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Congenital Disorders of Glycosylation

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Congenital Pediatric Disorders Trial in Houston (Busulfan, Cytoxan, Fludarabine)

Active, not recruiting
  • Congenital Pediatric Disorders
  • Houston, Texas
    Texas Children's Hospital
Feb 4, 2022

Neuroradiology Assesses Chiari Malformation's Impact on Airways,

Recruiting
  • Malformation Brain
  • Magnetic Resonance
  • Polysomnographic evaluation
  • Rome, Italy
    Fondazione Policlinico Gemelli IRCCS
Jun 18, 2023

Anesthesia; Adverse Effect, Congenital Heart Disease, Neurodevelopmental Disorders Trial in Palo Alto (Isoflurane, Fentanyl

Completed
  • Anesthesia; Adverse Effect
  • +2 more
  • Palo Alto, California
    Stanford Childrens hospital
May 6, 2022

Congenital Heart Disease in Children, Post-cardiac Surgery, Malnutrition, Infant Trial in Jeddah (Energy- Protein Enriched

Recruiting
  • Congenital Heart Disease in Children
  • +2 more
  • Energy- Protein Enriched Nutritional Formula
  • Jeddah, Outside North America, Saudi Arabia
    KFSHRC
Apr 12, 2023

Birth Defects, Multiple Congenital Anomaly, Neurodevelopmental Disorders Trial in Edinburg (Whole genome sequencing (WGS))

Recruiting
  • Birth Defects
  • +2 more
  • Whole genome sequencing (WGS)
  • Edinburg, Texas
    University of Texas Rio Grande Valley
Apr 10, 2022

Neurodevelopmental Disorder in Deaf Infants Under Ten Months of

Not yet recruiting
  • Congenital Sensorineural Hearing Loss
    • (no location specified)
    Jun 17, 2022

    Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary Trial in United States (EDIT-101)

    Active, not recruiting
    • Leber Congenital Amaurosis 10
    • +6 more
    • Miami, Florida
    • +4 more
    Dec 2, 2022

    Olfactory Disorders and Performance ofNew Olfactory Test in

    Completed
    • Congenital Cytomegalovirus Infection
    • olfactory tests, otoacoustic emissions (OAE)
    • Le Kremlin-Bicêtre, France
    • +1 more
    Jul 26, 2021

    Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis Trial in Worldwide (sepofarsen, Sham)

    Active, not recruiting
    • Leber Congenital Amaurosis 10
    • +9 more
    • Iowa City, Iowa
    • +13 more
    Mar 2, 2022

    Congenital Antithrombin Deficiency Trial in Worldwide (Atenativ)

    Recruiting
    • Congenital Antithrombin Deficiency
    • Washington, District of Columbia
    • +13 more
    Sep 16, 2022

    Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies Trial run by the

    Not yet recruiting
    • Inherited Bone Marrow Failure Syndrome
    • Familial Platelet Disorder With Predisposition to Myeloid Malignancies
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Oct 20, 2023

    Preterm Birth, Pediatric Cancer, Hirschsprung Disease Trial in Copenhagen, Hillerød, Lund (e-health device with application)

    Enrolling by invitation
    • Preterm Birth
    • +5 more
    • e-health device with application
    • Copenhagen, Denmark
    • +5 more
    Dec 7, 2022

    Rare Diseases, Congenital Disorders Trial in Roma (voice recordings)

    Not yet recruiting
    • Rare Diseases
    • Congenital Disorders
    • voice recordings
    • Roma, Italy
      Fondazione Policlinico Universitario A. Gemelli Irccs
    Jul 19, 2023

    Prader-Willi Syndrome, Aggression, Self-Injurious Behavior Trial in Brooklyn (Guanfacine Extended Release, Placebo)

    Recruiting
    • Prader-Willi Syndrome
    • +20 more
    • Guanfacine Extended Release
    • Placebo
    • Brooklyn, New York
      Maimonides Medical Center
    Dec 20, 2022

    Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1 Trial in Oakland (Patient-entered data)

    Recruiting
    • Myotonic Dystrophy
    • +15 more
    • Patient-entered data
    • Oakland, California
      Myotonic Dystrophy Foundation
    Oct 31, 2022

    Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis Trial in Iowa City, Philadelphia, Ghent (QR-110)

    Active, not recruiting
    • Leber Congenital Amaurosis 10
    • +9 more
    • Iowa City, Iowa
    • +2 more
    Feb 8, 2022

    Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

    Recruiting
    • Primary Immunodeficiency (PID)
    • +4 more
    • data collection
    • Pittsburgh, Pennsylvania
      UPMC Children's Hospital of Pittsburgh
    Feb 9, 2022

    Bullosa Epidermolysis, Vitamin D Deficiency, Osteoporosis Trial in Moscow (Vitamin D, Oral nutritional supplement)

    Recruiting
    • Bullosa Epidermolysis
    • +4 more
    • Vitamin D
    • Oral nutritional supplement
    • Moscow, Russian Federation
      National Medical Research Center for Children's Health
    Dec 2, 2021

    Cancer in Inherited Bone Marrow Failure Syndromes

    Recruiting
    • Diamond Blackfan Anemia
    • +4 more
      • Bethesda, Maryland
      • +1 more
      Jan 24, 2023

      Natural History Study of CEP290-Related Retinal Degeneration

      Completed
      • Blindness
      • +7 more
        • Miami, Florida
        • +6 more
        May 18, 2022

        Evaluating Prenatal Exome Sequencing Study

        Enrolling by invitation
        • Congenital Anomalies
        • +2 more
          • Leiden, Zuid-Holland, Netherlands
            Leiden University Medical Centre
          Mar 21, 2022

          TAAI Erasmus Research Initiative to Fight CF: Monitoring

          Recruiting
          • Cystic Fibrosis
          • +9 more
            • Rotterdam, Zuid-Holland, Netherlands
              Erasmus MC - Sophia Children's Hospital
            Feb 21, 2023

            Childhood Outcomes of Preterm Brain Abnormalities

            Recruiting
            • Premature Birth
            • +3 more
            • MRI
            • Neurodevelopmental/Neuropsychological Assessment
            • Los Angeles, California
              Children's Hospital Los Angeles
            Jun 13, 2022

            Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies Trial in Paris (Lower-limb powered dermoskeleton)

            Recruiting
            • Muscular Dystrophies
            • +4 more
            • Lower-limb powered dermoskeleton
            • Paris, France
              Institute of Myology
            Mar 28, 2022

            Anxiety, Stress, Satisfaction Trial in Kocasi?nan (Progressive Relaxation Exercises)

            Not yet recruiting
            • Anxiety
            • +4 more
            • Progressive Relaxation Exercises
            • Kocasi̇nan, Kayseri, Turkey
              Didem Kaya
            Mar 2, 2023