Clinical trials: Fetal Developmental Abnormality
Sickle Cell Disease
Improving Self-Management in Adolescents With Sickle Cell Disease
The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a…
- Trial name
- SCThrive (NCT02851615)
- Testing
- SCThrive Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
PIK3CA-Related Overgrowth Spectrum (PROS)
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the…
- Trial name
- EPIK-P1 (NCT04285723)
- Testing
- alpelisib
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Dysplasia, Congenital Hip
Efficacy of Tranexamic Acid for Reducing Blood Loss and Blood Transfusion After Periacetabular Osteotomy
The goal of this randomized controlled, double-blinded trial is to assess the efficacy of intravenous tranexamic acid, a drug, in reducing blood loss and transfusion in patients undergoing periacetabular osteotomy, an elective…
- Trial name
- NCT02253810
- Testing
- Tranexamic Acid
- Where
- United States
- Volunteers
- 89 people
- Sponsor
- Hospital for Special Surgery, New York
Colic
An Intervention to Decrease Infant Crying
This is a study looking at the Happiest Baby on the Block technique. The investigators hypothesized that infants of mothers given a 30 minute videotape demonstrating the Happiest Baby on the Block technique would fuss/cry less…
- Trial name
- NCT00796523
- Testing
- Happiest Baby videotape, control videotape
- Where
- United States
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Riverside Methodist Hospital
Facioscapulohumeral Muscular Dystrophy
Study of Testosterone and rHGH in FSHD
The purpose of this study is to investigate the safety and tolerability of combination therapy with recombinant human growth hormone (rHGH) and testosterone in adult male patients with facioscapulohumeral muscular dystrophy…
- Trial name
- STARFISH (NCT03123913)
- Testing
- Testosterone Enanthate, Somatropin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Rochester
Gaucher Disease
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care.
- Trial name
- SWITCH (NCT04718779)
- Testing
- Digital Engagement Application (GD App), No Intervention
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Takeda
Hemoglobin SC Disease
Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications.
- Trial name
- NCT02640573
- Testing
- Hydroxyurea
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Baylor College of Medicine
Molar Incisor Hypomineralization
Remineralization of Molar Incisor Hypomineralization (MIH) With a Hydroxyapatite Toothpaste
This study tests the remineralization of donor teeth (donorMIH) in situ in participants using 10% hydroxyapatite (HAP) toothpaste and sodium fluoride toothpaste.
- Trial name
- NCT05485662
- Testing
- Hydroxyapatite toothpaste, Sodium Fluoride Toothpaste
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
Sickle Cell Anemia Crisis
Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises?
The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room.
- Trial name
- NCT03541980
- Testing
- Acetaminophen, Normal saline
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Newark Beth Israel Medical Center
Congenital Bleeding Disorder, Haemophilia B
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP)…
- Trial name
- paradigm™5 (NCT01467427)
- Testing
- nonacog beta pegol
- Where
- 51 sites in United States, Brazil and 10 more
- Volunteers
- 25 people
- Sponsor
- Novo Nordisk A/S
Cystic Fibrosis
Efficacy of TOBI Podhaler vs. Tobramycin Inhalation Solution
Inhaled tobramycin is a Cystic Fibrosis Foundation recommended effective treatment for individuals with cystic fibrosis for the management of Pseudomonas aeruginosa airway colonization and improves the FEV1 and reduces the number…
- Trial name
- PETIS (NCT02038803)
- Testing
- TOBIpodhaler
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Rush University Medical Center
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
Primary Immune Deficiency
Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Trial name
- NCT00419341
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration
- Where
- 13 sites in United States
- Volunteers
- 49 people
- Sponsor
- CSL Behring
Cystic Fibrosis
Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
- Trial name
- GALAXY (NCT03801993)
- Where
- 26 sites in United States
- Volunteers
- 402 people
- Sponsor
- Chris Goss
Idiopathic Short Stature, Failure to Thrive
Ghrelin Levels in Children With Poor Growth
The investigators hypothesize that low serum ghrelin levels may characterize a group of patients with poor weight gain and/or linear growth who do not have any other identified cause for growth failure.
- Trial name
- NCT01070173
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Tripler Army Medical Center
Hemophilia B
BAX 326 Surgery Study in Hemophilia B Patients
The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency…
- Trial name
- NCT01507896
- Testing
- Recombinant factor IX
- Where
- 14 sites in Argentina, Bulgaria and 8 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are…
- Trial name
- NCT01531673
- Testing
- VX-661, Ivacaftor
- Where
- 36 sites in United States, Canada and 2 more
- Volunteers
- 194 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cystic Fibrosis
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
This study compares the safety of the tobramycin solution for inhalation with the tobramycin dry powder formulation, used with a simple inhaler
- Trial name
- EAGER (NCT00388505)
- Testing
- Tobramycin Inhalation Powder, Tobramycin Solution for Inhalation
- Where
- 72 sites in United States, Australia and 12 more
- Volunteers
- 517 people
- Sponsor
- Novartis Pharmaceuticals
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