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38 Companies |
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35 Products |
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35 Products |
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54 Mechanisms of Action |
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3 Trials |
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404 News |
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- Trial completion: Studies of Heritable Disorders of Connective Tissue (clinicaltrials.gov) - Dec 17, 2013
P=N/A, N=1335, Completed, Sponsor: National Institute on Aging (NIA)
Recruiting --> Completed
- | beloranib (ZGN-440) / Larimar Therap
Trial completion: An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome (clinicaltrials.gov) - Dec 16, 2013
P2, N=17, Completed, Sponsor: Zafgen, Inc.
Recruiting --> Completed Active, not recruiting --> Completed
- oxytocin / Generic mfg.
New P1 trial: Oxytocin Trial in Prader-Willi Syndrome (clinicaltrials.gov) - Dec 15, 2013
P1, N=24, Not yet recruiting, Sponsor: University of Florida
- Enrollment change: A Long-Term Study of Cholesterol Supplements for Smith-Lemli-Opitz Syndrome (clinicaltrials.gov) - Dec 11, 2013
P=N/A, N=8, Completed, Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Active, not recruiting --> Completed N=25 --> 8
- Trial completion: A Long-Term Study of Cholesterol Supplements for Smith-Lemli-Opitz Syndrome (clinicaltrials.gov) - Dec 11, 2013
P=N/A, N=8, Completed, Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
N=25 --> 8 Recruiting --> Completed
- Trial completion: Role of Pseudogene in Incontinentia Pigmenti, and Its Potential Treatment (clinicaltrials.gov) - Dec 2, 2013
P=N/A, N=50, Completed, Sponsor: National Taiwan University Hospital
Recruiting --> Completed Recruiting --> Completed
- Enrollment change: Phenotypic Properties in Individuals Affected With XLHED (clinicaltrials.gov) - Nov 24, 2013
P=N/A, N=30, Completed, Sponsor: Edimer Pharmaceuticals
Recruiting --> Completed N=150 --> 30
- | Trial completion: Phenotypic Properties in Individuals Affected With XLHED (clinicaltrials.gov) - Nov 24, 2013
P=N/A, N=30, Completed, Sponsor: Edimer Pharmaceuticals
N=150 --> 30 Recruiting --> Completed
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
Trial completion: A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200 (clinicaltrials.gov) - Nov 24, 2013
P1, N=6, Completed, Sponsor: Edimer Pharmaceuticals
Recruiting --> Completed Recruiting --> Completed
- Enrollment closed: GenTAC: National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (clinicaltrials.gov) - Nov 13, 2013
P=N/A, N=3800, Active, not recruiting, Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Recruiting --> Completed Recruiting --> Active, not recruiting
- | basmisanil (RG1662) / Roche
Trial completion: A Study of RG1662 in Individuals With Down Syndrome (clinicaltrials.gov) - Nov 12, 2013
P1, N=35, Completed, Sponsor: Hoffmann-La Roche
Recruiting --> Active, not recruiting Recruiting --> Completed
- Trial initiation date: Study of the Efficacy of New Non-invasive Prenatal Tests for Screening for Fetal Trisomies Using Maternal Blood (clinicaltrials.gov) - Nov 6, 2013
P=N/A, N=5600, Recruiting, Sponsor: Centre Hospitalier Universitaire de Qu
Recruiting --> Completed Initiation date: Aug 2013 --> Nov 2013
- Enrollment open: Study of the Efficacy of New Non-invasive Prenatal Tests for Screening for Fetal Trisomies Using Maternal Blood (clinicaltrials.gov) - Nov 6, 2013
P=N/A, N=5600, Recruiting, Sponsor: Centre Hospitalier Universitaire de Qu
Initiation date: Aug 2013 --> Nov 2013 Not yet recruiting --> Recruiting
- basmisanil (RG1662) / Roche
Enrollment change: A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662 (clinicaltrials.gov) - Nov 4, 2013
P1, N=13, Completed, Sponsor: Hoffmann-La Roche
Not yet recruiting --> Recruiting N=17 --> 13
- | basmisanil (RG1662) / Roche
Trial completion: A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662 (clinicaltrials.gov) - Nov 4, 2013
P1, N=13, Completed, Sponsor: Hoffmann-La Roche
N=17 --> 13 Recruiting --> Completed
- | intranasal carbetocin (ACP-101) / Acadia Pharma
New P2 trial: Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome (clinicaltrials.gov) - Oct 21, 2013
P2, N=38, Recruiting, Sponsor: Ferring Pharmaceuticals
- ||| basmisanil (RG1662) / Roche
Enrollment open: A Screening Protocol to Assess Adults and Adolescents With Down Syndrome for Eligibility For Upcoming Study of RG1662 (Study BP27832) (clinicaltrials.gov) - Oct 6, 2013
P=N/A, N=170, Recruiting, Sponsor: Hoffmann-La Roche
Recruiting --> Completed Not yet recruiting --> Recruiting
- MK-2206 / Merck (MSD)
Trial completion: MK2206 in Treating Younger Patients With Recurrent or Refractory Solid Tumors or Leukemia (clinicaltrials.gov) - Sep 26, 2013
P1, N=45, Completed, Sponsor: National Cancer Institute (NCI)
Not yet recruiting --> Recruiting Active, not recruiting --> Completed
- Enrollment open: Defining Immunodeficiency in Heterotaxy Syndrome: Pilot Study Data (clinicaltrials.gov) - Aug 26, 2013
P=N/A, N=10, Recruiting, Sponsor: Children's Hospital Boston
Active, not recruiting --> Completed Not yet recruiting --> Recruiting
- cytarabine / Generic mfg.
Enrollment change: Low-Dose Cytarabine in Treating Infants With Down Syndrome and Transient Myeloproliferative Disorder (clinicaltrials.gov) - Aug 21, 2013
P3, N=0, Withdrawn, Sponsor: Children's Oncology Group
Not yet recruiting --> Recruiting N=180 --> 0
- Enrollment change: Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia (clinicaltrials.gov) - Aug 20, 2013
P3, N=25, Active, not recruiting, Sponsor: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
N=180 --> 0 N=280 --> 25
- || Enrollment change: Non-invasive Chromosomal Examination of Trisomy Study (clinicaltrials.gov) - Aug 14, 2013
P=N/A, N=18955, Active, not recruiting, Sponsor: Ariosa Diagnostics, Inc
N=280 --> 25 N=25000 --> 18955
- || Enrollment closed: Non-invasive Chromosomal Examination of Trisomy Study (clinicaltrials.gov) - Aug 14, 2013
P=N/A, N=18955, Active, not recruiting, Sponsor: Ariosa Diagnostics, Inc
N=25000 --> 18955 Recruiting --> Active, not recruiting
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment open: IMAGO: Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (clinicaltrials.gov) - Aug 13, 2013
P2, N=18, Recruiting, Sponsor: Lumena Pharmaceuticals, Inc.
Recruiting --> Active, not recruiting Not yet recruiting --> Recruiting
- Enrollment closed: Cognitive Remediation in 22q11DS (clinicaltrials.gov) - Aug 4, 2013
P=N/A, N=36, Active, not recruiting, Sponsor: Duke University
Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting
- Byetta (exenatide) / AstraZeneca
Enrollment closed: Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome (clinicaltrials.gov) - Jul 24, 2013
P=N/A, N=20, Active, not recruiting, Sponsor: Children's Hospital Los Angeles
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
New P2 trial: IMAGO: Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (clinicaltrials.gov) - Jul 17, 2013
P2, N=18, Recruiting, Sponsor: Lumena Pharmaceuticals, Inc.
- Enrollment open: Hypoglycemia in Prader-Willi Syndrome (clinicaltrials.gov) - Jul 14, 2013
P=N/A, N=20, Recruiting, Sponsor: University of Florida
Recruiting --> Active, not recruiting Not yet recruiting --> Recruiting
- Trial termination: Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States (clinicaltrials.gov) - Jul 11, 2013
P=N/A, N=119, Terminated, Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Not yet recruiting --> Recruiting Recruiting --> Terminated
- Enrollment change: Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States (clinicaltrials.gov) - Jul 11, 2013
P=N/A, N=119, Terminated, Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Recruiting --> Terminated N=600 --> 119
- ||| Trial completion: Comparison of Aneuploidy Risk Evaluations (clinicaltrials.gov) - Jul 11, 2013
P=N/A, N=3000, Completed, Sponsor: Verinata Health, Inc.
N=600 --> 119 Recruiting --> Completed
- ||| Trial completion: X-CORS: X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Carrier Outlook Toward Reproduction Survey (clinicaltrials.gov) - Jul 6, 2013
P=N/A, N=500, Completed, Sponsor: Edimer Pharmaceuticals
Recruiting --> Completed Recruiting --> Completed
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
Enrollment open, IO biomarker: Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (clinicaltrials.gov) - Jul 2, 2013
P2, N=6, Recruiting, Sponsor: Edimer Pharmaceuticals
Recruiting --> Completed Active, not recruiting --> Recruiting
- Odomzo (sonidegib) / Sun Pharma
Enrollment change: Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS) (clinicaltrials.gov) - Jun 25, 2013
P2, N=10, Completed, Sponsor: Novartis Pharmaceuticals
Active, not recruiting --> Recruiting N=42 --> 10
- ||||| thioguanine / Generic mfg.
Enrollment change: Chemotherapy in Treating Children With Down Syndrome and Myeloproliferative Disorder, Acute Myelogenous Leukemia, or Myelodysplastic Syndrome (clinicaltrials.gov) - Jun 25, 2013
P3, N=254, Completed, Sponsor: Children's Oncology Group
N=42 --> 10 N=158 --> 254
- Odomzo (sonidegib) / Sun Pharma
Trial completion: Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS) (clinicaltrials.gov) - Jun 25, 2013
P2, N=10, Completed, Sponsor: Novartis Pharmaceuticals
N=158 --> 254 Recruiting --> Completed
- Trial completion: Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies (clinicaltrials.gov) - Jun 19, 2013
P1/2, N=5, Completed, Sponsor: University Hospital, Toulouse
Recruiting --> Completed Active, not recruiting --> Completed
- Enrollment change: Assessment of Bone Density and Bone Turnover Markers in Patients With Down Syndrome and Comparison to the Ts65Dn Model (clinicaltrials.gov) - Jun 18, 2013
P=N/A, N=60, Active, not recruiting, Sponsor: University of Arkansas
Active, not recruiting --> Completed N=30 --> 60
- topiramate / Generic mfg.
Trial completion: Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome (clinicaltrials.gov) - Apr 29, 2013
P=N/A, N=10, Completed, Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
N=30 --> 60 Recruiting --> Completed
- Trial completion: Informed Consent for Whole Genome Sequencing: Ideals and Norms Referenced by Early Participants (clinicaltrials.gov) - Mar 29, 2013
P=N/A, N=40, Completed, Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting --> Completed Recruiting --> Completed
- | beloranib (ZGN-440) / Larimar Therap
New P2 trial: An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome (clinicaltrials.gov) - Mar 25, 2013
P2, N=17, Completed, Sponsor: Zafgen, Inc.
- | dexamethasone / Generic mfg., lenalidomide / Generic mfg.
New P2 trial: Study to Evaluate Lenalidomide Plus Dexamethasone in Patients With Newly Diagnosed POEMS Syndrome (clinicaltrials.gov) - Mar 20, 2013
P2, N=50, Recruiting, Sponsor: Peking Union Medical College Hospital
- ||||| Erivedge (vismodegib) / Roche
New P2 trial: A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas (clinicaltrials.gov) - Mar 19, 2013
P2, N=200, Recruiting, Sponsor: Hoffmann-La Roche
- Enrollment open: Aortopathy in Persons With Bicuspid Aortic Valve, Turner and Marfan Syndrome (clinicaltrials.gov) - Mar 17, 2013
P=N/A, N=60, Recruiting, Sponsor: University of Aarhus
Recruiting --> Completed Not yet recruiting --> Recruiting
- Clinical: Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome (clinicaltrials.gov) - Mar 7, 2013
P=N/A, N=87, Recruiting, Sponsor: Children's Hospital of Philadelphia
- Enrollment change: Examining Social, Emotional, and Cognitive Functioning in People With Fragile X and Down Syndromes (clinicaltrials.gov) - Mar 4, 2013
P=N/A, N=511, Completed, Sponsor: University of California, Davis
Not yet recruiting --> Recruiting N=320 --> 511
- Trial completion: Examining Social, Emotional, and Cognitive Functioning in People With Fragile X and Down Syndromes (clinicaltrials.gov) - Mar 4, 2013
P=N/A, N=511, Completed, Sponsor: University of California, Davis
N=320 --> 511 Recruiting --> Completed
- losartan / Generic mfg., atenolol / Generic mfg.
Enrollment change: Effects of Losartan Versus Atenolol on Aortic and Cardiac Muscle Stiffness in Adults With Marfan Syndrome (clinicaltrials.gov) - Feb 3, 2013
P3, N=39, Completed, Sponsor: Brigham and Women's Hospital
Recruiting --> Completed N=50 --> 39
- losartan / Generic mfg., atenolol / Generic mfg.
Trial completion: Effects of Losartan Versus Atenolol on Aortic and Cardiac Muscle Stiffness in Adults With Marfan Syndrome (clinicaltrials.gov) - Feb 3, 2013
P3, N=39, Completed, Sponsor: Brigham and Women's Hospital
N=50 --> 39 Recruiting --> Completed
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
New P2 trial, IO biomarker: Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (clinicaltrials.gov) - Jan 23, 2013
P2, N=6, Recruiting, Sponsor: Edimer Pharmaceuticals