The Experts below are selected from a list of 249 Experts worldwide ranked by ideXlab platform

J. R. Groothuis - One of the best experts on this subject based on the ideXlab platform.

  • Safety of Palivizumab in Preterm Infants 29 to 32 Weeks' Gestational Age Without Chronic Lung Disease to Prevent Serious Respiratory Syncytial Virus Infection
    European Journal of Clinical Microbiology and Infectious Diseases, 2003
    Co-Authors: J. R. Groothuis
    Abstract:

    Respiratory syncytial virus is an important cause of hospitalization in preterm infants. Palivizumab, a humanized monoclonal antibody against the respiratory syncytial virus fusion protein, is currently the only Licensed Product in Europe available for prophylaxis of respiratory syncytial virus lower respiratory tract infection. This study was conducted to obtain additional European data on the safety of palivizumab in preterm infants 29–32 weeks' gestational age without chronic lung disease. Subjects less than 6 months old were enrolled between October 2000 and April 2001. Demographic information was obtained and physical examination was performed at enrollment. Subjects received 15 mg/kg palivizumab intramuscularly every 30 days for the duration of the respiratory syncytial virus season. Subjects hospitalized for respiratory illness were tested for respiratory syncytial virus infection with respiratory syncytial virus rapid antigen tests. At monthly visits, interim history for adverse events/respiratory illness and physical exam was performed. A total of 285 subjects were enrolled from 35 centers in 18 countries. The mean (±SD) gestational age was 30.8±1.1 weeks, the mean birth weight 1.5±0.4 kg, and 56% were 5%) were rhinitis, increased cough, fever, pharyngitis, bronchiolitis, and diarrhea. Only six subjects reported adverse events that were considered possibly related to palivizumab. No deaths were reported. Twenty subjects were hospitalized during the study; six of these were respiratory syncytial virus positive. Palivizumab is safe and well tolerated in preterm infants 29–32 weeks' gestation without chronic lung disease.

Joerg Breitkreutz - One of the best experts on this subject based on the ideXlab platform.

  • development of a taste masked granule formulation of sodium phenylbutyrate adapted for paediatric use
    Archives of Disease in Childhood, 2013
    Co-Authors: Nathalie Guffon, Yves Kibleur, Corinna Tissen, William Copalu, Joerg Breitkreutz
    Abstract:

    Background Sodium phenylbutyrate (NaPB), a treatment for urea cycle disorders, has an extremely unpleasant, bitter taste which can compromise compliance and metabolic control, particularly in children. Attempts to mask the taste in food or drinks are unsuccessful and can lead to feeding aversion further complicating management of these serious disorders. Objectives A new, taste-masked, coated-granule formulation (Luc 01) has been developed and the taste characteristics, dissolution and bioequivalence, including taste, safety and tolerability, of this form were compared to the available, Licensed granule Product. Results The in vitro taste profile of NaPB indicated a highly stimulant molecule. Luc 01 released NaPB only after a lag-time of ∼10 s followed by a very slow release during several minutes compared with complete, immediate release of NaPB from Licensed granules. Pharmacokinetic evaluation demonstrated bioequivalence of a 5 g dose of both Products in 13 healthy volunteers. No statistical difference was observed for maximal plasma concentration or area under the plasma concentration-time. Luc 01 was significantly more acceptable, less bitter and less salty (p Conclusions In vitro and in vivo taste profiles indicate that Luc 01 can be swallowed before stimulation of taste receptors by NaPB and is bioequivalent to the Licensed Product. The availability of Luc 01 should improve compliance/efficacy of NaPB treatment and alleviate the burden of administration particularly in children.

  • developing a new formulation of sodium phenylbutyrate
    Archives of Disease in Childhood, 2012
    Co-Authors: Nathalie Guffon, Yves Kibleur, Corinna Tissen, William Copalu, Joerg Breitkreutz
    Abstract:

    Background Sodium phenylbutyrate (NaPB) is used as a treatment for urea cycle disorders (UCD). However, the available, Licensed granule form has an extremely bad taste, which can compromise compliance and metabolic control. Objectives A new, taste-masked, coated-granule formulation (Luc 01) under development was characterised for its in vitro taste characteristics, dissolution profiles and bioequivalence compared with the commercial Product. Taste, safety and tolerability were also compared in healthy adult volunteers. Results The in vitro taste profile of NaPB indicated a highly salty and bitter tasting molecule, but Luc 01 released NaPB only after a lag time of ∼10 s followed by a slow release over a few minutes. In contrast, the Licensed granules released NaPB immediately. The pharmacokinetic study demonstrated the bioequivalence of a single 5 g dose of the two Products in 13 healthy adult volunteers. No statistical difference was seen either for maximal plasma concentration (C max ) or for area under the plasma concentration–time curve (AUC). CI for C max and AUC 0–inf of NaPB were included in the bioequivalence range of 0.80–1.25. One withdrawal for vomiting and five reports of loss of taste perception (ageusia) were related to the Licensed Product. Acceptability, bitterness and saltiness assessed immediately after administration indicated a significant preference for Luc 01 (p Conclusions In vitro dissolution, in vitro and in vivo taste profiles support the view that the newly developed granules can be swallowed before release of the bitter active substance, thus avoiding stimulation of taste receptors. Moreover, Luc 01 was shown to be bioequivalent to the Licensed Product. The availability of a taste-masked form should improve compliance which is critical to the efficacy of NaPB treatment in patients with UCD.

Barney S Graham - One of the best experts on this subject based on the ideXlab platform.

  • strategic priorities for respiratory syncytial virus rsv vaccine development
    Vaccine, 2013
    Co-Authors: Larry J Anderson, Philip R Dormitzer, D J Nokes, Rino Rappuoli, Anna Roca, Barney S Graham
    Abstract:

    Although RSV has been a high priority for vaccine development, efforts to develop a safe and effective vaccine have yet to lead to a Licensed Product. Clinical and epidemiologic features of RSV disease suggest there are at least 4 distinct target populations for vaccines, the RSV naive young infant, the RSV naive child ≥6 months of age, pregnant women (to provide passive protection to newborns), and the elderly. These target populations raise different safety and efficacy concerns and may require different vaccination strategies. The highest priority target population is the RSV naive child. The occurrence of serious adverse events associated with the first vaccine candidate for young children, formalin inactivated RSV (FI-RSV), has focused vaccine development for the young RSV naive child on live virus vaccines. Enhanced disease is not a concern for persons previously primed by a live virus infection. A variety of live-attenuated viruses have been developed with none yet achieving licensure. New live-attenuated RSV vaccines are being developed and evaluated that maybe sufficiently safe and efficacious to move to licensure. A variety of subunit vaccines are being developed and evaluated primarily for adults in whom enhanced disease is not a concern. An attenuated parainfluenza virus 3 vector expressing the RSV F protein was evaluated in RSV naive children. Most of these candidate vaccines have used the RSV F protein in various vaccine platforms including virus-like particles, nanoparticles, formulated with adjuvants, and expressed by DNA or virus vectors. The other surface glycoprotein, the G protein, has also been used in candidate vaccines. We now have tools to make and evaluate a wide range of promising vaccines. Costly clinical trials in the target population are needed to evaluate and select candidate vaccines for advancement to efficacy trials. Better data on RSV-associated mortality in developing countries, better estimates of the risk of long term sequelae such as wheezing after infection, better measures of protection in target populations, and data on the costs and benefits of vaccines for target populations are needed to support and justify funding this process. Addressing these challenges and needs should improve the efficiency and speed of achieving a safe and effective, Licensed RSV vaccine.

D J Nokes - One of the best experts on this subject based on the ideXlab platform.

  • strategic priorities for respiratory syncytial virus rsv vaccine development
    Vaccine, 2013
    Co-Authors: Larry J Anderson, Philip R Dormitzer, D J Nokes, Rino Rappuoli, Anna Roca, Barney S Graham
    Abstract:

    Although RSV has been a high priority for vaccine development, efforts to develop a safe and effective vaccine have yet to lead to a Licensed Product. Clinical and epidemiologic features of RSV disease suggest there are at least 4 distinct target populations for vaccines, the RSV naive young infant, the RSV naive child ≥6 months of age, pregnant women (to provide passive protection to newborns), and the elderly. These target populations raise different safety and efficacy concerns and may require different vaccination strategies. The highest priority target population is the RSV naive child. The occurrence of serious adverse events associated with the first vaccine candidate for young children, formalin inactivated RSV (FI-RSV), has focused vaccine development for the young RSV naive child on live virus vaccines. Enhanced disease is not a concern for persons previously primed by a live virus infection. A variety of live-attenuated viruses have been developed with none yet achieving licensure. New live-attenuated RSV vaccines are being developed and evaluated that maybe sufficiently safe and efficacious to move to licensure. A variety of subunit vaccines are being developed and evaluated primarily for adults in whom enhanced disease is not a concern. An attenuated parainfluenza virus 3 vector expressing the RSV F protein was evaluated in RSV naive children. Most of these candidate vaccines have used the RSV F protein in various vaccine platforms including virus-like particles, nanoparticles, formulated with adjuvants, and expressed by DNA or virus vectors. The other surface glycoprotein, the G protein, has also been used in candidate vaccines. We now have tools to make and evaluate a wide range of promising vaccines. Costly clinical trials in the target population are needed to evaluate and select candidate vaccines for advancement to efficacy trials. Better data on RSV-associated mortality in developing countries, better estimates of the risk of long term sequelae such as wheezing after infection, better measures of protection in target populations, and data on the costs and benefits of vaccines for target populations are needed to support and justify funding this process. Addressing these challenges and needs should improve the efficiency and speed of achieving a safe and effective, Licensed RSV vaccine.

Nathalie Guffon - One of the best experts on this subject based on the ideXlab platform.

  • development of a taste masked granule formulation of sodium phenylbutyrate adapted for paediatric use
    Archives of Disease in Childhood, 2013
    Co-Authors: Nathalie Guffon, Yves Kibleur, Corinna Tissen, William Copalu, Joerg Breitkreutz
    Abstract:

    Background Sodium phenylbutyrate (NaPB), a treatment for urea cycle disorders, has an extremely unpleasant, bitter taste which can compromise compliance and metabolic control, particularly in children. Attempts to mask the taste in food or drinks are unsuccessful and can lead to feeding aversion further complicating management of these serious disorders. Objectives A new, taste-masked, coated-granule formulation (Luc 01) has been developed and the taste characteristics, dissolution and bioequivalence, including taste, safety and tolerability, of this form were compared to the available, Licensed granule Product. Results The in vitro taste profile of NaPB indicated a highly stimulant molecule. Luc 01 released NaPB only after a lag-time of ∼10 s followed by a very slow release during several minutes compared with complete, immediate release of NaPB from Licensed granules. Pharmacokinetic evaluation demonstrated bioequivalence of a 5 g dose of both Products in 13 healthy volunteers. No statistical difference was observed for maximal plasma concentration or area under the plasma concentration-time. Luc 01 was significantly more acceptable, less bitter and less salty (p Conclusions In vitro and in vivo taste profiles indicate that Luc 01 can be swallowed before stimulation of taste receptors by NaPB and is bioequivalent to the Licensed Product. The availability of Luc 01 should improve compliance/efficacy of NaPB treatment and alleviate the burden of administration particularly in children.

  • developing a new formulation of sodium phenylbutyrate
    Archives of Disease in Childhood, 2012
    Co-Authors: Nathalie Guffon, Yves Kibleur, Corinna Tissen, William Copalu, Joerg Breitkreutz
    Abstract:

    Background Sodium phenylbutyrate (NaPB) is used as a treatment for urea cycle disorders (UCD). However, the available, Licensed granule form has an extremely bad taste, which can compromise compliance and metabolic control. Objectives A new, taste-masked, coated-granule formulation (Luc 01) under development was characterised for its in vitro taste characteristics, dissolution profiles and bioequivalence compared with the commercial Product. Taste, safety and tolerability were also compared in healthy adult volunteers. Results The in vitro taste profile of NaPB indicated a highly salty and bitter tasting molecule, but Luc 01 released NaPB only after a lag time of ∼10 s followed by a slow release over a few minutes. In contrast, the Licensed granules released NaPB immediately. The pharmacokinetic study demonstrated the bioequivalence of a single 5 g dose of the two Products in 13 healthy adult volunteers. No statistical difference was seen either for maximal plasma concentration (C max ) or for area under the plasma concentration–time curve (AUC). CI for C max and AUC 0–inf of NaPB were included in the bioequivalence range of 0.80–1.25. One withdrawal for vomiting and five reports of loss of taste perception (ageusia) were related to the Licensed Product. Acceptability, bitterness and saltiness assessed immediately after administration indicated a significant preference for Luc 01 (p Conclusions In vitro dissolution, in vitro and in vivo taste profiles support the view that the newly developed granules can be swallowed before release of the bitter active substance, thus avoiding stimulation of taste receptors. Moreover, Luc 01 was shown to be bioequivalent to the Licensed Product. The availability of a taste-masked form should improve compliance which is critical to the efficacy of NaPB treatment in patients with UCD.