Clinical trials: Neonatal Disease
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes
This clinical trial studies idarubicin, cytarabine, and pravastatin sodium in treating patients with newly diagnosed acute myeloid leukemia or myelodysplastic syndromes.
- 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
- NCT01831232
- Testing
- pravastatin sodium, idarubicin, cytarabine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Fred Hutchinson Cancer Center
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Myeloid Leukemia/Transient Myeloproliferative Disorder
Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia
RATIONALE: Giving chemotherapy, such as fludarabine phosphate, busulfan, and cyclophosphamide, and total-body radiation therapy before a donor peripheral stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Myeloid Leukemia/Transient Myeloproliferative Disorder, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00322101
- Testing
- total-body irradiation, allogeneic hematopoietic stem cell transplantation, cyclophosphamide
- Where
- 8 sites in United States, Germany
- Volunteers
- 25 people
- Sponsor
- Fred Hutchinson Cancer Center
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Cystic Fibrosis
A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the cystic…
- Trial name
- NCT03150719
- Testing
- Tezacaftor/Ivacaftor, Ivacaftor
- Where
- 53 sites in United States, France and 1 more
- Volunteers
- 98 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pancreatic Cancer
Secretin (ChiRhoStim) Pancreas Perfusion for Pancreatic Adenocarcinoma
The purpose of this study is to test if secretin-enhanced CT is a useful noninvasive screening tool for pancreatic cancer in a high-risk population.
- Trial name
- NCT00587132
- Testing
- Synthetic Human Secretin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Mayo Clinic
Cystic Fibrosis Related Bone Disease
Cystic Fibrosis Related Bone Disease: the Role of CFTR
The purpose of this study is to determine whether ivacaftor, a recently FDA-approved CFTR potentiator, improves bone micro-architecture and strength in patients with cystic fibrosis with at least one G551D CFTR mutation.
- Trial name
- NCT01549314
- Where
- United States
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Tuberous Sclerosis Complex
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to…
- Trial name
- NCT02849457
- Testing
- Early Vigabatrin
- Where
- 13 sites in United States
- Volunteers
- 84 people
- Sponsor
- Martina Bebin
Hemophilia A
Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO
This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named…
- Trial name
- NCT01959919
- Testing
- Refacto FusENGO
- Where
- 19 sites in Italy
- Volunteers
- 86 people
- Sponsor
- Pfizer
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Angelman Syndrome
A Trial of Levodopa in Angelman Syndrome
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
- Trial name
- NCT01281475
- Testing
- Levodopa
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Wen-Hann Tan
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Fabry Disease
Open-Label Phase 3 Long-Term Safety Study of Migalastat
This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry disease who completed treatment in a previous…
- Trial name
- AT1001-041 (NCT01458119)
- Testing
- migalastat hydrochloride
- Where
- 25 sites in United States, Argentina and 11 more
- Volunteers
- 85 people
- Sponsor
- Amicus Therapeutics
Lamellar Ichthyosis
A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis
This is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with…
- Trial name
- NCT03738800
- Testing
- CD5789 Cream 200 µg/g, CD5789 Cream 100 µg/g, CD5789 Cream Vehicle
- Where
- 36 sites in United States, Australia and 7 more
- Volunteers
- 65 people
- Sponsor
- Mayne Pharma International Pty Ltd
Phenylketonuria
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
The purpose of this study is to evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU.
- Trial name
- NCT01212744
- Testing
- rAvPAL-PEG
- Where
- 9 sites in United States
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Familial Adenomatous Polyposis
Lyophilized Black Raspberries in Adults With Familial Adenomatous Polyposis (FAP)
This is a 36 week dietary intervention pilot study to evaluate the effects of lyophilized black raspberries on rectal polyp burden and biomarkers in subjects with FAP.
- Trial name
- NCT00770991
- Testing
- Black raspberry (BRB) Slurry, Black Raspberry (BRB) Suppositories
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- The Cleveland Clinic
Becker Muscular Dystrophy
(-)- Epicatechin Becker Muscular Dystrophy
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial.
- Trial name
- NCT03236662
- Testing
- (-)-Epicatechin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Craig McDonald, MD
Atopic Dermatitis
Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of EP262 in Subjects With Atopic Dermatitis
This Phase 2a trial will evaluate the effects of EP262 in subjects with atopic dermatitis
- Trial name
- EASE (NCT06144424)
- Testing
- Oral EP262
- Where
- 10 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Escient Pharmaceuticals, Inc
Alpha1-antitrypsin Deficiency
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study
The purpose of the study is 2-fold: (1) to evaluate the safety and potential immunogenicity of GLASSIA following intravenous (IV) administration via in-line filtration; and (2) to assess the effects of GLASSIA augmentation…
- Trial name
- NCT02525861
- Testing
- GLASSIA
- Where
- 18 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Baxalta now part of Shire
Alpha-1 Antitrypsin Deficiency
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT).
- Trial name
- NCT02363946
- Testing
- ARC-AAT Injection, Diphenhydramine
- Where
- 4 sites in Australia, Germany and 2 more
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Polycystic Kidney, Autosomal Dominant
Daily Caloric Restriction and Intermittent Fasting in Overweight and Obese Adults With Autosomal Dominant Polycystic Kidney Disease
The proposed research will determine the feasibility of delivering two behavioral weight loss interventions for 1 year in adults with autosomal dominant polycystic kidney disease (ADPKD) who are overweight or obese.
- Trial name
- NCT03342742
- Testing
- Weight Loss
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Colorado, Denver
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