Clinical trials: Genetic Conditions
Leukemia, Myelogenous, Chronic, Breakpoint Cluster Region-Abelson Proto-oncogene (BCR-ABL) Positive
A Multicenter Phase 3, Open-Label Study of Bosutinib Versus Imatinib in Adult Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia
Phase 3, 2-arm, randomized, open label trial. Patients will be randomized to receive bosutinib or imatinib for the duration of the study.
- Trial name
- NCT02130557
- Testing
- Bosutinib, Imatinib
- Where
- 189 sites in United States, Australia and 24 more
- Volunteers
- 536 people
- Sponsor
- Pfizer
X-linked Hypophosphatemia
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
- Trial name
- NCT06525636
- Testing
- KK8123
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 39 people
- Sponsor
- Kyowa Kirin Co., Ltd.
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to compare the bioavailability of the Test Product, Nitisinone 10 mg Tablet, under fasting and fed conditions (food-effect).
- Trial name
- NCT02750332
- Testing
- Nitisinone
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Cystic Fibrosis
Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis
Cystic Fibrosis is defined as a genetic disorder affecting approximately 100,000 individuals worldwide. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene.
- Trial name
- NCT01958944
- Testing
- Nitric oxide
- Where
- 2 sites in Israel
- Volunteers
- 9 people
- Sponsor
- Beyond Air Inc.
Myotonic Dystrophy Type 1 (DM1)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
- Trial name
- NCT06926621
- Testing
- VX-670
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Retinitis Pigmentosa (RP), Usher Syndrome
24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- NCT07290530
- Testing
- N-acetylcysteine amide
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Epidermolysis Bullosa (EB)
Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa
The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at study entry.
- Trial name
- NCT07482787
- Testing
- SD-101 Dermal Cream (6%), Vehicle (SD-101 0%)
- Where
- 4 sites in United States
- Volunteers
- 80 people
- Sponsor
- Paradigm Therapeutics
Aneuploidy, Down Syndrome
Clinical Evaluation of the SEQureDx Trisomy Test in Low Risk Pregnancies
Pregnant women with low risk indicators for fetal chromosomal aneuploidy will be enrolled. Study blood will be collected in the first or second trimester at a scheduled prenatal screening visit, processed to plasma, and stored…
- Conditions
- Aneuploidy, Down Syndrome, Noninvasive Prenatal Screening
- Trial name
- NCT01597063
- Testing
- SEQureDx Trisomy Test
- Where
- 27 sites in United States
- Volunteers
- 3,333 people
- Sponsor
- Sequenom, Inc.
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study.
- Trial name
- NCT00630747
- Testing
- Idursulfase
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 94 people
- Sponsor
- Shire
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST)
Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study
The purpose of this study was to allow continued use of nilotinib in patients who were on nilotinib treatment in a Novartis-sponsored, Oncology Clinical Development \& Medical Affairs (CD\&MA) study and were benefiting from the…
- Conditions
- Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
- Trial name
- NCT01735955
- Testing
- Nilotinib
- Where
- 33 sites in United States, Austria and 14 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Retinitis Pigmentosa (RP), Usher Syndrome
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- SLORP Preserve (NCT07710196)
- Testing
- NPI-001
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Indolent Systemic Mastocytosis
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.
- Trial name
- AB06006 (NCT00814073)
- Testing
- Masitinib, Best Supportive Care
- Where
- 24 sites in United States, France
- Volunteers
- 135 people
- Sponsor
- AB Science
Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy
Dilated cardiomYopathy iNtervention With Allogeneic MyocardIally-regenerative Cells (DYNAMIC)
To determine the safety profile of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with DCM. The study will further explore safety and exploratory efficacy endpoints of CAP-1002.
- Conditions
- Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy, Heart Failure
- Trial name
- DYNAMIC (NCT02293603)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CDCs)
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Capricor Inc.
Arthritis, Gouty
Efficacy and Safety of a Single Dose of Canakinumab (ACZ885) in Hospitalized Patients With Acute Gout
This is an exploratory proof-of-concept study to evaluate the safety and efficacy of canakinumab (ACZ885) for inflammation and pain associated with acute gouty arthritis.
- Trial name
- NCT00663169
- Testing
- canakinumab, dexamethasone
- Where
- 4 sites in United States, Switzerland and 1 more
- Volunteers
- 6 people
- Sponsor
- Novartis
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose…
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM)
- Trial name
- NCT06371417
- Testing
- RAY121
- Where
- 77 sites in United States, Australia and 18 more
- Volunteers
- 144 people
- Sponsor
- Chugai Pharmaceutical
Advanced Solid Tumors Cancer, MSI-H Cancer
A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.
The goal of this clinical trial is to learn if NDI-219216 is safe for patients, and if NDI-219216 might be a possible treatment for advanced solid tumors in the later phases of the study.
- Trial name
- NCT06898450
- Testing
- NDI-219216
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 134 people
- Sponsor
- Nimbus Wadjet, Inc.
Infertility, Male Infertility
To Investigate if the Harvester® Improves Sperm Motility and Blastocyst Utilization (the Percent of Fertilized Eggs That Develop to the Point That They Are Able to be Transferred) in IVF Cycles.
The goal of this prospective, multicenter randomized controlled clinical trial is to evaluate whether the SwimCount Harvester microfluidic sperm preparation device can achieve equivalent or superior clinical outcomes compared to…
- Conditions
- Infertility, Male Infertility, InVitro Fertilization, Aneuploidy Rate
- Trial name
- NCT07369362
- Testing
- microfludics semen processing device
- Where
- United States
- Volunteers
- 1,600 people
- Sponsor
- MotilityCount aps
Lennox-Gastaut Syndrome
Safety and Effectiveness of Open-Label Clobazam in Subjects With Lennox-Gastaut Syndrome
The objective of this study is to evaluate the long-term safety and effectiveness of open-label clobazam in the treatment of drop seizures in subjects with LGS.
- Trial name
- LGS (NCT01160770)
- Testing
- Clobazam
- Volunteers
- 267 people
- Sponsor
- Lundbeck LLC
Functional Gastrointestinal Disorders
Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms
To select among different dietary and clinical conditions the most appropriate to assess the impact of a fermented milk product on gas-related symptoms
- Trial name
- FLATBACK (NCT02936713)
- Testing
- Fermented milk product with probiotics (FMPP)
- Where
- Spain
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Danone Global Research & Innovation Center