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54767414AMY3001: A Randomized Phase 3 Study to Evaluate the Efficacy and Safety of Daratumumab in Combination with Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) Compared With CyBorD Alone in Newly Diagnosed Systemic AL Amyloidosis (CyBorD)
Rochester, Minn.,
Jacksonville, Fla.,
Scottsdale/Phoenix, Ariz.
The purpose of this study is to evaluate the efficacy and safety of daratumumab plus cyclophosphamide, bortezomib and dexamethasone (CyBorD) compared with CyBorD alone in treatment of newly diagnosed amyloid light chain (AL) amyloidosis participants.
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A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3
Rochester, Minn.
The purpose of this study is to monitor the survival of patients who are no longer actively participating in ISIS 420915-CS2 or ISIS 420915-CS3 and were in the ISIS 420915-CS2 safety population.
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A Phase 1 Study of ION251 Administered by Intravenous Infusion
to Patients with Relapsed/Refractory Multiple Myeloma
Rochester, Minn.,
Scottsdale/Phoenix, Ariz.,
Jacksonville, Fla.
The purpose of this study is to determine the maximum-tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION251 in patients with relapsed/refractory multiple myeloma.
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A Phase 1/2, Dose Escalation Safety and Tolerability Study of AO-176 as Monotherapy and in Combination with Bortezomib and Dexamethasone in Adults with Relapsed or Refractory Multiple Myeloma (AO-176-102)
Rochester, Minn.,
Scottsdale/Phoenix, Ariz.,
Jacksonville, Fla.
The purpose of this study is to evaluate the safety and tolerability of AO-176, including dose-limiting toxicities (DLT), maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) when administered as monotherapy, and in combination with dexamethasone (AO-176+DEX) in adult patients with Relapsed/Refractory Multiple Myleoma (R/R MM).
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A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Rochester, Minn.
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 the maximum tolerated dose.
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A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy
Rochester, Minn.
The purpose of this study is to evaluate the efficacy and safety of ISIS-TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy
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A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction (PRONTO)
Rochester, Minn.,
Jacksonville, Fla.
This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.
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A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis (VITAL)
Jacksonville, Fla.,
Rochester, Minn.
This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached.
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A Phase IIb, Randomized, Double-Blind, Placebo-Controlled Study of NEOD001 in Previously Treated Subjects with Light Chain Amyloidosis who have Persistent Cardiac Dysfunction (PRONTO)
Jacksonville, Fla.,
Rochester, Minn.
The purpose of this study is to assess the effectiveness and safety of NEOD001 administered intravenously in adults with light chain amyloidosis who have had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.
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An Open Label, Prospective, Study to Assess the Safety, Tolerability, Efficacy and Pharmacokinetics of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD) (APL2-CP-AIHA-208)
Rochester, Minn.
This study is to assess the safety, tolerability, preliminary efficacy, and pharmacokinetics of APL-2 in subjects with warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD).
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An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)
Rochester, Minn.
This study evaluates the safety and tolerability of extended dosing with ISIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
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Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)
Rochester, Minn.
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
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In Vitro Studies in Cold Agglutinin Disease
Rochester, Minn.
The purpose of this study is to assess antibody activity IN vitro in cold agglutinin disease (CAD) patients.
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ION-682884-CS3 A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Rochester, Minn.
Evaluating the efficacy of ION-682884 after administration for 65 weeks, as compared to the historical control of the placebo cohort (inotersen) in the NEURO-TTR trial, based on the change from Baseline in serum TTR concentration, mNIS+7 and in the Norfolk Quality of Life.
Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) in patients with hATTR. To evaluate the efficacy of ION-682884, as compared to the placebo cohort in the NEURO-TTR trial, based on the change from Baseline in the following measures:
- Neuropathy Symptom and Change Score (NSC)
- Physical component summary (PCS) score of 36-Item Short Form Survey (SF-36)
- Polyneuropathy disability (PND) score
- Modified body mass index (mBMI)
To evaluate safety and tolerability in hATTR-PN patients treated with ION-682884 including the change from Baseline in platelet count and renal function, the presence of adverse events (AEs). To evaluate the efficacy of ION-682884 in mNIS+7 at Week 85, compared to Baseline.
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Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of NEOD001 in Subjects with Light Chain (AL) Amyloidosis (OLE)
Rochester, Minn.
The purpose of this study is to provide additional treatment with NEOD001 and evaluate long term safety and tolerability for patients wih light chain amyloidosis who completed the previous study NEOD001-001.
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Prospective, Multi-Site Pilot Study to Evaluate Improvement in Disease Management and Communication for Patients with Multiple Myeloma or Amyloidosis Using the “Patient Appointment Companion” Tool
Rochester, Minn.
The purpose of this study is to pilot the patient appointment companion (PAC) tool in use with patients with amyloidosis and multiple myeloma (MM). Patients with hematologic malignancies have unique information and communication needs compared to those with solid tumors. The internet has shifted the dynamics of the patient–doctor relationship, toward one of more mutual participation whereby power and responsibility are shared. The PAC was designed as a patient-centered, user-friendly platform that aids patients’ preparation for their visits with clinicians. PAC provides an online tool that empowers patients to direct their treatment preferences and care alongside their physician teams per the provisions of HIPAA. Data will be collected to:
- Assess the feasibility of using PAC to prepare for clinic vists; and
- Assess the helpfulness/acceptability of the tool.
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