Clinical trials: Multiple Congenital Anomaly
Healthy
Controlled Randomized Study With Athena Base Plate Versus SenSura Base Plate on Perspiring Skin
Aim The aim of the Athena project is to develop an ostomy product that is softer and more flexible in order to improve the comfort for people with a stoma.
- Trial name
- NCT01245413
- Testing
- SenSura, Athena
- Where
- Denmark
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Coloplast A/S
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma
Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma
This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute…
- Conditions
- Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma
- Trial name
- NCT01190930
- Testing
- Cyclophosphamide, Cytarabine, Dexamethasone
- Where
- 243 sites in United States, Australia and 5 more
- Volunteers
- 9,350 people
- Sponsor
- Children's Oncology Group
Long QT Syndrome
Study of LQT-1213 on QTc-induced Prolongation in Healthy Adult Subjects (Part1) and on Congenital Long QT in Patients Diagnosed With Type 2 or 3 Long QT Syndrome (Part 2).
Part 1: This is a Phase 1b, randomized, double-blind, crossover, dose escalation, placebo-controlled study to evaluate the effect of oral LQT-1213 on dofetilide-induced QTc prolongation in healthy adult subjects.
- Trial name
- NCT05906732
- Testing
- LQT-1213, Dofetilide 250 μg Cap
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Thryv Therapeutics, Inc.
Primary Immunodeficiency Disease
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study.
- Trial name
- NCT04842643
- Testing
- IGSC 20% infusion
- Where
- 8 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Hyperphagia, Prader-Willi Syndrome
Pilot Study of Startle-response Test to Assess Transcranial Direct Current Stimulation-induced Modulation of Hyperphagia in Prader-Willi Syndrome
The purpose of this study is to determine the effects of transcranial direct current stimulation (tDCS) as it modifies hyperphagia in obese subjects, non-obese subjects, and subjects with Prader-Willi syndrome (PWS).
- Trial name
- NCT01863017
- Testing
- tDCS
- Where
- 3 sites in United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Healthy Subject
HPV Vaccine Intervention for Young Sexual Minority Men
This phase III trial studies how well the Outsmart HPV intervention works in increasing HPV vaccine initiation and completion among young individuals who report having a history of same-sex partners, being sexually attracted to…
- Trial name
- NCT04032106
- Testing
- Best Practice, Informational Intervention, Questionnaire Administration
- Where
- 2 sites in United States
- Volunteers
- 1,227 people, healthy volunteers welcome
- Sponsor
- Ohio State University Comprehensive Cancer Center
Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive).
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
- Trial name
- NCT03472287
- Testing
- Diacerein 1% Ointment
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Castle Creek Pharmaceuticals, LLC
PTCH1 or SMO Activated Solid and Hematologic Tumors
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT02002689)
- Testing
- LDE225
- Where
- 10 sites in United States
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Human Immunodeficiency Virus Infection, Sexually Transmitted Diseases
Men Together Making a Difference: Reducing HIV/STD Risk Behavior Among South African Men
Sub-Saharan Africa has about 10% of the world's population, but was home to more than 60% of all people living with HIV in 2003.
- Trial name
- NCT01490359
- Testing
- Men Making a Difference HIV/STD Risk Reduction Intervention, Health Promotion Control
- Volunteers
- 1,181 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Zollinger-Ellison Syndrome, Multiple Endocrine Neoplasia
Treatment of Zollinger-Ellison Syndrome With Prevacid
The purpose of this study is to study the safety and efficacy of high dose Prevacid in the long-term treatment of patients who secrete abnormally large amounts of gastric acid.
- Trial name
- NCT00204373
- Testing
- Lansoprazole (Prevacid)
- Volunteers
- 72 people
- Sponsor
- Charles Mel Wilcox, MD
Developmental Disabilities, Weight Loss
Weight Management for Adolescents With IDD
The goal of this study is to explore the impact of two different diets (conventional vs. enhanced stop light) and two different delivery systems (face-to-face vs.
- Conditions
- Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome
- Trial name
- IDDAdol (NCT02561754)
- Testing
- Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF)
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- University of Kansas Medical Center
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Diet as a Potential Treatment for Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to learn if dietary habits can affect vasopressin secretion in patients with autosomal dominant polycystic kidney disease.
- Trial name
- NCT02225860
- Testing
- Diet and water adjustment
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Tufts Medical Center
Autosomal Dominant Polycystic Kidney Disease
Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease
This study aims to better understand electrolyte handling in patients with autosomal dominant polycystic kidney disease treated with the SGLT2 inhibitor Empagliflozin.
- Trial name
- SIDIA (NCT06435858)
- Testing
- Empagliflozin
- Where
- 2 sites in Switzerland
- Volunteers
- 16 people
- Sponsor
- Cantonal Hospital Graubuenden
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Prader-Willi Syndrome
Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)
The purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation…
- Trial name
- NCT00399893
- Testing
- Octreotide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke University
Basal Cell Nevus Syndrome
Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
Multicenter, double-blind, randomized, vehicle-controlled study that evaluates the efficacy and safety of patidegib gel 2% and 4% in comparison with vehicle in participants at least 18 years of age that meet the diagnostic…
- Trial name
- NCT02762084
- Testing
- Patidegib, Vehicle gel
- Where
- 2 sites in United Kingdom
- Volunteers
- 17 people
- Sponsor
- PellePharm, Inc.
Advanced Breast Cancer
Study of Efficacy and Safety of LEE011 in Men and Postmenopausal Women With Advanced Breast Cancer.
The main aim of this study was to evaluate the efficacy and safety of adding ribociclib to fulvestrant in men and postmenopausal women with hormone receptor positive (HR+), HER2-negative advanced breast cancer.
- Trial name
- MONALEESA-3 (NCT02422615)
- Testing
- Ribociclib, Fulvestrant
- Where
- 174 sites in United States, Australia and 28 more
- Volunteers
- 726 people
- Sponsor
- Novartis Pharmaceuticals