Treatment Trials

9 Clinical Trials for Various Conditions

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COMPLETED
Infant Aphakia Treatment Study (IATS)
Description

The primary purpose is to determine whether infants with a unilateral congenital cataract are more likely to develop better vision following cataract extraction surgery if they undergo primary implantation of an intraocular lens or if they are treated primarily with a contact lens. In addition, the study will compare the occurrence of postoperative complications and the degree of parental stress between the two treatments.

RECRUITING
The Myelin Disorders Biorepository Project
Description

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

Conditions
COMPLETED
LeukoSEQ: Whole Genome Sequencing As a First-Line Diagnostic Tool for Leukodystrophies
Description

Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes. While recent work has demonstrated that whole genome sequencing (WGS), has the potential to dramatically increase diagnostic efficiency, significant questions remain around the impact on downstream clinical management approaches versus standard diagnostic approaches.

COMPLETED
A Randomized Study in Children Ages Birth Through Three Years Undergoing Unilateral Cataract Extraction With or Without Lens Replacement
Description

The purpose of this study is to evaluate the effect of OMS302 compared to phenylephrine when administered in irrigation solution during cataract extraction on intraoperative pupil diameter, acute postoperative pain, and safety in children birth through 3 years.

SUSPENDED
Artisan Aphakia Lens for the Correction of Aphakia in Children
Description

The purpose of this study is to determine the safety and effectiveness of the Artisan Aphakia Lens in the treatment of aphakia in children.

Conditions
COMPLETED
Clinical and Genetic Studies of Families With Congenital or Hereditary Cataracts
Description

This study will explore the clinical and hereditary (genetic) features of inherited cataracts. A cataract is a clouding of the eye lens, which focuses light on the back of the eye. When the lens becomes cloudy and does not let light through, vision is impaired. Cataracts may be associated with vision problems alone, or with other problems, such as changes in facial appearance or skin problems. A better understanding of these genetic conditions may help in the development of better diagnostic tests. Patients with inherited cataracts and their family members may be eligible for this study. Participants will be drawn from patients enrolled in other studies of inherited cataracts at the NEI and collaborating clinics. Participants will undergo the following tests and procedures: * Medical and surgical history; * Verification of diagnosis; * Construction of a family tree regarding familial vision problems; * Complete eye examination, including dilation of the pupils and photography of the lens, tests of color vision and field of vision, and of the ability to see in the dark; * Blood sample collection (20 ml, or 4 teaspoons) for genetic studies of hereditary cataracts.

Conditions
COMPLETED
Studies of Families With Hereditary Cataracts
Description

The objective of this study is to discover the genes responsible for the development of hereditary cataracts in families. A cataract is clouding of the lens of the eye that obstructs the passage of light and may impair vision. Information from this study may provide a better understanding of why hereditary cataracts form and perhaps lead to the development of a test that can predict who will likely be affected and to what degree. Patients or family members of patients with inherited cataracts who participate in this study will be asked questions about their family history, especially concerning eye disease or cataracts, and a family tree will be drawn. They will undergo a complete eye examination, including photographs to document the clarity or opacity of the lens. In addition, a small blood sample will be drawn for use in gene mapping studies of inherited cataract.

RECRUITING
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
Description

The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle Disease International Registry (CMDIR) to acquire necessary data for adverse event calculations (intake survey and medical records curation). To support this study and become a participant, we ask that you register in the CMDIR. You can do this by visiting www.cmdir.org. There is no travel required. The registry includes affected individuals with congenital muscular dystrophy, congenital myopathy, and congenital myasthenic syndrome and registers through the late onset spectrum for these disease groups. The CMDIR was created to identify the global congenital muscle disease population for the purpose of raising awareness, standards of care, clinical trials and in the future a treatment or cure. Simply put, we will not be successful in finding a treatment or cure unless we know who the affected individuals are, what the diagnosis is and how the disease is affecting the individual. Registering in the CMDIR means that you will enter demographic information and complete an intake survey. We would then ask that you provide records regarding the diagnosis and treatment of CMD, including genetic testing, muscle biopsy, pulmonary function testing, sleep studies, clinic visit notes, and hospital discharge summaries. Study hypothesis: 1. To use patient and proxy reported survey answers and medical reports to build a longitudinal care and outcomes database across the congenital muscle diseases. 2. To generate congenital muscle disease subtype specific adverse event rates and correlate with key care parameters.

Conditions
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) DeficiencyAlpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations)Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy)Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C))Alpha-Dystroglycanopathy (Fukuyama CMD)Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I))Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K))Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M))Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N))Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O))Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T))Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U))Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related)Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB))Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS))Choline Kinase B Receptor - CHKBCollagen VI Related DisordersCollagen XII Related DisordersCongenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive)Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID)Congenital Muscular Dystrophy With Joint HyperlaxityCongenital Muscular Dystrophy With Rigid Spine Related to ACTA1Emery-Dreifuss Muscular DystrophyGOLGA2-related Congenital Muscle Dystrophy With Brain InvolvementLMNA Related DisordersMerosin Deficient CMD (Full or Partial)Nesprin Related MD (SYNE1)SELENON Related Disorders (Previously Known as SEPN1)SELENON Related Myopathy (Aka SEPN1)Telethonin CMDCongenital Myasthenic SyndromeLimb-Girdle Muscular DystrophyLGMDD01 - DNAJB6 (Formerly LGMD1D)LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant)LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G)LGMDR08 - TRIM Related (Formerly LGMD2H)LGMDR09 - FKRP Related (Formerly LGMD2I)LGMDR10 - Titin (TTN) Related (Formerly LGMD2J)LGMDR11 - POMT1 Related (Formerly LGMD2K)LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M)LGMDR14 - POMT2 Related (Formerly LGMD2N)LGMDR15 - POMGnT1 Related (Formerly LGMD2O)LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P)LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q)LGMDR18 - TRAPPC11 Related (Formerly LGMD2S)LGMDR19 - GMPPB Related (Formerly LGMD2T)LGMDR20 - ISPD Related (Formerly LGMD2U)LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive)LGMDR23 - LAMA2 RelatedLGMDR24 - POMGnT2 Related
RECRUITING
Determination of the Sensitivity and Specificity of a Smartphone Application to Detect Retinoblastoma
Description

This study seeks to determine whether a smartphone application called CRADLE (ComputeR Assisted Detection of LEukocoria) has the potential to improve the detection of leukocoria. There will be no impact on participants' health outcome. This study will be performed in two parts, each with a distinct cohort of patients. Part 1 will assess the feasibility of various techniques/conditions for using CRADLE within patients known to have leukocoria. Part 2 will estimate the sensitivity and specificity of CRADLE to detect leukocoria (using the techniques selected from information gathered in Part 1) as compared to an ophthalmoscope, within patients referred to the clinic for suspected leukocoria. PRIMARY OBJECTIVES: * To determine the most effective usage of a camera phone application (CRADLE) to maximize detection of leukocoria in patients with retinoblastoma, congenital cataracts, and glaucoma. * To estimate the sensitivity and specificity of a camera phone application (CRADLE) in detecting leukocoria.