Neoplasms

All Conditions

Digestive System Diseases

Gland and Hormone Related Diseases

Rare Diseases

Carcinoma

Adenocarcinoma

Pancreatic Neoplasms

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Gastrointestinal Neoplasms

Digestive System Neoplasms

Endocrine Gland Neoplasms

Gastrointestinal Diseases

Pancreatic Diseases

Endocrine System Diseases

Pancreatic Cancer

Antineoplastic Agents

All Drugs and Chemicals

Micronutrients

Anti-Infective Agents

Gastrointestinal Agents

Hematinics

Vitamins

Gemcitabine

Leucovorin

Fluorouracil

Levoleucovorin

Oxaliplatin

Erythromycin

Erythromycin Estolate

Erythromycin Ethylsuccinate

Erythromycin stearate

Asparaginase

Pancrelipase

Pancreatin

Eryaspase

Antimetabolites

Antidotes

Protective Agents

Folic Acid

Vitamin B Complex

Trace Elements

Immunosuppressive Agents

Immunologic Factors

Folinic Acid

Folate

Vitamin B9

ERY001

oxaliplatin 85 mg/m2 levo-leucovorin 200 mg/m2 5-FU 400 mg/m2

5-fluoro-uracil/oxaliplatin/leucovorin (folfox)

Pascal Hammel, Pr MD

A new approach that aims to destroy pancreatic tumor cells through modification of the tumor environment.

Asparagine synthetase (ASNS) is an enzyme wich synthetise asparagine. Asparagine is an essential nutriment for pancreatic cancer cells which have no or low level of ASNS.

by L-asparaginase encapsulated in erythrocytes deplete (supress) Plasma asparagine.

in selected patients having no or low ASNS, may provide a new therapeutic approach.

Efficacy and Safety of L-asparaginase Encapsulated in RBC Combined With Gemcitabine or FOLFOX in 2nd Line for Progressive Metastatic Pancreatic Carcinoma

Phase II, Randomized, Controlled, Clinical Trial Exploring Efficacy and Safety of ERY001 (L-asparaginase Encapsulated in Red Blood Cells) in Association With Gemcitabine or FOLFOX4 in Second-line Therapy for Patients With Progressive Metastatic Pancreatic Carcinoma

Evaluate the effects of eryaspase when combined with chemotherapy for the second line treatment of patients with pancreatic adenocarcinoma in terms of OS, whose tumors has low or no ASNS expression (ASNS 0 or 1+)

Evaluate the effects of eryaspase when combined with chemotherapy for the second line treatment of patients with pancreatic adenocarcinoma in terms of PFS, whose tumors has low or no ASNS expression (ASNS 0 or 1+)

Compare the safety profile in patients treated with eryaspase in combination with chemotherapy versus chemotherapy alone, including adverse events, vital signs and clinical laboratory assessments

Evaluate the effects of eryaspase in combination with chemotherapy on investigator-assessed OS in all randomized patients (all patients) and in patients with ASNS 2+/3+ expressing tumors.

Evaluate the effects of eryaspase in combination with chemotherapy on investigator-assessed PFS in all randomized patients (all patients) and in patients with ASNS 2+/3+ expressing tumors.

Evaluate the effect of eryaspase in combination with chemotherapy on the ORR, and the duration in all comers, patients with ASNS 0/1+ expressing tumors, and those with ASNS 2+/3+ expressing tumors.

Evaluate the effect of eryaspase in combination with chemotherapy on the DCR in all comers, patients with ASNS 0/1+ expressing tumors, and those with ASNS 2+/3+ expressing tumors.

Evaluate the effect of eryaspase in combination with chemotherapy on the DoR in all comers, patients with ASNS 0/1+ expressing tumors, and those with ASNS 2+/3+ expressing tumors.

Evaluate the relationship of clinical outcome (i.e. OS, PFS, ORR, DCR and DoR) with tumor markers, namely cancer antigen (CA19-9), and carcinoembryonic antigen test (CEA).

Assess the effect of eryaspase in combination with chemotherapy on PFS, OS, ORR, BOR, and other clinical outcomes in ASNS subsets, as determined by optical density reading.

Compare the 2 treatment arms with respect to change in quality of life status, the change of QOL relative to baseline

ERYtech Pharma

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Symptoms and General Pathology

Anemia, Sickle Cell

Thalassemia

Anemia

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Congenital

Hematologic Diseases

Hemoglobinopathies

Genetic Diseases, Inborn

Sickle Cell Anemia

Patients with SCD who are chronically transfused (in the U.S. and Brazil)

Patients with thalassemia who are chronically transfused in the U.S.

Patients in U.S. with pediatric oncologic diagnoses with hypo-proliferative bone marrow requiring single unit red blood cell transfusion

Allogenic blood donors in the US (estimated: 10,200) and allogenic blood donors in Brazil (estimated: 2,100) with extended donation genotyping using an investigational hematology array.

Simple RBC transfusion or partial manual exchange

Red Blood Cell (RBC) Transfusion

Eldad A Hod, MD

Brian Custer, PhD, MPH

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.

Sickle cell disease (SCD) and thalassemia are genetic disorders inducing anemia of differing pathophysiology. A primary therapy for preventing certain SCD complications (e.g., stroke) and for thalassemia major is regular red blood cell (RBC) transfusion, coupled with iron chelation to prevent the complications of transfusion-induced iron overload. For patients with pediatric hematology-oncology diagnoses with chemotherapy-induced aplasia, RBC transfusion is also common, but the degree of transfusion-induced iron overload and its implications for these patients is incompletely understood. Because iron-related tissue toxicity is a major cause of morbidity and mortality in regularly transfused patients, developing strategies to minimize iron loading and iron toxicity is a key objective of this proposal (study Aim #2), stemming from the objective to optimize RBC unit characteristics that patients with SCD and thalassemia receive beyond RBC phenotype matching for Rh C, E and K antigens (study Aim #1). The study will enroll patients with SCD, thalassemia or pediatric oncologic diagnoses receiving eligible transfusion at 6 hospital sites in the United States, as well as patients with SCD at 5 hemocenters in Brazil.

Specimens to be stored for future use from enrolled red cell transfusion recipients include whole blood, plasma and serum.

The study will also retain a sample of packed RBCs from the transfused RBC unit, and a retention tube from the donor of that transfused unit (to be used for extended genotyping but not retained in a long-term biorepository).

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients (RBC-IMPACT)

De-identified public use datasets will be created and delivered to NHLBI at the end of the study (and end of the REDS-IV-P program) and will be made available indefinitely for future analysis

Recipient oxidative stress pre-transfusion is associated with "RBC survival"

Recipient inflammation pre-transfusion is associated with "RBC survival"

Transfusion reactions are associated with "RBC survival"

Change in hemoglobin A or hemoglobin level per day in between subsequent transfusion episodes, for sickle cell disease and thalassemia cohorts, respectively

For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion

Hemoglobin increment \[defined as Hb/HbA(post-transfusion)visit(i) - Hb/HbA(pre-transfusion)visit(i)\] is associated with "RBC survival"

Includes serum iron, indirect bilirubin, or plasma free hemoglobin

Hepcidin level at time of transfusion is a predictor of change in iron parameters (i.e., transferrin saturation, serum iron) following transfusion

NTBI levels in patients with pediatric oncologic diagnoses with aplasia are elevated at baseline and increase following transfusion

Increased NTBI, serum iron, or transferrin saturation following transfusion is associated with increased risk of clinical adverse effects (i.e., new infections, SCD complications)

National Heart, Lung, and Blood Institute (NHLBI)

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Westat

Anti-Inflammatory Agents

Antirheumatic Agents

Analgesics

Chrysarobin

Epoetin Alfa

Peginesatide starting dose of 0.1 milligram per kilogram (mg/kg) administered subcutaneously (SC) once every 3 weeks (Q3W) for a total of 4 doses.

Peginesatide starting dose of 0.15 mg/kg administered SC Q3W for a total of 4 doses.

Peginesatide starting dose of 0.2 mg/kg administered SC Q3W for a total of 4 doses.

Peginesatide starting dose of 0.05 mg/kg administered SC Q3W for a total of 4 doses.

peginesatide

Affymax

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and pharmacokinetics (PK) of multiple subcutaneously administered injections of peginesatide in anemic cancer participants receiving chemotherapy.

This was a Phase 2, open-label, multi-center, sequential dose finding study with up to 6 treatment cohorts receiving chemotherapy with 15 participants per cohort. The primary objective of this study was to determine the dose of peginesatide administered every 3 weeks (Q3W) by subcutaneous injection associated with a hemoglobin increase of ≥ 1 g/dL in ≥ 50% of anemic cancer participants receiving chemotherapy at 9 weeks following the first dose.

Study of Subcutaneously Administered Peginesatide in Anemic Cancer Patients Receiving Chemotherapy

A Phase 2, Open-Label, Multi-Center Dose Escalation Study of the Safety, Pharmacodynamics, and Pharmacokinetics of Subcutaneously Administered Peginesatide in Anemic Cancer Patients Receiving Chemotherapy

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Kidney Diseases

Kidney Failure, Chronic

Renal Insufficiency

Renal Insufficiency, Chronic

Urologic Diseases

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Male Urogenital Diseases

Chronic Disease

Disease Attributes

Chronic Graft Versus Host Disease

Single injection administered intravenously

Single peginesatide dose of 0.025 milligram per kilogram (mg/kg) administered intravenously.

Single peginesatide dose of 0.05 mg/kg administered intravenously.

Single peginesatide dose of 0.10 mg/kg administered intravenously.

Placebo

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and minimum pharmacologically active dose of peginesatide in Healthy Volunteers.

This was a Phase 1, double-blind, placebo-controlled trial of peginesatide, an erythropoiesis stimulating agent, with approximately 7 treatment cohorts and 7 healthy volunteer participants per cohort. In each cohort of 7 participants, a ratio of 5:2 participants was randomly assigned to receive a single dose of peginesatide or placebo, respectively, administered as an intravenous infusion. Planned peginesatide dose levels were to escalate by cohort. The study was conducted at a single clinical center.

Safety and Pharmacokinetics of Single Intravenous Doses of Peginesatide in Healthy Volunteers

A Phase 1, Double-blind, Placebo-controlled, Dose Escalation Study of the Safety and Pharmacokinetics of Single Intravenous Doses of Peginesatide in Healthy Volunteers

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy and Safety of L-asparaginase Encapsulated in RBC Combined With Gemcitabine or FOLFOX in 2nd Line for Progressive Metastatic Pancreatic Carcinoma	NCT02195180	Pancreatic Aden...	ERY001 Gemcitabine 5-fluoro-uracil...	18 Years -	ERYtech Pharma
Efficacy and Safety of L-asparaginase Encapsulated in RBC Combined With Gemcitabine or FOLFOX in 2nd Line for Progressive Metastatic Pancreatic Carcinoma	NCT02195180	Pancreatic Aden...	ERY001 Gemcitabine 5-fluoro-uracil...	18 Years -	ERYtech Pharma
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients	NCT05255445	Sickle Cell Dis... Thalassemia Pediatric Cance...	Red Blood Cell ...	-	Westat
Study of Subcutaneously Administered Peginesatide in Anemic Cancer Patients Receiving Chemotherapy	NCT00272662	Chemotherapy In... Cancer	peginesatide	18 Years - 80 Years	Affymax
Safety and Pharmacokinetics of Single Intravenous Doses of Peginesatide in Healthy Volunteers	NCT00097747	Anemia Chronic Kidney ... Chronic Renal F... Cancer	Placebo peginesatide	18 Years - 40 Years	Affymax

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