N/A N=53 Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)
Congenital Sucrase-isomaltase Deficiency (CSID)
Primary: Prevalence of CSID Genetic Variants — 27; 0 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=5 CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Primary: Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) — 2; 0 Participants
N/A N=43 Clinical Utility of Handheld Hydrogen Breathalyzer in Identification of Food Sensitivities (AIRE Study)
SIBO · Small Bowel Bacterial Overgrowth Syndrome
Primary: Hydrogen Content in Parts Per Million (Ppm) — 3.5 ppm
Phase 2 N=45 Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
GLUT1DS1 · Epilepsy · Glut1 Deficiency Syndrome 1, Autosomal Recessive
Primary: Neuropsychological Score of Sustained Attention — 11 Participants
Phase 3 N=41 A Double-blind, Randomized, Multicenter, Cross-over Study to Compare the Effect of Creon N and Creon® on Fat Digestion in Subjects ≥ 12 Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Primary: Coefficient of Fat Absorption (CFA) — 88.1; 89.5 percentage of fat intake
Phase 1 N=14 Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
N/A N=50 Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
Inflammatory Bowel Disease · Glycogen Storage Disease Type Ia
Primary: Serologic, Genetic and Inflammatory Markers Consistent With Inflammatory Bowel Disease — 11 participants testing positive
Phase 3 N=12 Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Primary: Mean Intravenous Glucose Infusion Rate — 4.33; 9.51 mg/kg/minute — p=0.0037
Phase 2 N=12 Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Phase 2 N=6 A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
Phase 2 N=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Phase 3 N=32 A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Primary: Hypoglycemia Episode Rate — 5.29; 5.85; -3.05; -3.15 episodes per week — p=0.5028
Phase 2 N=6 A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 53; 128; 0; 1 number of events
N/A N=40 The Effect of CSP01 on Chronic Idiopathic Constipation and Irritable Bowel Syndrome With Constipation
Constipation · Chronic Idiopathic Constipation · Irritable Bowel Syndrome With Constipation
Primary: Change From Baseline in Colonic Transit Time (CTT) — -727; -15; 366 minutes
N/A N=6,623 Continuos Subcutaneous Insulin Infusion in Italy
Type 1 Diabetes
Primary: Efficacy Assessed by Median of HbA1c, — 60 mmol/mol
N/A N=402 Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
Cystic Fibrosis
Primary: Number of Participants Who Fully Completed at Least One ePRO — 133; 196; 130; 194 Participants
Phase 2 N=23 An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Primary: Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT) — 81.7; 72.9; 83.4…
Phase 2 N=20 An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Primary: Reported Change in Seizures Frequency From Baseline at 13 Weeks — 4.4; 189.5 seizures/two weeks
Phase 2 N=15 Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
Cystic Fibrosis · Exocrine Pancreatic Insufficiency
Primary: The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) — 6.9 % CFA — p=0.2179
N/A N=7 Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome
Focal Segmental Glomerulosclerosis · Steroid Resistant Nephrotic Syndrome
Primary: Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) — 0.69; 0.35 Palb — p=0.009
Phase 2 N=9 Metabolic Balance Study of Apraglutide in Patients With Short Bowel Syndrome, Intestinal Failure (SBS-IF) and Colon-in-Continuity (CIC)
Short Bowel Syndrome
Primary: Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) — 9; 5; 3; 1 Participants
Phase 2 N=3 Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency
Gastrointestinal Inflammation Associated With CVID · CVID Enteropathy
Primary: Number of Participants With Increase in Infection or Serious Adverse Events (SAEs) Related to Single Dose Administration of Ustekinumab — 0 participants
Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Phase 2 N=12 The Beta Cell Responsiveness to Glucose-dependent Insulinotropic Polypeptide (GIP) With and Without Sulfonylurea in Patients With Type 2 Diabetes
Diabetes Mellitus, Type 2
Primary: Insulin Secretion — 56766 pmol/l * 90 minutes
Phase 3 N=1,538 Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease
Iron Deficiency Anaemia · Iron Deficiency Anemia · Chronic Kidney Disease
Primary: Change in Hemoglobin (Hb) From Baseline to Week 8 — 1.22; 1.14 g/dL
Phase 2 N=15 Use of Pancreatic Enzymes in Short Bowel Syndrome
Short Bowel Syndrome
Primary: Change in Coefficient of Fat Absorption — 2.3 percentage of fat absorbed
Phase 3 N=9 Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
Cystic Fibrosis · Pancreatic Insufficiency
Primary: Percent Coefficient of Fat Absorption (CFA) — 34.5; 82.7 Percent CFA — p=0.0013
Phase 2 N=128 Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia
Congenital Hyperinsulinism (CHI) · Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
Primary: Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism — 34; 10; 4; 52 cases
N/A N=24 Special Investigation (All Cases) of LipaCreon in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Cystic Fibrosis · Exocrine Pancreatic Insufficiency · Pancreatic Diseases
Primary: Number of Patients With Adverse Drug Reaction — 1 participants
N/A N=26 Effect of Chronic Incretin-based Therapy in Cystic Fibrosis
Cystic Fibrosis · Pancreatic Insufficiency
Primary: Change in Second-phase Insulin Response Derived From the Glucose-potentiated Arginine Test as a Measure of β-cell Sensitivity to Glucose at Baseline and at 6 Months…