Clinical trials: Genetic Syndrome
Fabry Disease
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Behçet Disease
Dusquetide for the Treatment of Behcet's Disease
This is a clinical study to see if dusquetide can treat flares of oral and genital ulcers caused by Behcet's Disease. Study participants will receive an infusion of dusquetide twice a week for 4 weeks (8 treatments total), with…
- Trial name
- NCT06386744
- Testing
- Dusquetide
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Soligenix
Cystinuria
TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this…
- Trial name
- TCUPS (NCT02538016)
- Testing
- Tolvaptan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Caleb Nelson
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Achondroplasia
A Study to Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Emphysema, Alpha 1-proteinase Inhibitor Deficiency
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v).
- Trial name
- NCT00670007
- Testing
- Alpha1- proteinase inhibitor [human]
- Where
- 21 sites in Australia, Canada and 9 more
- Volunteers
- 140 people
- Sponsor
- CSL Behring
Adult Rhabdomyosarcoma, Adult Synovial Sarcoma
Cixutumumab in Treating Patients With Relapsed or Refractory Solid Tumors
This phase II trial is studying the side effects and how well cixutumumab works in treating patients with relapsed or refractory solid tumors. Monoclonal antibodies, such as cixutumumab, can block tumor growth in different ways.
- Conditions
- Adult Rhabdomyosarcoma, Adult Synovial Sarcoma, Childhood Hepatoblastoma, Childhood Synovial Sarcoma
- Trial name
- NCT00831844
- Testing
- cixutumumab, laboratory biomarker analysis
- Where
- 105 sites in United States, Australia and 1 more
- Volunteers
- 116 people
- Sponsor
- National Cancer Institute (NCI)
Cystinuria
Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria
The purpose of this study is to evaluate the effect of escalating doses of cystine biding thiol drugs, including tiopronin and d-penicillamine, on the urinary cystine capacity, which is a measure of the amount of cystine in the…
- Trial name
- NCT02125721
- Testing
- Tiopronin 1g per day, Tiopronin 2g per day, Tiopronin 3g per day
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- NYU Langone Health
HIV
Barbershop Talk: HIV Prevention for African American Heterosexual Men
This program utilizes a community-engaged research approach to implementing and evaluating a program that seeks to reduce sexual risk behavior among Black adult heterosexual men.
- Trial name
- NCT01980771
- Testing
- BTWB intervention, Cancer prevention and screening
- Where
- 2 sites in United States
- Volunteers
- 860 people, healthy volunteers welcome
- Sponsor
- State University of New York - Downstate Medical Center
Cystic Fibrosis
Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations
Based on previous clinical findings, the investigator hypothesize that ivacaftor will have synergistic effects with drugs that facilitate truncated but partially active W1282X CFTR protein processing (tezacaftor) in patients with…
- Trial name
- NCT03624101
- Testing
- Symdeko, Ivacaftor and Symdeko
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Advanced Breast Cancer
Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR-positive, HER2-negative, Advanced Breast Cancer (aBC) With PIK3CA Mutation, Whose Disease Progressed on or After Endocrine Treatment
The purpose of this study was to determine the safety of alpelisib plus fulvestrant in men and post-menopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, advanced or…
- Trial name
- ALPINIST (NCT05631795)
- Testing
- Alpelisib, Fulvestrant
- Where
- 12 sites in India
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Gout and Hyperuricemia
Phase II Dose Finding Study of RDEA3170 Versus Placebo in Japanese Patients With Gout or Asymptomatic Hyperuricemia
This study is to examine the hypothesis that administration of RDEA3170 to Japanese patients with gout or asymptomatic hyperuricemia in doses of 5 mg, 7.5 mg, 10 mg, 12.5 mg and 15 mg once daily, respectively will result in…
- Trial name
- NCT02078219
- Testing
- RDEA3170, Allopurinol
- Where
- 14 sites in Japan
- Volunteers
- 204 people
- Sponsor
- AstraZeneca
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
Cystic Fibrosis, Bronchiectasis
Effect of High Frequency Chest Wall Oscillation Vests on Spirometry Measurements
The impact of high-frequency chest wall oscillation therapy on spirometry values (Forced Expiratory Volume, Forced Vital Capacity, Peak Expiratory Flow, Forced Expiratory Flow and Tidal Volume is investigated during use of…
- Trial name
- NCT03534986
- Testing
- International Biophysics AffloVest, Hill-Rom The Vest, Respirtech inCourage
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- International Biophysics Corporation
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Acute Heart Failure
Study of Safety, Tolerability and Pharmacokinetics of Serelaxin in Japanese Acute Heart Failure (AHF) Patients
This is a multicenter, randomized, double-blind, placebo-controlled study to assess safety, tolerability and pharmacokinetics and to explore efficacy of IV infusion of 10 µg/kg/day and 30 µg/kg/day serelaxin for 48 hours compared…
- Trial name
- NCT02002702
- Testing
- Serelaxin
- Where
- 15 sites in Japan
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibromatosis 2, Vestibular Schwannoma
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system.
- Trial name
- NCT00973739
- Testing
- Lapatinib
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Leber Congenital Amaurosis
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
- Trial name
- NCT00749957
- Testing
- rAAV2-CB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Beacon Therapeutics
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