Clinical trials: Malformation
Gout
Safety and Efficacy Study of PEG-uricase in the Treatment of Hyperuricemic Patients With Symptomatic Gout
These are two replicate studies to evaluate the safety and efficacy of PEG (polyethylene glycol)-uricase in controlling the uric acid level in symptomatic gout patients with high uric acid levels who are unable to take standard…
- Trial name
- NCT00325195
- Testing
- pegloticase
- Where
- 56 sites in United States, Canada and 1 more
- Volunteers
- 225 people
- Sponsor
- Savient Pharmaceuticals
Atopic Dermatitis, Atopic Hand Eczema
A 32-week Trial to Evaluate the Efficacy and Safety of Tralokinumab in Subjects With Moderate-to-severe Atopic Hand Eczema Who Are Candidates for Systemic Therapy
The purpose of this study is to test if treatment with tralokinumab is safe and efficacious in treating moderate-to-severe atopic hand eczema.
- Trial name
- ADHAND (NCT05958407)
- Testing
- Tralokinumab
- Where
- 69 sites in United States, Belgium and 8 more
- Volunteers
- 235 people
- Sponsor
- LEO Pharma
Leukemia, Myelodysplastic Syndromes
Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Zosuquidar trihydrochloride, a modulator of multidrug resistance (MDR), may help daunorubicin and cytarabine…
- Trial name
- NCT00046930
- Testing
- filgrastim, sargramostim, cytarabine
- Where
- 92 sites in United States, Israel
- Volunteers
- 449 people
- Sponsor
- Eastern Cooperative Oncology Group
Cystic Fibrosis
A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
identify and validate biomarkers that might reflect partial restoration of CFTR function and can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR modulators and other relevant therapies in…
- Trial name
- PROSPECT (NCT02477319)
- Testing
- Observational
- Where
- 38 sites in United States
- Volunteers
- 452 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Tourette Syndrome
Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 8 weeks of treatment.
- Trial name
- NCT02581865
- Testing
- NBI-98854
- Where
- 30 sites in United States
- Volunteers
- 124 people
- Sponsor
- Neurocrine Biosciences
Hereditary Angioedema (HAE)
FIRAZYR General Drug Use-Results Survey (Japan)
The objectives of this survey are to collect data to report the safety and efficacy of Firazyr (Icatibant acetate) in the post-marketing phase in participants diagnosed with Hereditary Angioedema (HAE).
- Trial name
- NCT04057131
- Testing
- Firazyr
- Where
- 18 sites in Japan
- Volunteers
- 179 people
- Sponsor
- Shire
Cystic Fibrosis
Dose Escalation Study of KB001 in Cystic Fibrosis Patients Infected With Pseudomonas Aeruginosa
The primary objective of this study is to evaluate the safety and tolerability of a single dose of KB001 in Cystic Fibrosis patients infected with Pseudomonas aeruginosa (Pa)
- Trial name
- NCT00638365
- Testing
- KB001
- Where
- 11 sites in United States
- Volunteers
- 27 people
- Sponsor
- Humanigen, Inc.
Tetralogy of Fallot
Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -
Tetralogy of Fallot (TOF) is the most common cyanotic congenital heart defect with the vast majority of survivors of corrective surgery left with some degree of right ventricular (RV) volume overload due to pulmonary…
- Trial name
- TOFandPVR (NCT03634072)
- Testing
- PVR
- Where
- 6 sites in United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital of Philadelphia
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Decitabine in Treating Patients With Previously Untreated Acute Myeloid Leukemia
This phase II trial is studying how well decitabine works in treating patients with previously untreated acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT00492401
- Testing
- decitabine, laboratory biomarker analysis, pharmacological study
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Telangiectasia, Hereditary Hemorrhagic, Epistaxis
North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.
- Trial name
- NOSE (NCT01408030)
- Testing
- Sterile saline, Bevacizumab, Estriol
- Where
- 6 sites in United States
- Volunteers
- 123 people
- Sponsor
- James Gossage
Dermatitis, Atopic, Eczema, Atopic
Safety and Efficacy of Quadriderme® in the Treatment of Impetiginous Eczema (Study P05134AM4)
This is a parallel-group, randomized, active-controlled, double-blind, Phase 4 trial comparing three creams in the treatment of impetiginous eczema: Arm A: QUADRIDERME® cream (betamethasone diproprionate, clotrimazole, and…
- Conditions
- Dermatitis, Atopic, Eczema, Atopic, Skin Diseases, Eczematous
- Trial name
- NCT00671528
- Testing
- Cream (betamethasone diproprionate, clotrimazole, and gentamicin sulfate), Cream (betamethasone diproprionate and gentamicin), Cream (betamethasone diproprionate)
- Volunteers
- 3 people
- Sponsor
- Organon and Co
Microcystic Lymphatic Malformation
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
- Trial name
- NCT05050149
- Testing
- PTX-022
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Palvella Therapeutics, Inc.
Atopic Dermatitis
A Study of Long-term Baricitinib (LY3009104) Therapy in Atopic Dermatitis
The purpose of this study is to evaluate the long-term safety and efficacy of baricitinib in participants with atopic dermatitis.
- Trial name
- BREEZE-AD3 (NCT03334435)
- Testing
- Baricitinib
- Where
- 185 sites in Argentina, Australia and 17 more
- Volunteers
- 1,645 people
- Sponsor
- Eli Lilly and Company
Cystic Fibrosis, Chronic Pancreatitis
Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis…
- Trial name
- NCT05069597
- Testing
- CREON
- Where
- 21 sites in United States
- Volunteers
- 30 people
- Sponsor
- AbbVie
Sickle Cell Disease
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with…
- Trial name
- NCT00445978
- Testing
- Sapropterin Dihydrochloride
- Where
- 12 sites in United States
- Volunteers
- 32 people
- Sponsor
- BioMarin Pharmaceutical
BRAF or NRAS Mutant Metastatic Melanoma
A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma
The study will assess the safety and efficacy of single-agent MEK162 in adult patients with locally advanced and unresectable or metastatic malignant cutaneous melanoma, harboring BRAFV600E or NRAS mutations.
- Trial name
- NCT01320085
- Testing
- MEK162
- Where
- 25 sites in United States, Germany and 3 more
- Volunteers
- 183 people
- Sponsor
- Pfizer
Solid Tumor Malignancy
Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR…
- Trial name
- NCT03822117
- Testing
- Pemigatinib
- Where
- 90 sites in United States, Denmark and 9 more
- Volunteers
- 111 people
- Sponsor
- Incyte Corporation
Chronic Motor or Vocal Tic Disorder, Tourette's Disorder
Aripiprazole in Children and Adolescents With Chronic Tic Disorder or Tourette's Disorder
The purpose of this trial is to demonstrate the efficacy and safety of aripiprazole in children and adolescents aged 6~18 years with chronic tic disorders or Tourette's disorder
- Trial name
- NCT00706589
- Testing
- aripiprazole
- Where
- 6 sites in South Korea
- Volunteers
- 61 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
Down Syndrome
Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome
The purpose of this study is to test in adults with Down Syndrome the safety, tolerability and immunogenicity of a vaccine, ACI-24.
- Trial name
- 3-Star (NCT02738450)
- Testing
- ACI-24 low dose, ACI-24 high dose
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- AC Immune SA
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of atumelnant (CRN04894) in participants with classic congenital adrenal hyperplasia (CAH)…
- Trial name
- NCT05907291
- Testing
- atumelnant (CRN04894)
- Where
- 27 sites in United States, Argentina and 5 more
- Volunteers
- 38 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
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