Ferriprox CR (deferiprone controlled-release) / Apotex - LARVOL DELTA

Development of Nanocomposite Microspheres for Nasal Administration of Deferiprone in Neurodegenerative Disorders. (PubMed, J Funct Biomater) - "To ensure the deposition of the particles in the nasal cavity and avoid exhalation or deposition into the small airways, the nanoparticles were incorporated into composite structures of sodium alginate obtained by spray drying. Deferiprone demonstrated sustained release from the nanocomposite microspheres and high iron-chelating activity."

Journal • Agranulocytosis • CNS Disorders • Granulocytopenia • Hematological Disorders • Neutropenia • Otorhinolaryngology

The 3D trial: Deferiprone to Delay Dementia (The 3D Study) (clinicaltrials.gov) - P2 | N=81 | Completed | Sponsor: Neuroscience Trials Australia | Active, not recruiting ➔ Completed | N=171 ➔ 81

Enrollment change • Trial completion • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • APOE

RENAL FUNCTION IN TRANSFUSION-DEPENDENT Β-THALASSEMIA PATIENTS: A DECADE OF FOLLOW-UP AND COMPARISON BETWEEN CHELATION REGIMES (EHA 2018) - "26 pts received deferasirox (DFX) for a mean period of 59 months; this was the first ICT for 6 of them; 10 pts were treated with deferoxamine (DFO) or DFO + deferiprone (DFP) (4 and 6 pts, respectively; hereafter DFO+/-DFP group). Ahigh prevalence of renal tubular abnormalities was observed in our pediatric and adult -TM pts, particularly in the DFX group. The marker of tubular injuryuNAGwas negatively associated with mean 10-year serum ferritin, suggesting ICT's involvement in tubular injury. Moreover, uNAG was associated with transfusional iron burden in the DFO+/-DFP group, but not in the DFX group, proposing a mechanism other than iron overload for the pathogenesis of tubular injury in DFX-treated pts."

Clinical • Hematological Malignancies

The 3D trial: Deferiprone to Delay Dementia (The 3D Study) (clinicaltrials.gov) - P2; N=171; Active, not recruiting; Sponsor: Neuroscience Trials Australia; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • MRI

The 3D trial: Deferiprone to Delay Dementia (The 3D Study) (clinicaltrials.gov) - P2; N=171; Recruiting; Sponsor: Neuroscience Trials Australia; Trial completion date: Dec 2021 ➔ Sep 2023; Trial primary completion date: Jul 2021 ➔ Sep 2022

Clinical • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • MRI

[VIRTUAL] SAFETY AND EFFICACY OF BRINCIDOFOVIR FOR REFRACTORY ADENOVIRUS INFECTION IN CHILDREN RECEIVING ALLOGENEIC SCT: A RETROSPECTIVE ANALYSES FROM THE ITALIAN PEDIATRIC HEMATOLOGY ONCOLOGY ASSOCIATION (AIEOP) (EBMT 2020) - "Within the few therapeutic strategies, Cidofovir (CDV) is the only available approach. Brincidofovir (BCV, CMX001) is an orally bioavailable lipid-conjugate of CDV, effective against double-strand DNA viruses and with a lower toxicity profile...First ADV infection episode occurred at a median time of 3 months after HSCT (range: 20 days-14 months); 61% were receiving Cyclosporine and 61% had diarrhea... BCV resulted effective and well-tolerated in treating CDV-refractory ADV infection in very high risk children after HSCT, the overall BCV response rate being 72%. BCV deserves to be tested in a prospective study."

Retrospective data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Transplantation

[VIRTUAL] SAFETY AND EFFICACY OF BRINCIDOFOVIR FOR REFRACTORY ADENOVIRUS INFECTION IN CHILDREN RECEIVING ALLOGENEIC SCT: A RETROSPECTIVE ANALYSES FROM THE ITALIAN PEDIATRIC HEMATOLOGY ONCOLOGY ASSOCIATION (AIEOP) (EBMT 2020) - "Within the few therapeutic strategies, Cidofovir (CDV) is the only available approach. Brincidofovir (BCV, CMX001) is an orally bioavailable lipid-conjugate of CDV, effective against double-strand DNA viruses and with a lower toxicity profile...First ADV infection episode occurred at a median time of 3 months after HSCT (range: 20 days-14 months); 61% were receiving Cyclosporine and 61% had diarrhea... BCV resulted effective and well-tolerated in treating CDV-refractory ADV infection in very high risk children after HSCT, the overall BCV response rate being 72%. BCV deserves to be tested in a prospective study."

Retrospective data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Transplantation

[VIRTUAL] SAFETY AND EFFICACY OF BRINCIDOFOVIR FOR REFRACTORY ADENOVIRUS INFECTION IN CHILDREN RECEIVING ALLOGENEIC SCT: A RETROSPECTIVE ANALYSES FROM THE ITALIAN PEDIATRIC HEMATOLOGY ONCOLOGY ASSOCIATION (AIEOP) (EBMT 2020) - "Within the few therapeutic strategies, Cidofovir (CDV) is the only available approach. Brincidofovir (BCV, CMX001) is an orally bioavailable lipid-conjugate of CDV, effective against double-strand DNA viruses and with a lower toxicity profile...First ADV infection episode occurred at a median time of 3 months after HSCT (range: 20 days-14 months); 61% were receiving Cyclosporine and 61% had diarrhea... BCV resulted effective and well-tolerated in treating CDV-refractory ADV infection in very high risk children after HSCT, the overall BCV response rate being 72%. BCV deserves to be tested in a prospective study."

Retrospective data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Transplantation

[VIRTUAL] SAFETY AND EFFICACY OF BRINCIDOFOVIR FOR REFRACTORY ADENOVIRUS INFECTION IN CHILDREN RECEIVING ALLOGENEIC SCT: A RETROSPECTIVE ANALYSES FROM THE ITALIAN PEDIATRIC HEMATOLOGY ONCOLOGY ASSOCIATION (AIEOP) (EBMT 2020) - "Within the few therapeutic strategies, Cidofovir (CDV) is the only available approach. Brincidofovir (BCV, CMX001) is an orally bioavailable lipid-conjugate of CDV, effective against double-strand DNA viruses and with a lower toxicity profile...First ADV infection episode occurred at a median time of 3 months after HSCT (range: 20 days-14 months); 61% were receiving Cyclosporine and 61% had diarrhea... BCV resulted effective and well-tolerated in treating CDV-refractory ADV infection in very high risk children after HSCT, the overall BCV response rate being 72%. BCV deserves to be tested in a prospective study."

Retrospective data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Transplantation

Protective effects of grape seed proanthocyanidins against iron overload-induced renal oxidative damage in rats. (PubMed, J Trace Elem Med Biol) - "GSPAs have protective effects on nephrotoxicity in rats with iron overload. Thus, further investigation of GSPAs as a new and natural phytochemo-preventive agent against iron overload is warranted."

Journal • Preclinical • Renal Disease