Clinical Digest: Recent Clinical Trials on Microcephaly
To assist researchers and healthcare practitioners in staying up-to-date with the latest advancements in clinical research, the Clinical Digest Team has compiled a comprehensive list of recent clinical trials related to Microcephaly. These trials are organized by their release dates for your convenience. (Last updated on: 2025-05-02)
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TABLE 1: Clinical Digest: Recent Clinical Trials on Microcephaly
| Clinical Trial | Contact(s) | Date | |
|---|---|---|---|
| 1 | Is A Third Dose of Measles-mumps-rubella-(Varicella) Vaccine (MMR(V)) Vaccine Beneficial for The Adult Population in Alberta? PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: The current recommendation for a full course of measles-mumps-rubella-(varicella) vaccine (MMR(V)) is two doses. The problem is, many individuals within the vaccinated cohort show antibody levels that are below the level considered to be protective, even after two doses of vaccine. Because of these waning antibody levels, it is currently unknown whether highly vaccinated populations are protected from infection against measles, mumps, rubella, … |
UNIVERSITY OF ALBERTA | 2024-11-09 |
| 2 | Clinical Spectrum of Infants of Diabetic Mothers , in Neonatal Intensive Care Unit of Assiut University Children’s Hospital PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Determine spectrum of clinical presentations and complications associated with maternal diabetes mellitus in their newborns attending and admitted to Neonatal Intensive Care Unit of Assiut University Children’s Hospital. |
DINA NASSER MAHROUS DANIAL; | 2024-03-01 |
| 3 | Radiological Evaluation of Hypoxic Ischemic Encephalopathy in Neonatal Intensive Care Unit Of Assuit University Children’s Hospital PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: To compare between Transcranial Ultrasound , MRI and CT in patients with Hypoxic Ischemic Encephalopathyas regards diagnostic accuracy and prognostic value . |
ESRAA AZAB ABDELATTY; | 2023-11-13 |
| 4 | Microcephaly, Fanconi Anemia and Praxial Disorders Related Papers Related Patents Related Grants Related Experts View Highlight: Fanconi Anemia (FA) is mentioned in children with congenital malformations including kidney, hart and skeletal malformations (absence or abnormal thumb or forearm), and bone marrow failure or myelodysplasia with a progressive onset in childhood or adulthood. No study has focused on microcephaly, a reduction in brain volume, which is present in 20% of children, and its consequences on cognitive and structural level of the brain. Since 2014, … |
SANDRINE PASSEMARD; | 2023-01-30 |
| 5 | A Prospective, Multicenter, Randomized, Evaluator-Blinded, Study to Evaluate The Safety and Efficacy of AMZEEQ® Companion Treatment With Oral Isotretinoin Therapy in Patients With Moderate to Severe Acne Vulgaris PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Acne vulgaris is a common disease of both males and females, usually manifesting initially during adolescence. The use of retinoic acid analogues such as adapalene, tazarotene and isotretinoin are also commonly prescribed to treat inflammation; dysregulated sebum production and comedonal acne. This study seeks to evaluate the efficacy and safety of concomitant use of both AMZEEQ® and oral isotretinoin compared to oral isotretinoin only use and to explore sequence dosing of both products as part of a long-term management protocol for acne vulgaris. |
EDWARD LAIN; | 2021-06-28 |
| 6 | Microcephaly at Birth As A Potential Predictor of Poor Prognosis in Infants Weighing 500 Grams or Less: A Retrospective Cohort Study PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: This is a retrospective single-center cohort study. The comparison in short- and long-term outcomes will be made between those with and without primary microcephaly in infants weighing ≤ 500 g. |
RYO ITOSHIMA; | 2021-04-22 |
| 7 | Tolerability and Efficacy of L-Serine in Patients With GRIN-related Encephalopathy PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: GRIN-related disorders encompass a new group of Inborn Errors of Metabolism according to the recent nosology published by Ferreira et al (Genet Med, 2019). These rare conditions represent a subtype of paediatric encephalopathies leading to intellectual disability, hypotonia, communication deficits and motor impairment (Orphanet entries: 178469, 289266, 101685, for GRIN1, GRIN2A and GRIN2B, respectively). Mutations leading to glutamatergic hypotransmission can be potentially treated with L-Serine leading to significant clinical benefits in patients according to a pilot study published by our group (Soto et al, 2019). In our study, … |
ÁNGELS GARCIA CAZORLA; | 2020-07-30 |
| 8 | Neuro-COVID-19: Neurological Complications of COVID-19 Related Papers Related Patents Related Grants Related Experts View Highlight: The prevalence and typical patterns of neurological complications in hospitalized COVID-19 patients admitted to the intensive care units of the University Hospital Zurich will be investigated. The impact of neurological complications among COVID-19 patients on mortality, functional outcome, and organizational outcomes will be analyzed. |
EMANUELA KELLER; | 2020-05-01 |
| 9 | Neurologic Manifestations of COVID 19 in Children PF:6 Related Papers Related Patents Related Grants Related Experts View Highlight: The purpose of this study is to perform a secondary analysis of pre-existing de-identified limited datasets obtained from sites participating in Tier 1 (STUDY20040154: Neurologic Manifestations of COVID 19 in Children) and Tier 2 (STUDY20040278: Neuro COVID – Outcomes). The data will be used to determine the prevalence and severity of neurological symptoms among patients requiring critical care admission for confirmed or suspected novel coronavirus (COVID-19) and to determine the predictors for limitation of care in the study population, as well as, … |
ERICKA FINK; | 2020-04-29 |
| 10 | Evaluation of Medical Conditions Associated With Zika Virus Infection in Managua, Nicaragua PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Zika virus (ZIKV) infection spread throughout the Americas with devastating consequences. Recent limited evidence suggests the potential for neurological effects associated with postnatally acquired ZIKV infection in humans; however, the impact on children is unknown. The researchers will conduct a longitudinal study of approximately 450 Nicaraguan children who were ages 2-12 in 2016 to evaluate the presence and persistence of neurological symptoms associated with ZIKV infection and to test whether ZIKV-infected children are at greater risk for developing neurological outcomes compared to uninfected children. |
JILL F. LEBOV; | 2019-10-01 |
| 11 | Prognostic Factors for Sequelae in Children With A History of Shaken Baby Syndrome in The Central Region Related Papers Related Patents Related Grants Related Experts View Highlight: This is an observational, prognostic and prospective study. It is about the Prognostic Factors for sequelae in children with a history of Shaken Baby Syndrome (SBS) in the Central region in France. The study hypothesis is the prognosis of the sequelae children develop according to the severity criteria presents in admission to the hospital for the Shaken Baby Syndrome. The severity criteria are clinical high intracranial pressure, presence of a coma, vitreous hemorrhage, an age under 6 months, other cerebral lesion than subdural hematoma (parenchymal lesion, … |
UNIVERSITY HOSPITAL TOURS | 2019-06-28 |
| 12 | A Prospective Epidemiological Study of Pregnancy Outcomes and of Events of Interest in Pregnant Women, Neonates and Infants (PEPNI) PF:6 Related Papers Related Patents Related Grants Related Experts View Highlight: The purpose of this study is to assess pregnancy outcomes, and maternal, as well as neonatal events of interest in healthy pregnant women and their new-borns. The study will also determine incidence of lower respiratory tract illness (LRTI) caused by respiratory syncytial virus (RSV) in the new-borns during their first year of life. |
GLAXOSMITHKLINE | 2019-05-30 |
| 13 | Surveillance of Zika-related Microcephaly in Sub-Saharan Africa and Asia PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: This study will explore whether ZIKV is currently responsible for neurological complications, and particularly microcephaly, in Aedes-infested regions of Sub-Saharan Africa (SSA) and Asia. This will inform regional public health strategies, such as vaccination of women of child-bearing age. It will also demonstrate the public health impact of ZIKV infection and increase the understanding of other regional infectious (e.g. cytomegalovirus) causes of microcephaly. |
ARNAUD FONTANET; | 2019-05-20 |
| 14 | Intensive Therapy for Children With Microcephaly, Hyperkinetic Movements, or Global Developmental Delay Related Papers Related Patents Related Grants Related Experts View Highlight: This trial will consist of a clinical series of up to 50 children with Global Developmental Delay and concomitant microcephaly or hyperkinetic movements. All children will be assessed for psychomotor function using standardized assessments, goal specific assessments, with the potential addition of neuroimaging assessment, prior to and after receiving an intensive burst of neuromotor therapy. The interventional effects will be explored by comparing the pre and post interventional assessments and neuroimaging. |
STEPHANIE DELUCA; | 2019-04-30 |
| 15 | Screening For Critical Congenital Anomalies In NICU And Their Out Come in Assiut Universty PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: The congenital malformation (CMF) include disturbance in normal process of organogenesis occurring before birth . – The (CMF) are structural ,functional, or (biochemical Molecular) defect presenting at birth. – The structural defect resulting from prenatal insult during period of embryogeneses. – The neonate with congenital malformation have differences in physiology, anatomy, in response to stress. – The (CMF) are important causes of infant disability , illness, deaths. – (CMF) classified into mild, moderate, severe and lethal |
AMIRA THARWAT HELMY; | 2019-03-19 |
| 16 | Eltrombopag for Patients With Fanconi Anemia PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new drug will help people with this disease. Objective: To find out if a new drug, eltrombopag, … |
ANDRE LAROCHELLE; | 2018-11-02 |
| 17 | Fetal Alcohol Spectrum Disorder-Is This A Ciliopathy? PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Urine sample and exhaled Nitric Oxide will analyzed and compared between children diagnosed with Fetal Alcohol Spectrum Disorder and healthy control. Pilot study- 5 children in each group |
HELLY GOEZ; | 2018-09-01 |
| 18 | Children (CH) Cohort for The Evaluation of Developmental and Neurological Abnormalities in Infants Born to Mothers Residing in Areas With Zika Virus Transmission During Pregnancy PF:5 Related Papers Related Patents Related Grants Related Experts View Highlight: The aim of this proposal is to evaluate the causal relationship between Zika virus (ZIKV) infections in pregnancy and congenital malformations. The study will estimate the absolute and relative risks of congenital malformations and other adverse outcomes of pregnancy among women who become infected with ZIKV during pregnancy compared to uninfected pregnant women, also leading to further validation of the Congenital Zika Syndrome. |
THOMAS JAENISCH; | 2018-01-15 |
| 19 | Study on The Mechanism of Neurodevelopment Dysplasia of Fetal Brain Caused By ASNS Gene Mutation PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: The investigators propose to analyze a brain sample and/or peripheral blood by single cell RNA seq from aborted embryos with ASNS mutation. |
BO CHEN; | 2017-10-28 |
| 20 | Efficacy of Aquatic Physiotherapy in Stress Level and Muscle Tone of Children Between 3 and 24 Months With Microcephaly By Zika Virus Congenital Syndrome PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Microcephaly associated with Congenital Syndrome of the Zika virus (SCVZ) was highlighted due to atypical structural and behavioral changes of the central nervous system. Such changes contribute to the appearance of functional impairments and difficulties of adaptation of the child to the necessary therapeutic follow-up. In this context, the search for adjuvant therapies that allow better management of these alterations has grown and, given its advantages, aquatic physiotherapy may be an auxiliary resource in the care of this population. The aquatic environment is capable of promoting a state of relaxation that, when associated with a kinesiotherapy protocol, … |
MILENA GUIMARÃES MONTEIRO; | 2017-06-01 |
| 21 | Pregnant Women (PW) Cohort for Evaluation of Risks of Congenital Malformations and Other Adverse Pregnancy Outcomes After Zika Virus Infection (Part of ZIKAlliance) PF:6 Related Papers Related Patents Related Grants Related Experts View Highlight: The aim of this proposal is to evaluate the causal relationship between Zika virus (ZIKV) infections in pregnancy and congenital malformations. We will estimate the absolute and relative risks of congenital malformations and other adverse outcomes of pregnancy among women who become infected with ZIKV during pregnancy compared to uninfected pregnant women, also leading to further validation of the Congenital Zika Syndrome. |
THOMAS JAENISCH; | 2017-05-24 |
| 22 | Guangzhou Surveillance and Clinical Study in Microcephaly PF:9 Related Papers Related Patents Related Grants Related Experts View Highlight: Screening for microcephaly is important at birth and during early childhood. The Guangzhou Surveillance and Clinical Study in Microcephaly (GSCSM) aims to establish a multicentric surveillance system for microcephaly in newborns and infants, to develop a new head circumference reference and microcephaly criteria basing on the local population in Guangzhou, to improve the prediction model of microcephaly, and to follow up the outcomes of the children diagnosed with microcephaly. |
XIU QIU; | 2017-02-10 |
| 23 | Study of Zika Virus and Related Arbovirus Infections in Deferred Blood Donors PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Background: Zika virus is mostly passed on by the bite of an infected mosquito. It usually causes mild illness. But in pregnant women it can cause serious birth defects to the baby. The virus can also spread by blood transfusion and sexual intercourse. This is why the U.S. Food & Drug Administration (FDA) recommended that people should not give blood if possibly exposed to Zika virus. Dengue virus and chikungunya virus are passed by the same mosquitoes as Zika virus. These can cause severe reactions if passed through transfused blood. Donated blood is usually not tested for these three viruses. Researchers want to count the infections in people who have been exposed because of travel or sexual exposure. They want to learn the risk these viruses might pose to the U.S. blood supply. They also want to study the natural history of these viruses by following infected people over time. Objective: To study the risk of Zika, dengue, and chikungunya viruses to the U.S. blood supply. Eligibility: Adults age 18 or older who were turned down for donating blood because of possible exposure to certain viruses. Design: Participants will have blood and urine tests. They will answer questions about their travel. They will be called in about a week with virus test results. Participants with negative results do not have any more study visits. Participants with positive results will be asked to stay in the study for 6 months. They will have weekly clinic visits and tests until results are negative for 2 straight weeks. Once test results are negative, they will have monthly visits. Visits will include physical exams, blood and urine samples, and optional semen samples from men. Most people will have 3-4 weekly visits and 5 monthly visits. |
ROBERT D ALLISON; | 2016-06-08 |
| 24 | International Prospective Observational Cohort Study of Zika in Infants PF:7 Related Papers Related Patents Related Grants Related Experts View Highlight: The overall objective of this multisite, multicountry Zika in Infants and Pregnancy (ZIP) study is to assess the strength of the association between Zika virus infection (ZIKV) during pregnancy and adverse maternal/fetal outcomes and the risk of vertical transmission. The study will prospectively enroll a cohort of pregnant women up to 17 weeks and 6 days gestation and subjects at any gestational age with acute Zika infection, confirmed by serology or PCR (polymerase chain reaction) test. The study will follow these women through their pregnancy to identify for clinical evidence of acute ZIKV, … |
RICARDO XIMENES; | 2016-06-01 |
| 25 | Risk Evaluation of Fetal Nervous System and Neurodevelopment Disorders in Infants of Women Exposed to Zika Virus Infection During Pregnancy PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: This prospective cohort study will determine the natural history of fetal exposure to Zika virus (ZIKV) and its effects on the fetus and newborn with emphasis on neurodevelopment outcome. Exposure of the fetus will be determined by maternal symptomatology, RT-PCR ZIKV (blood and urine) and serologic test specific for ZIKV. Neonates will be classified according to trimester of infection and as exposed and unexposed to ZIKV. |
MARIO AUGUSTO ROJAS; | 2016-05-01 |
| 26 | Cytomegalovirus Testing and Intervention Protocol for Newborn Nursery and Newborn Intensive Care Unit PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Objective: Congenital cytomegalovirus (cCMV) is the most common non-genetic cause of pediatric sensorineural hearing loss (SNHL) and an important cause of neurodevelopmental delay. Infants with cCMV can be symptomatic, asymptomatic, or asymptomatic except for hearing loss. Symptomatic infants may be more readily identified and quickly referred for intervention because they may present with classic common clinical findings, but the majority of infants (85-90%) with cCMV are asymptomatic at birth and do not have the classic clinical, laboratory, or radiologic findings and therefore often have delayed identification and intervention. Often, … |
ANN ANDERSON BERRY; | 2016-03-01 |
| 27 | Rash Accompanied By Three or More Dengue-like Symptoms During Pregnancy and Microcephaly: Case-control Study PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: It is suspected that zika virus infection during pregnancy is associated with microcephaly. The objective of this study is to investigate the occurrence of rash accompanied by three or more dengue-like signs and symptoms during pregnancy is related to the microcephaly using the case-control design. |
GUSTAVO BARCELOS BARRA; | 2016-02-01 |
| 28 | Screening for Congenital Cytomegalovirus Infection in Newborns With Failed Hearing Screen or Who Are Small for Gestational Age PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: Our central hypothesis is that screening newborn infants who either fail their newborn hearing test or have a diagnosis of small for gestational age (SGA) will lead to better identification of infants with congenital CMV infection and enhanced rates of therapeutic intervention. This has the potential to significantly improve outcomes for infants with this common viral infection. This particular cohort of patients have not been well studied locally or regionally. In addition, in view of current legislation that will be effective in January, … |
MARCELA RODRIGUEZ; | 2016-02-01 |
| 29 | HIV Exposure, Disease Acquisition and Progression Among Children: Role of Maternal Immunogenetics, Viral Genetic Diversity, HAART Exposure, Co-morbidities and Psycho-Social Status: (UZ-CHS Birth Cohort) PF:5 Related Papers Related Patents Related Grants Related Experts View Highlight: Background Commencement of lifelong highly active antiretroviral therapy (HAART) immediately after HIV diagnosis (option B+), for treatment of human immunodeficiency virus (HIV), has greatly improved maternal-infant health in sub Saharan Africa (SSA). However, this development has also dramatically increased the number of maternally HAART/HIV-exposed-uninfected (HEU) infants in areas of high HIV prevalence. Compared to their HIV-unexposed uninfected (HUU) counterparts, HEU infants show increased mortality, higher rates of adverse birth outcomes, infectious and non-communicable diseases and impaired growth, … |
KERINA DURI; | 2016-01-26 |
| 30 | Vertical Exposure to Zika Virus and Its Consequences for Child Neurodevelopment: Cohort Study in Fiocruz/IFF PF:4 Related Papers Related Patents Related Grants Related Experts View Highlight: The recent increase in the number of cases of congenital microcephaly observed in Brazil is a reason of great concern. This increase occurred a few months after Zika virus (ZIKV) was introduced in the country, which was associated with reports of pregnant women presenting fever and rash illness during pregnancy. Thus, the hypothesis of a relationship between ZIKV infection and microcephaly became plausible. However, studies on the pathophysiology of maternal ZIKV infection, its consequences for the fetus, … |
MARIA E MOREIRA; | 2016-01-02 |
| 31 | The Safety Assessment of Vedolizumab During Pregnancy in IBD Patient PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: The safety assessment of Vedolizumab during pregnancy in IBD patients. The primary objective of this study is: 1. To quantify incidence of major structural birth defects and other birth outcomes in infants born to women with UC/CD with exposure to Entyvio compared to women with exposure to other biological agents or conventional IBD therapy during pregnancy 2. To assess the health and developmental status of infants up to 1yr of age. |
ARIELLA SHITRIT; | 2015-10-01 |
| 32 | Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies (North American Mitochondrial Disease Consortium, Rare Diseases Clinical Research Network, Project 7413) PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Children and adults with pyruvate dehydrogenase complex deficiency (PDCD) are participating in a research study seeking to better understand the genetic causes, symptoms, usefulness of current treatments, and outcomes for these disorders. The research project involves completing a questionnaire about the individual or family’s medical history and experiences with PDCD, review of medical records by the researchers, and in some cases, advanced genetic testing. |
JIRAIR KRIKOR BEDOYAN; | 2015-09-01 |
| 33 | Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol Related Papers Related Patents Related Grants Related Experts View Highlight: Background: – People with inborn errors of metabolism can t turn food into energy the right way. This can affect a person s growth and health. Researchers want to know how this condition affects a pregnant woman and her baby. Objectives: – To collect data from the medical records of women with an inborn error of metabolism. Also, to create a pregnancy registry of inborn errors of metabolism. Eligibility: * Women with an inborn error of metabolism who either: * have been pregnant in the past, * are currently pregnant, … |
CHARLES P VENDITTI; | 2014-12-19 |
| 34 | Correlates of Maternal Characteristics in CMV Infection in Pregnancy With Maternal-fetal Transmission PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Analysis of several characteristics of blood from pregnant women with CMV infection according to maternal-fetal transmission. These include CMV viral load, cytokine profile in response to in-vitro stimulation with CMV peptides, meticulous analysis of anti CMV antibodies, maternal DNA polymorphism and microarray of gene expression. |
YECHIEL SCHLESINGER; | 2014-06-01 |
| 35 | Randomized Trial of Maternal Progesterone Therapy to Improve Neurodevelopmental Outcomes in Infants With Congenital Heart Disease PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Neurodevelopmental disability is now recognized as the most common long-term complication after cardiac surgery in neonates. Research studies have shown that progesterone is critical to the development of the brain and in a variety of clinical situations including brain injury can protect the brain. The purpose of this research study is to determine whether progesterone administered during the 3rd trimester of pregnancy (24-39 weeks) to pregnant women protects the brain of unborn babies with CHD and improves their neurodevelopmental outcomes after heart surgery. |
J. WILLIAM GAYNOR; | 2014-05-01 |
| 36 | Microcephaly Genetic Deficiency in Neural Progenitors: Genotyping, Phenotyping and Functional Neuro-anatomy and Neurobiology Comparative Primitive Microcephaly (MCPH) and The Fanconi Anemia (FA) PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: The purpose of this study is to: I. Compare neuroradiological phenotype and cognitive functioning of MCPH patients caused by ASPM mutations already characterized and published (Passemard et al. 2009a) with other MCPH-related patients (patients with MCPH1, WDR62, CDK5RAP2, CEP 152, CENPJ, STIL, or PCNT mutations) II. Describe the neuro-radiological and cognitive phenotype of microcephalic patients suffering from Fanconi anemia, and compared them to subjects with: – Fanconi anemia but normal OFC (head circumference) – MCPH patients – Healthy control subjects Our hypothesis is that mutations in genes responsible of microcephaly impact differentially cortical brain development and functioning |
ALAIN VERLOES; | 2013-10-01 |
| 37 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy Related Papers Related Patents Related Grants Related Experts View Highlight: Prospective study to compare the bone mineral density in adults with HPA on KUVAN™ therapy to those not on therapy. The investigators hypothesize that after one year of KUVAN™ therapy, there will be an improvement in their bone mineral density. |
HEATHER SAAVEDRA; | 2011-06-01 |
| 38 | A Double-blind, Placebo-controlled, Randomized Study to Evaluate The Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU. |
BIOMARIN PHARMACEUTICAL | 2010-08-01 |
| 39 | AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study Related Papers Related Patents Related Grants Related Experts View Highlight: The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+ stem cells) found in the peripheral blood of patients with Fanconi anemia. While AMD3100 has been used successfully in adult volunteers and cancer patients, it has not been used in children or patients with Fanconi anemia and in only a few children with cancer. Fanconi anemia is a rare genetic disease. Most Fanconi anemia patients eventually develop bone marrow failure, … |
STELLA DAVIES; | 2007-05-01 |
| 40 | Short-Term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome PF:2 Related Papers Related Patents Related Grants Related Experts View Highlight: This 10-week study will evaluate and compare behavior changes in children with Smith-Lemli-Opitz syndrome (SLOS) who are taking cholesterol supplementation versus those who are not on cholesterol supplementation. SLOS is a genetic disorder that affects the development of children both before and after birth. An enzyme deficiency in these children results in low levels of cholesterol, which can cause a variety of birth defects and behavioral problems. Typical abnormal physical features of patients include a small head, drooping eyelids, small upturned nose, small chin, cleft palate, heart defects, … |
FORBES PORTER; | 2005-06-01 |
| 41 | Clinical and Basic Investigations Into Smith-Lemli-Opitz Syndrome PF:3 Related Papers Related Patents Related Grants Related Experts View Highlight: Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder (autosomal recessive) caused by an abnormality in the production of cholesterol. The disorder can occur in both a mild or severe form. SLOS is associated with multiple birth defects and mental retardation. Some of the birth defects include; abnormal facial features, poor muscle tone, poor growth, shortened life span, and abnormalities of the heart, lungs, brain, gastrointestinal tract, limbs, genitalia, … |
SAMAR N RAHHAL; | 1998-09-13 |
| 42 | Trial of Indomethacin Prophylaxis in Preterm Infants (TIPP) PF:5 Related Papers Related Patents Related Grants Related Experts View Highlight: This trial was to determine whether giving low-dose indomethacin to infants weight 500 to 999 grams (approximately 1 to 2 pounds) at birth improves their survival without cerebral palsy or developmental problems at 18 to 22 months of age. |
DAVID K. STEVENSON; | 1993-11-01 |
| 43 | Effects of Maternal Phenylketonuria (PKU) on Pregnancy Outcome PF:4 Related Papers Related Patents Related Grants Related Experts View Highlight: Phenylketonuria (PKU) is a rare genetic condition. If not treated, PKU can cause severe mental retardation. Women with PKU are advised to eat a special diet when pregnant to prevent mental retardation in their children. This study will evaluate the effects of that diet on the children of mothers with PKU. |
RICHARD KOCH; | 1984-05-01 |