Clinical Trials on Congenital Sucrase-isomaltase Deficiency (CSID)

Total 368 results

QOL Medical, LLC

Recruiting

7-Day Trial of Sucraid for Alleviating CSID Symptoms in Subjects With Low, Moderate, and Normal Sucrase Levels

Congenital Sucrase-Isomaltase Deficiency | CSID | Sucrase Isomaltase Deficiency

United States
QOL Medical, LLC
Johns Hopkins University; Massachusetts General Hospital; Baylor College of Medicine and other collaborators

Completed

Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS) (CSID GPS)

Congenital Sucrase-isomaltase Deficiency (CSID)

United States
Steno Diabetes Center Copenhagen
The Novo Nordisk Foundation Center for Basic Metabolic Research; Department... and other collaborators

Completed

The Effect on Metabolism, Food Intake and Preferences of a Knockout Gene Variant Involved in Carbohydrate Metabolism

Diabetes Mellitus, Type 2 | Metabolic Disease | Sucrase Isomaltase Deficiency | Sucrose Intolerance Congenital

Greenland
QOL Medical, LLC

Completed

Adult Pilot Study for Reference Ranges and Optimal Cut-offs for the Sucrose Challenge Test and Sucrose Breath Test

Congenital Sucrase-Isomaltase Deficiency

United States
Lovisenberg Diakonale Hospital
University of Bergen

Not yet recruiting

Sucrase-isomaltase Deficiency as a Cause of Irritable Bowel Syndrome

Irritable Bowel Syndrome | Functional Gastrointestinal Disorders | Sucrase Isomaltase Deficiency | Sucrose Intolerance Due to Sucrase-Isomaltase Deficiency | Carbohydrate; Malabsorption
Nottingham University Hospitals NHS Trust
CICbioGUNE; Christian-Albrechts-University of Kiel; University of Veterinary...

Not yet recruiting

Genetic Carbohydrate Maldigestion as a Model to Study Food Hypersensitivity (GenMalCarb)

Irritable Bowel Syndrome (IBS) | Sucrase Isomaltase Deficiency
QOL Medical, LLC

Withdrawn

A Trial to Evaluate the Frequency of Genetic Sucrase-Isomaltase Deficiency Genotypes, and the Efficacy and Safety of Sucraid® (Sacrosidase) Oral Solution in Subjects With Chronic Diarrhea and Sucrase Deficiency (GSID-E)

Genetic Sucrase-Isomaltase Deficiency
Region Skane

Enrolling by invitation

The Effect of Sugar Load in IBS Patients Depending on Sucrase-isomaltase Genes

Irritable Bowel Syndrome | Sucrase-Isomaltase Deficiency

Sweden
National Human Genome Research Institute (NHGRI)
National Institutes of Health Clinical Center (CC); National Cancer Institute... and other collaborators

Completed

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

ADA-SCID | Adenosine Deaminase Deficiency

United States
University Hospital, Strasbourg, France

Completed

Severe PID With Lymphoproliferation and Neutropenia (DICEP)

Primary Immune-Deficiency (PID) Common Variable Immune Deficiency (CVID)

France
National Institute of Allergy and Infectious Diseases...
National Center for Advancing Translational Sciences (NCATS); Office of Rare... and other collaborators

Enrolling by invitation

Natural History Study of SCID Disorders

Omenn Syndrome | Reticular Dysgenesis | Severe Combined Immunodeficiency (SCID) | XSCID | Leaky SCID | ADA SCID

United States, Canada
National Institute of Allergy and Infectious Diseases...
National Center for Advancing Translational Sciences (NCATS); Primary Immune...

Enrolling by invitation

Patients Treated for SCID (1968-Present)

ADA-SCID | Omenn Syndrome | SCID | Reticular Dysgenesis | XSCID | Leaky SCID

United States, Canada
National Institute of Allergy and Infectious Diseases...

Completed

Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency

CVID Enteropathy | Gastrointestinal Inflammation Associated With CVID

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Immune Regulation in Patients With Common Variable Immunodeficiency and Related Syndromes

CVID | XLA

United States
Oslo University Hospital

Completed

The Rifaximin Study in CVID

Common Variable Immunodeficiency (CVID)

Norway
Centers for Disease Control and Prevention

Completed

Limb Loss Self-Management Program

Congenital or Acquired Limb Deficiency

United States
Novartis Pharmaceuticals

Completed

Study of Efficacy of CDZ173 in Patients With APDS/PASLI

Common Variable Immunodeficiency (CVID), APDS / PASLI

Germany, Russian Federation, Ireland, Italy, United Kingdom, Belarus, Netherlands, Czechia, United States
National Institute of Allergy and Infectious Diseases...

Enrolling by invitation

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

Severe Combined Immune Deficiency (SCID)

United States
Leadiant Biosciences, Inc.

Completed

EZN-2279 in Patients With ADA-SCID

Severe Combined Immunodeficiency | ADA-SCID | Adenosine Deaminase Deficiency

United States
Great Ormond Street Hospital for Children NHS Foundation...
Orchard Therapeutics

Completed

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Adenosine Deaminase Deficiency | Severe Combined Immunodeficiencies (SCID)

United Kingdom
University of California, Los Angeles
Great Ormond Street Hospital for Children NHS Foundation Trust

Enrolling by invitation

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Adenosine Deaminase Deficiency | Severe Combined Immunodeficiency (SCID)

United States, United Kingdom
Jasper Therapeutics, Inc.

Recruiting

JSP191 Antibody Targeting Conditioning in SCID Patients

SCID

United States
National Cardiovascular Center Harapan Kita Hospital...

Unknown

Thiamine Influenced on Substrate Energy Effectiveness in Indonesian Children Undergoing Cardiopulmonary Bypass

Cardiopulmonary Bypass | Congenital Heart Disease | Thiamine Deficiency

Indonesia
Children's Hospital of Eastern Ontario
McGill University; Ottawa Hospital Research Institute; The Ottawa Hospital; Children...

Completed

Prevention of Vitamin D Deficiency Following Pediatric CHD Surgery: a Phase II Dose Evaluation Randomized Controlled Trial Comparing Usual Care With a High Dose Pre-operative Supplementation Regimen Based on the Institute of Medicine Daily Upper Tolerable Intake Level (HICCUPS 2)

Vitamin D Deficiency | Heart Defects, Congenital | Thoracic Surgery | Pediatric Disorders

Canada
Queen Fabiola Children's University Hospital

Recruiting

Iron Deficiency in Pediatric Heart Surgery

Iron Deficiency Anemia | Congenital Heart Disease in Children | Cardiac Surgical Procedures | Iron Deficiencies

Belgium
Duke University
National Institute of Allergy and Infectious Diseases (NIAID); University of... and other collaborators

Recruiting

COVID-19 Vaccine Responses in PIDD Subjects

Common Variable Immunodeficiency | X-linked Agammaglobulinemia | Primary Immune Deficiency | Primary Antibody Deficiencies | CVID | XLA | Secondary Hypogammaglobulinemia

United States
University of Minnesota

Recruiting

Treatment of LSCD With DM

Limbal Stem-cell Deficiency | Congenital Aniridia

United States
Northwestern University

Completed

The Effects of Metreleptin in Congenital Leptin Deficiency

Congenital Leptin Deficiency (Disorder)

United States
Maggie's Pearl, LLC

Active, not recruiting

Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

Pmm2-CDG | Phosphomannomutase 2 Deficiency | Phosphomannomutase 2 Congenital Disorder of Glycosylation | Phosphomannomutase II Congenital Disorder of Glycosylation | Phosphomannomutase II Deficiency

United States
Pfizer

Completed

Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe

Factor VIII Deficiency, Congenital | Hemophilia A, Congenital | Factor 8 Deficiency, Congenital | Autosomal Hemophilia A | Classic Hemophilia
Shenzhen Geno-Immune Medical Institute

Unknown

Gene Transfer for ADA-SCID Using an Improved Lentiviral Vector (TYF-ADA)

Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)

China
AIO-Studien-gGmbH
Takeda; Prof. Hartmut Link; MMF GmbH; AIO AG Supportivtherapie; AGSMO (Arbeitsgemeinschaft...

Completed

Quality Assurance of Secondary Immunodeficiencies (SID) in CLL/MM Patients (QS-SID)

Secondary Immunodeficiencies (SID)

Germany
Universitas Sumatera Utara

Not yet recruiting

The Role of Thiamine After Transcatheter Closure in Children With Left-to-Right Shunt Congenital Heart Disease

Atrial Septal Defect | Congenital Heart Disease | Patent Ductus Arteriosus | Ventricular Septal Defect | Thiamine Deficiency

Indonesia
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients (HTLP Necker)

Pediatric Patients | Any Type of Severe Combined Immunodeficiency (SCID) | Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

France
National Human Genome Research Institute (NHGRI)

Active, not recruiting

Molecular and Clinical Studies of Primary Immunodeficiency Diseases

Wiskott- Aldrich Syndrome | ADA Deficient SCID

United States
Paul Szabolcs

Enrolling by invitation

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases (BOLT+BMT)

Chronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...

United States
University of Alabama at Birmingham

Completed

Feasibility, Acceptability and Directional Signal Effect on Blood Folate Levels of Iodized Salt Fortified With Folic Acid: Clinical Study (FASALT)

Folic Acid Deficiency | Neural Tube Defects | Fortification

United States
Octapharma

Recruiting

Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Congenital Antithrombin Deficiency

Spain, United States, Italy, France, Hungary, Austria, Germany, United Kingdom
Kyowa Hakko Kirin UK, Ltd.

Completed

A Study of KW-3357 in Congenital Antithrombin Deficiency

Congenital Antithrombin Deficiency

France, Germany, Italy, Sweden, United Kingdom
National Human Genome Research Institute (NHGRI)

Withdrawn

Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents

ADA-SCID
Cure CMD
Congenital Muscle Disease International Registr

Withdrawn

LAMA2-related Muscular Dystrophy Brain Study

LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)

United States
Laboratoire français de Fractionnement et de Biotechnologies

Completed

Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency

Hypofibrinogenemia, Congenital | Afibrinogenemia, Congenital

France, Lebanon, Morocco, Turkey
The University of Hong Kong
Queen Mary Hospital, Hong Kong

Not yet recruiting

Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait

Thalassemia | Folic Acid Deficiency Anemia
Novo Nordisk A/S

Completed

Observational Study on Safety and Efficacy of NovoSeven® in Subjects With Congenital FVII Deficiency

Congenital Bleeding Disorder | Congenital FVII Deficiency

Japan
Novo Nordisk A/S

Enrolling by invitation

A Study of Safety and Effectiveness of NovoThirteen® (rFXIII) During Treatment of Congenital FXIII Deficiency in Japan

Congenital Bleeding Disorder | Congenital FXIII Deficiency

Japan
Novo Nordisk A/S

Completed

Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study (mentor™6)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

Spain, United States, Canada, Italy, United Kingdom, Hungary
Novo Nordisk A/S

Completed

Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (mentor™5)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom, Israel, United States
Novo Nordisk A/S

Completed

Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency (mentor™4)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United States, Israel, United Kingdom
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of Recombinant Factor XIII in Healthy Volunteers

Healthy | Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of Recombinant Factor XIII Administration in Healthy Volunteers

Healthy | Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom

Clinical Trials on Congenital Sucrase-isomaltase Deficiency (CSID)

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