Clinical trials: Newborns
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Treatment of Bleeding, Prophylaxis of Bleeding
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood.
- Conditions
- Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
- Trial name
- Alfa-PROTECT (NCT05147662)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 18 sites in United States, Argentina and 5 more
- Volunteers
- 36 people
- Sponsor
- Bayer
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Graft Versus Host Disease, Leukemia
Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis w/ Tacrolimus & Mycophenolate Mofetil
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00608517
- Testing
- anti-thymocyte globulin, cyclophosphamide, fludarabine phosphate
- Where
- 4 sites in United States
- Volunteers
- 6 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Hemolytic Disease of the Fetus and Newborn
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)
The purpose of this study is to evaluate the safety in mother and neonate/infant of M281 administered to pregnant women who are at high risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN).
- Trial name
- NCT03842189
- Testing
- M281
- Where
- 19 sites in United States, Australia and 7 more
- Volunteers
- 25 people
- Sponsor
- Janssen Research & Development, LLC
Hereditary Angioedema
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
The objectives of the study are to: 1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema 2.
- Trial name
- NCT01095497
- Testing
- CINRYZE
- Where
- 7 sites in United States
- Volunteers
- 26 people
- Sponsor
- Shire
Hereditary Angioedema
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
- Trial name
- NCT04739059
- Testing
- CSL312
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 171 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Preventing Acute Chest Syndrome by Transfusion Feasibility Study
Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS.
- Trial name
- PROACTIVE (NCT00951808)
- Testing
- Single blood transfusion, Standard care
- Where
- 24 sites in United States
- Volunteers
- 237 people
- Sponsor
- Carelon Research
Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma
Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205)
The purpose of this study is to evaluate the safety and efficacy of pemigatinib plus pembrolizumab or pemigatinib alone versus the standard of care for participants with metastatic or unresectable urothelial carcinoma who are not…
- Trial name
- NCT04003610
- Testing
- Pemigatinib, Pembrolizumab, Gemcitabine
- Where
- 79 sites in United States, Austria and 14 more
- Volunteers
- 7 people
- Sponsor
- Incyte Corporation
Atrial Fibrillation, Deep Vein Thrombosis
Randomized Trial of Interventions to Improve Warfarin Adherence
The purpose of this research study is to see if a lottery which provides the opportunity to win money, a reminder system using a "Med-eMonitor", or the combination of both might be useful in helping patients to achieve better…
- Conditions
- Atrial Fibrillation, Deep Vein Thrombosis, Dilated Cardiomyopathies
- Trial name
- WIN3 (NCT00904982)
- Testing
- Financial Incentive and Med-eMonitor, 2Med-eMonitor
- Where
- 2 sites in United States
- Volunteers
- 268 people
- Sponsor
- University of Pennsylvania
Clubfoot
Outcomes of Compliance With Brace Wear in Clubfoot
It has been shown that compliance with brace wear can significantly improve the treatment outcome of idiopathic clubfoot deformities.
- Trial name
- NCT01481324
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- Shriners Hospitals for Children
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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
Gout
Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout
The purpose of this study is to evaluate the efficacy and safety of febuxostat 40 mg extended release (XR) and 80 mg XR in comparison with febuxostat 40 mg immediate release (IR) and 80 mg IR, respectively, in participants with…
- Trial name
- NCT02139046
- Testing
- Febuxostat IR, Febuxostat XR, Colchicine
- Where
- 192 sites in United States
- Volunteers
- 1,790 people
- Sponsor
- Takeda
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Ixazomib (MLN9708) in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This phase 2 trial studies how well ixazomib(MLN9708) works in treating study participants 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
- NCT02030405
- Testing
- Ixazomib
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Steven E. Coutre
Anemia, Sickle Cell
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization.
- Trial name
- NCT00530270
- Testing
- Dexamethasone
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Down Syndrome
Efficacy and Safety of Memantine Hydrochloride in Enhancing the Cognitive Abilities of Young Adults With Down Syndrome
The purpose of this 16-week research study is to determine whether a drug called memantine hydrochloride (memantine) has the potential to help improve memory and other cognitive abilities of young adults with Down syndrome (DS).
- Trial name
- NCT01112683
- Testing
- Memantine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Colorado, Denver
Dermatitis, Atopic, Dermatitis
Effect of Benralizumab in Atopic Dermatitis
Atopic Dermatitis (AD), also known as eczema, is a common skin disease characterized by itchy lesions. The prevalence of AD has increased over the past few decades, with 15-30% of children and 2-10%of adults being affected.
- Conditions
- Dermatitis, Atopic, Dermatitis, Eczema, Skin Diseases
- Trial name
- NCT03563066
- Testing
- Benralizumab
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- McMaster University
VHL Gene Mutation, VHL
MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)
The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL participants treated with MK-3795.
- Conditions
- VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03108066
- Testing
- MK-3795
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
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