease patients who are receiving erythropoietic agents…
- Trial name
- NCT00224042
- Testing
- Sodium Ferric Gluconate complex in sucrose, Ferrous sulfate tablets
- Where
- 24 sites in United States, Puerto Rico
- Volunteers
- 52 people
- Sponsor
- Watson Pharmaceuticals
Amyotrophic Lateral Sclerosis (ALS), Parkinson Disease (PD)
Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers
This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS), Parkinson Disease (PD), Alzheimer Disease (AD), Multiple Sclerosis (MS)
- Trial name
- NCT05395624
- Testing
- 18F-OP-801
- Where
- 3 sites in United States
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Ashvattha Therapeutics, Inc.
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Study of Electrical Impedance Myography (EIM) in ALS
This trial is studying Electrical Impedance Myography (EIM) for measuring muscle health. The trial is studying people with Amyotrophic Lateral Sclerosis (ALS), other neuromuscular diseases, and healthy volunteers to see if the…
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
- Trial name
- NCT02011204
- Testing
- Electrical impedance myography (EIM)
- Where
- 6 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Light-Chain Amyloidosis
Study of Oral Ixazomib in Adult Participants With Relapsed or Refractory Light Chain Amyloidosis
This study will include participants with previously treated systemic relapsed or refractory light-chain (AL) amyloidosis who require further therapy and will be aimed at determining the safety profile and the maximum tolerated…
- Trial name
- NCT01318902
- Testing
- Ixazomib, Dexamethasone
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 27 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma
Expanded Access to Venetoclax
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to Venetoclax prior to approval by the local regulatory agency. Availability will depend on territory eligibility.
- Conditions
- Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma, Acute Myeloid Leukemia (AML), Non-Hodgkin's Lymphoma
- Trial name
- NCT03123029
- Testing
- Venetoclax
- Sponsor
- AbbVie
Secondary Immune Deficiency
Single-arm Study of IgPro20 in Adults With Secondary Immune Deficiencies Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies
This is a prospective, multicenter, open-label, single-arm study to assess the efficacy, safety, and pharmacokinetics (PK) of IgPro20 in adults with hematologic malignancies treated with B-cell targeting Chimeric antigen receptor…
- Trial name
- NCT07648264
- Testing
- IgPro20
- Where
- 60 sites in United States, Belgium and 9 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
Amyloid Light Chain (AL) Amyloidosis
Study to Evaluate Change in Disease Activity and Adverse Events of Subcutaneous Etentamig Compared With Daratumumab Plus Cyclophosphamide Plus Bortezomib Plus Dexamethasone (Dara-CyBorD) in Adults With Amyloid Light Chain (AL) Amyloidosis
Amyloid light chain (AL) amyloidosis is a rare disease caused by abnormal plasma cells producing misfolded light chain proteins that deposit in organs, leading to organ dysfunction and failure.
- Trial name
- NCT07793422
- Testing
- Etentamig, Daratumumab, Cyclophosphamide
- Volunteers
- 370 people
- Sponsor
- AbbVie
Amyloidosis; Systemic
A Study to Characterize the Biodistribution of 124I-Labeled AT-03 in Patients With Systemic Amyloidosis
This is a single center Phase 1 study is to evaluate the biodistribution of radiolabeled AT-03 in patients with systemic amyloidosis.
- Trial name
- AT03-001 (NCT05201911)
- Testing
- 124I-AT03
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Attralus, Inc.
Newly Diagnosed Amyloidosis, Amyloidosis Stage I-IIIA
Linvoseltamab Versus Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone (Dara-Vcd) in Adult Participants With Newly Diagnosed Systemic Light Chain Amyloidosis
This study is researching a Regeneron drug called linvoseltamab ("study drug") alone compared to standard of care (SoC) combination to see if it can help treat participants who are newly diagnosed and previously untreated for…
- Trial name
- LINKER-AL1 (NCT07901725)
- Testing
- Linvoseltamab, Daratumumab, Bortezomib
- Volunteers
- 382 people
- Sponsor
- Regeneron Pharmaceuticals
AL Amyloidosis
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down.
- Trial name
- NCT04512235
- Testing
- CAEL-101, cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
- Where
- 110 sites in United States, Australia and 17 more
- Volunteers
- 281 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer