Rho Inc.

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News Article | May 31, 2017
Site: globenewswire.com

SOLANA BEACH, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ:EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) diseases, today announced that it has entered into an agreement with Rho, Inc., a regulatory consulting and contract research organization (CRO). Rho will assist Evoke with the preparation and submission of its planned 505(b)(2) New Drug Application (NDA) for Gimoti™, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. “We are actively laying the necessary groundwork to submit an NDA package for Gimoti to the U.S. Food and Drug Administration (FDA). We believe that it was imperative to select a CRO with relevant experience to optimize the chance of a successful submission leading to approval for Gimoti,” stated Dave Gonyer, R.Ph. President and CEO. “Rho has worked on other successful NDA submissions that have led to FDA approval of GI products, and we look forward to leveraging the experience of their dedicated team in the preparation of our application. In parallel, we remain on track to initiate and complete our PK study in the second half of this year with our NDA submission by late 2017 or early 2018.” Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The Company is developing Gimoti, a metoclopramide nasal spray for the relief of symptoms associated with acute and recurrent gastroparesis in women with diabetes mellitus. Diabetic gastroparesis is a disorder afflicting millions of sufferers worldwide, in which the stomach takes too long to empty its contents resulting in serious digestive system symptoms. Metoclopramide is the only product currently approved in the United States to treat gastroparesis, and is currently available only in oral and intravenous forms. Gimoti is a novel formulation of this drug, designed to provide systemic delivery of metoclopramide through nasal administration. Visit www.EvokePharma.com for more information. Rho, a privately-held, contract research organization (CRO) located in Chapel Hill, NC, provides a broad range of clinical research services across the entire drug development process. For more than 32 years, Rho has been a trusted partner to some of the industry’s leading pharmaceutical, biotechnology, and medical device companies, as well as academic and government organizations. Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negatives of these terms or other similar expressions. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding: Evoke’s plans to prepare and submit the Gimoti NDA with Rho’s assistance; and the timing of the NDA submission, if any. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke's business, including, without limitation: risks associated with successfully commencing and receiving favorable results from the planned PK study; later developments with the Food and Drug Administration (FDA) that may be inconsistent with the already completed pre-new drug application (NDA) meetings, including inconsistent conclusions reflected in the official meeting minutes from the FDA; the inherent risks of clinical development of Gimoti; Evoke is entirely dependent on the success of Gimoti, and Evoke cannot be certain that it will be able to submit an NDA for Gimoti or obtain regulatory approval for or successfully commercialize Gimoti; risks associated with manufacturing new formulations of Gimoti for use in the PK trial; Evoke’s dependence on third parties for the manufacture of Gimoti as well as the conduct of the PK trial; Evoke’s dependence on Rho to assist with the NDA submission; Evoke may require additional funding to complete the PK trial and submit the NDA, and will require substantial additional funding to commercialize Gimoti, and may be unable to raise capital when needed, including to fund ongoing operations; and other risks detailed in Evoke's prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.


News Article | May 31, 2017
Site: globenewswire.com

SOLANA BEACH, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ:EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) diseases, today announced that it has entered into an agreement with Rho, Inc., a regulatory consulting and contract research organization (CRO). Rho will assist Evoke with the preparation and submission of its planned 505(b)(2) New Drug Application (NDA) for Gimoti™, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. “We are actively laying the necessary groundwork to submit an NDA package for Gimoti to the U.S. Food and Drug Administration (FDA). We believe that it was imperative to select a CRO with relevant experience to optimize the chance of a successful submission leading to approval for Gimoti,” stated Dave Gonyer, R.Ph. President and CEO. “Rho has worked on other successful NDA submissions that have led to FDA approval of GI products, and we look forward to leveraging the experience of their dedicated team in the preparation of our application. In parallel, we remain on track to initiate and complete our PK study in the second half of this year with our NDA submission by late 2017 or early 2018.” Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The Company is developing Gimoti, a metoclopramide nasal spray for the relief of symptoms associated with acute and recurrent gastroparesis in women with diabetes mellitus. Diabetic gastroparesis is a disorder afflicting millions of sufferers worldwide, in which the stomach takes too long to empty its contents resulting in serious digestive system symptoms. Metoclopramide is the only product currently approved in the United States to treat gastroparesis, and is currently available only in oral and intravenous forms. Gimoti is a novel formulation of this drug, designed to provide systemic delivery of metoclopramide through nasal administration. Visit www.EvokePharma.com for more information. Rho, a privately-held, contract research organization (CRO) located in Chapel Hill, NC, provides a broad range of clinical research services across the entire drug development process. For more than 32 years, Rho has been a trusted partner to some of the industry’s leading pharmaceutical, biotechnology, and medical device companies, as well as academic and government organizations. Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negatives of these terms or other similar expressions. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding: Evoke’s plans to prepare and submit the Gimoti NDA with Rho’s assistance; and the timing of the NDA submission, if any. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke's business, including, without limitation: risks associated with successfully commencing and receiving favorable results from the planned PK study; later developments with the Food and Drug Administration (FDA) that may be inconsistent with the already completed pre-new drug application (NDA) meetings, including inconsistent conclusions reflected in the official meeting minutes from the FDA; the inherent risks of clinical development of Gimoti; Evoke is entirely dependent on the success of Gimoti, and Evoke cannot be certain that it will be able to submit an NDA for Gimoti or obtain regulatory approval for or successfully commercialize Gimoti; risks associated with manufacturing new formulations of Gimoti for use in the PK trial; Evoke’s dependence on third parties for the manufacture of Gimoti as well as the conduct of the PK trial; Evoke’s dependence on Rho to assist with the NDA submission; Evoke may require additional funding to complete the PK trial and submit the NDA, and will require substantial additional funding to commercialize Gimoti, and may be unable to raise capital when needed, including to fund ongoing operations; and other risks detailed in Evoke's prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.


News Article | May 31, 2017
Site: globenewswire.com

SOLANA BEACH, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ:EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) diseases, today announced that it has entered into an agreement with Rho, Inc., a regulatory consulting and contract research organization (CRO). Rho will assist Evoke with the preparation and submission of its planned 505(b)(2) New Drug Application (NDA) for Gimoti™, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. “We are actively laying the necessary groundwork to submit an NDA package for Gimoti to the U.S. Food and Drug Administration (FDA). We believe that it was imperative to select a CRO with relevant experience to optimize the chance of a successful submission leading to approval for Gimoti,” stated Dave Gonyer, R.Ph. President and CEO. “Rho has worked on other successful NDA submissions that have led to FDA approval of GI products, and we look forward to leveraging the experience of their dedicated team in the preparation of our application. In parallel, we remain on track to initiate and complete our PK study in the second half of this year with our NDA submission by late 2017 or early 2018.” Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The Company is developing Gimoti, a metoclopramide nasal spray for the relief of symptoms associated with acute and recurrent gastroparesis in women with diabetes mellitus. Diabetic gastroparesis is a disorder afflicting millions of sufferers worldwide, in which the stomach takes too long to empty its contents resulting in serious digestive system symptoms. Metoclopramide is the only product currently approved in the United States to treat gastroparesis, and is currently available only in oral and intravenous forms. Gimoti is a novel formulation of this drug, designed to provide systemic delivery of metoclopramide through nasal administration. Visit www.EvokePharma.com for more information. Rho, a privately-held, contract research organization (CRO) located in Chapel Hill, NC, provides a broad range of clinical research services across the entire drug development process. For more than 32 years, Rho has been a trusted partner to some of the industry’s leading pharmaceutical, biotechnology, and medical device companies, as well as academic and government organizations. Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negatives of these terms or other similar expressions. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding: Evoke’s plans to prepare and submit the Gimoti NDA with Rho’s assistance; and the timing of the NDA submission, if any. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke's business, including, without limitation: risks associated with successfully commencing and receiving favorable results from the planned PK study; later developments with the Food and Drug Administration (FDA) that may be inconsistent with the already completed pre-new drug application (NDA) meetings, including inconsistent conclusions reflected in the official meeting minutes from the FDA; the inherent risks of clinical development of Gimoti; Evoke is entirely dependent on the success of Gimoti, and Evoke cannot be certain that it will be able to submit an NDA for Gimoti or obtain regulatory approval for or successfully commercialize Gimoti; risks associated with manufacturing new formulations of Gimoti for use in the PK trial; Evoke’s dependence on third parties for the manufacture of Gimoti as well as the conduct of the PK trial; Evoke’s dependence on Rho to assist with the NDA submission; Evoke may require additional funding to complete the PK trial and submit the NDA, and will require substantial additional funding to commercialize Gimoti, and may be unable to raise capital when needed, including to fund ongoing operations; and other risks detailed in Evoke's prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.


News Article | May 31, 2017
Site: globenewswire.com

SOLANA BEACH, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ:EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) diseases, today announced that it has entered into an agreement with Rho, Inc., a regulatory consulting and contract research organization (CRO). Rho will assist Evoke with the preparation and submission of its planned 505(b)(2) New Drug Application (NDA) for Gimoti™, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. “We are actively laying the necessary groundwork to submit an NDA package for Gimoti to the U.S. Food and Drug Administration (FDA). We believe that it was imperative to select a CRO with relevant experience to optimize the chance of a successful submission leading to approval for Gimoti,” stated Dave Gonyer, R.Ph. President and CEO. “Rho has worked on other successful NDA submissions that have led to FDA approval of GI products, and we look forward to leveraging the experience of their dedicated team in the preparation of our application. In parallel, we remain on track to initiate and complete our PK study in the second half of this year with our NDA submission by late 2017 or early 2018.” Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The Company is developing Gimoti, a metoclopramide nasal spray for the relief of symptoms associated with acute and recurrent gastroparesis in women with diabetes mellitus. Diabetic gastroparesis is a disorder afflicting millions of sufferers worldwide, in which the stomach takes too long to empty its contents resulting in serious digestive system symptoms. Metoclopramide is the only product currently approved in the United States to treat gastroparesis, and is currently available only in oral and intravenous forms. Gimoti is a novel formulation of this drug, designed to provide systemic delivery of metoclopramide through nasal administration. Visit www.EvokePharma.com for more information. Rho, a privately-held, contract research organization (CRO) located in Chapel Hill, NC, provides a broad range of clinical research services across the entire drug development process. For more than 32 years, Rho has been a trusted partner to some of the industry’s leading pharmaceutical, biotechnology, and medical device companies, as well as academic and government organizations. Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negatives of these terms or other similar expressions. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding: Evoke’s plans to prepare and submit the Gimoti NDA with Rho’s assistance; and the timing of the NDA submission, if any. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke's business, including, without limitation: risks associated with successfully commencing and receiving favorable results from the planned PK study; later developments with the Food and Drug Administration (FDA) that may be inconsistent with the already completed pre-new drug application (NDA) meetings, including inconsistent conclusions reflected in the official meeting minutes from the FDA; the inherent risks of clinical development of Gimoti; Evoke is entirely dependent on the success of Gimoti, and Evoke cannot be certain that it will be able to submit an NDA for Gimoti or obtain regulatory approval for or successfully commercialize Gimoti; risks associated with manufacturing new formulations of Gimoti for use in the PK trial; Evoke’s dependence on third parties for the manufacture of Gimoti as well as the conduct of the PK trial; Evoke’s dependence on Rho to assist with the NDA submission; Evoke may require additional funding to complete the PK trial and submit the NDA, and will require substantial additional funding to commercialize Gimoti, and may be unable to raise capital when needed, including to fund ongoing operations; and other risks detailed in Evoke's prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.


McGowan E.C.,Johns Hopkins University | Bloomberg G.R.,University of Washington | Gergen P.J.,National Institute of Allergy and Infectious Diseases | Visness C.M.,Rho Inc | And 6 more authors.
Journal of Allergy and Clinical Immunology | Year: 2015

Objective Previous data suggest that food allergy (FA) might be more common in inner-city children; however, these studies have not collected data on both sensitization and clinical reactivity or early-life exposures.Methods Children in the Urban Environment and Childhood Asthma birth cohort were followed through age 5 years. Household exposures, diet, clinical history, and physical examinations were assessed yearly; levels of specific IgE to milk, egg, and peanut were measured at 1, 2, 3, and 5 years of age. On the basis of sensitization (IgE ≥0.35 kU/L) and clinical history over the 5-year period, children were classified as having FA or being possibly allergic, sensitized but tolerant, or not allergic/not sensitized.Results Five hundred sixteen children were included. Overall, 55.4% were sensitized (milk, 46.7%; egg, 31.0%; and peanut, 20.9%), whereas 9.9% were categorized as having FA (peanut, 6.0%; egg, 4.3%; and milk, 2.7%; 2.5% to >1 food). The remaining children were categorized as possibly allergic (17.0%), sensitized but tolerant (28.5%), and not sensitized (44.6%). Eighteen (3.5%) reported reactions to foods for which IgE levels were not measured. Food-specific IgE levels were similar in children with FA versus sensitized but tolerant children, except for egg, levels of which were higher in patients with FA at ages 1 and 2 years. FA was associated with recurrent wheeze, eczema, aeroallergen sensitization, male sex, breast-feeding, and lower endotoxin exposure in year 1 but not with race/ethnicity, income, tobacco exposure, maternal stress, or early introduction of solid foods.Conclusions Even given that this was designed to be a high-risk cohort, the cumulative incidence of FA is extremely high, especially considering the strict definition of FA that was applied and that only 3 common allergens were included. © 2014 American Academy of Allergy, Asthma & Immunology.


Salo P.M.,U.S. National Institutes of Health | Calatroni A.,Rho Inc | Gergen P.J.,National Institute of Allergy and Infectious Diseases | Hoppin J.A.,U.S. National Institutes of Health | And 4 more authors.
Journal of Allergy and Clinical Immunology | Year: 2011

Background: The National Health and Nutrition Examination Survey (NHANES) 2005-2006 was the first population-based study to investigate levels of serum total and allergen-specific IgE in the general US population. Objective: We estimated the prevalence of allergy-related outcomes and examined relationships between serum IgE levels and these outcomes in a representative sample of the US population. Methods: Data for this cross-sectional analysis were obtained from NHANES 2005-2006. Study subjects aged 6 years and older (n = 8086) had blood taken for measurement of total IgE and 19 specific IgE levels against common aeroallergens, including Alternaria alternata, Aspergillus fumigatus, Bermuda grass, birch, oak, ragweed, Russian thistle, rye grass, cat dander, cockroach, dog dander, dust mite (Dermatophagoides farinae and Dermatophagoides pteronyssinus), mouse and rat urine proteins, and selected foods (egg white, cow's milk, peanut, and shrimp). Serum samples were analyzed for total and allergen-specific IgE by using the Pharmacia CAP System. Information on allergy-related outcomes and demographics was collected by questionnaire. Results: In NHANES 2005-2006, 6.6% reported current hay fever, and 23.5% had current allergies. Allergy-related outcomes increased with increasing total IgE levels (adjusted odds ratios for a 10-fold increase in total IgE level of 1.86 [95% CI, 1.44-2.41] for hay fever and 1.64 [95% CI, 1.41-1.91] for allergies). Increased levels of plant-, pet-, and mold-specific IgE contributed independently to allergy-related symptoms. The greatest increase in odds was observed for hay fever and plant-specific IgE (adjusted odds ratio, 4.75; 95% CI, 3.83-5.88). Conclusion: In the US population self-reported allergy symptoms are most consistently associated with increased levels of plant-, pet-, and mold-specific IgE. © 2011 American Academy of Allergy, Asthma & Immunology.


Szefler S.J.,National Jewish Health | Gergen P.J.,National Institute of Allergy and Infectious Diseases | Mitchell H.,Rho Inc | Morgan W.,University of Arizona
Journal of Allergy and Clinical Immunology | Year: 2010

For children living in inner cities, asthma tends to be more frequent and severe. To characterize, understand, and treat children with asthma living in the inner city more effectively, the National Institute of Allergy and Infectious Diseases established an Inner-City Asthma Program in 1991. In addition, the revised National Asthma Education and Prevention Program Expert Panel 3 report was introduced with new concepts for asthma management that are now centered on asthma control. The purpose of this review is to highlight features of the National Institute of Allergy and Infectious Diseases Inner-City Asthma Consortium Asthma Control Evaluation study that enhance our knowledge regarding the application of the asthma guidelines and to provide a summary of lessons learned from that important study. We recognized that asthma symptoms and exacerbations are theoretically linked to underlying inflammation of airways but are not direct indicators of inflammation. Based on the observations from the Asthma Control Evaluation study, we were impressed that a systematic guidelines-based approach improved asthma control significantly over the course of the 1-year treatment period.


Thornburg C.D.,Duke University | Calatroni A.,Rho Inc | Panepinto J.A.,Medical College of Wisconsin
Journal of Pediatric Hematology/Oncology | Year: 2011

Hydroxyurea is a safe and efficacious medication for children with sickle cell disease (SCD). Our objective was to compare health-related quality of life (HRQL) between children taking hydroxyurea and those not taking hydroxyurea. We conducted a retrospective cohort study of children with SCD who had completed the PedsQL 4.0 at Duke University Medical Center or the Midwest Sickle Cell Center. Our primary outcome was HRQL in children receiving hydroxyurea therapy compared with those not receiving hydroxyurea. One hundred and ninety-one children with SCD were included in the study. Children in the hydroxyurea group had higher self-reported Total PedsQL median scores than children in the no hydroxyurea group (P=0.04). Child self-reported physical functioning scores were significantly higher for children in the hydroxyurea group (P=0.01). In conclusion, children with SCD who received hydroxyurea therapy reported better overall HRQL and better physical HRQL than children who did not receive this therapy despite disease severity. Further research assessing the impact of hydroxyurea therapy on HRQL, such as prospective assessment over time, would aid in our understanding of the effectiveness of hydroxyurea for individual children. Ultimately, this may aid in decreasing the barriers to the use of hydroxyurea. Copyright © 2011 by Lippincott Williams & Wilkins.


Arbes Jr. S.J.,Rho Inc. | Matsui E.C.,Johns Hopkins University
Journal of Allergy and Clinical Immunology | Year: 2011

The hygiene hypothesis contends that fewer opportunities for infections and microbial exposures have resulted in more widespread asthma and atopic disease. Consistent with that hypothesis, decreases in infectious oral diseases over the past half century have coincided with increases in the prevalence of asthma and other allergic diseases. This observation has led some researchers to speculate that exposures to oral bacteria, including pathogens associated with periodontal diseases, such as gingivitis and periodontitis, might play a protective role in the development of asthma and allergy. Colonization of the oral cavity with bacteria, including some species of periodontal pathogens, begins shortly after birth, and the detection of serum antibodies to oral pathogens in early childhood provides evidence of an early immune response to these bacteria. Current knowledge of the immune response to oral bacteria and the immunologic pathogenesis of periodontal diseases suggests biologically plausible mechanisms by which oral pathogens could influence the risk of allergic disease. However, studies investigating the association between oral pathogen exposures and allergic disease are few in number and limited by cross-sectional or case-control design, exclusion of young children, and use of surrogate measures of oral bacterial colonization. Additional studies, particularly well-designed case-control studies among very young children and prospective birth cohort studies, are needed. © 2011 American Academy of Allergy, Asthma & Immunology.


Ware R.E.,Baylor College of Medicine | Helms R.W.,Rho Inc.
Blood | Year: 2012

Stroke is a devastating complication of sickle cell anemia (SCA) with high recurrence if untreated. Chronic transfusions reduce recurrent strokes but have associated morbidities including iron overload. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) was a multicenter phase 3 randomized trial comparing standard treatment (transfusions/chelation) to alternative treatment (hydroxyurea/phlebotomy) for children with SCA, stroke, and iron overload. SWiTCH was a noninferiority trial with a composite primary end point, allowing an increased stroke risk but requiring superiority for removing iron. Subjects on standard treatment received monthly transfusions plus daily deferasirox iron chelation. Subjects on alternative treatment received hydroxyurea plus overlap transfusions during dose escalation to maximum tolerated dose (MTD), followed by monthly phlebotomy. Subjects on standard treatment (N = 66) maintained 30% sickle hemoglobin (HbS) and tolerated deferasirox at 28.2 ± 6.0 mg/kg/d. Subjects on alternative treatment (N = 67) initiated hydroxyurea and 60 (90%) reached MTD at 26.2 ± 4.9 mg/kg/d with 29.1% ± 6.7% fetal hemoglobin (HbF). Adjudication documented no strokes on transfusions/chelation but 7 (10%) on hydroxyurea/ phlebotomy, still within the noninferiority stroke margin. The National Heart, Lung, and Blood Institute closed SWiTCH after interim analysis revealed equivalent liver iron content, indicating futility for the composite primary end point. Transfusions and chelation remain a better way to manage children with SCA, stroke, and iron overload. This clinical trial was registered at ClinicalTrials.gov NCT00122980. © 2012 by The American Society of Hematology.

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