Clinical trials: Congenital Anomaly
Cystic Fibrosis
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
- Trial name
- NCT04362761
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 29 sites in Australia, Belgium and 2 more
- Volunteers
- 172 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
- Trial name
- NCT01835496
- Testing
- single 1500 mg dose of Ferriprox
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- ApoPharma
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
Anemia, Sickle Cell, Sickle Cell Disease
Preventing Sickle Cell Kidney Disease
Untreated hypertension and renal injury are risk factors for increased morbidity and mortality in sickle cell disease, yet early markers of progressive disease have not been identified and therapies to prevent the development of…
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease, Kidney Disease, Hypertension
- Trial name
- NCT02373241
- Testing
- Losartan
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
The purpose of the study is to evaluate the safety and potential side effects of everolimus (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been diagnosed with a brain tumor (astrocytoma) The…
- Trial name
- NCT00411619
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay
This clinical trial uses a laboratory test called a high throughput sensitivity assay in planning treatment for patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01872819
- Testing
- antitumor drug screening assay, chemotherapy, biological therapy
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Washington
Sickle Cell Disease
Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting
The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events.
- Trial name
- ESCAPED (NCT02411396)
- Where
- 3 sites in United States
- Volunteers
- 483 people
- Sponsor
- Johns Hopkins University
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Bortezomib in Treating Patients With High-Risk Acute Myeloid Leukemia in Remission
This phase II trial studies how well bortezomib works in treating patients with high-risk acute myeloid leukemia (AML) in remission.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT01465386
- Testing
- bortezomib
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Fred Hutchinson Cancer Center
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Sepsis, Congenital Diaphragmatic Hernia
Inflammatory Cytokine Quantification in Infants
This study proposes to quantify inflammatory cytokine profiles in three neonatal disease states, namely, neonatal sepsis, infants with a congenital diaphragmatic hernia defect, and infants suffering cardiopulmonary failure…
- Conditions
- Sepsis, Congenital Diaphragmatic Hernia, Neonatal Cardiopulmonary Failure
- Trial name
- NCT01155830
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- University of Utah
Epilepsy, Dravet Syndrome
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- GWPCARE5 (NCT02224573)
- Testing
- GWP42003-P
- Volunteers
- 681 people
- Sponsor
- GW Research Ltd
Atopic Dermatitis
Comparing Cetaphil Restoraderm System Versus Standard Skin Care in Infants
The goal of this study is to determine what effect Cetaphil® Restoraderm® system has on babies' skin versus Johnson \& Johnson baby lotion and skin cleanser.
- Trial name
- NCT01375205
- Testing
- Cetaphil Restoraderm, Standard of Care
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Oregon Health and Science University
Atopic Dermatitis
A Study to Determine the Efficacy of ZPL-3893787 in Subjects With Atopic Dermatitis
This was a randomized, double blind, placebo controlled, parallel group study in approximately 90 subjects with moderate-severe AD Eczema Area and Severity Index (EASI) ≥12 and ≤ 48 (0-72 scale).
- Trial name
- NCT02424253
- Testing
- ZPL-3893787
- Where
- 17 sites in Belgium, Germany and 2 more
- Volunteers
- 98 people
- Sponsor
- Ziarco Pharma Ltd
Cystic Fibrosis
Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P.
- Trial name
- NCT03905642
- Testing
- Arikayce™
- Where
- 11 sites in Belgium, Hungary and 5 more
- Volunteers
- 49 people
- Sponsor
- Insmed Incorporated
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia
A Phase II Trial of Regadenoson in Sickle Cell Anemia
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case…
- Trial name
- NCT01788631
- Testing
- Regadenoson
- Where
- 12 sites in United States
- Volunteers
- 100 people
- Sponsor
- Dana-Farber Cancer Institute
Inherited Epidermolysis Bullosa
Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
The purpose of this study was to compare intra-individually the reepithelialization of skin lesion(s) in inherited Epidermolysis bullosa (either 1 wound ≥10 cm2 and ≤200 cm2 in size divided in 2 equal halves or 2 comparable…
- Trial name
- NCT01294241
- Testing
- Oleogel-S10, Mepilex® soft silicone faced polyurethane foam dressing
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Birken AG
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