Clinical trials: Phase 2
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 14 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Friedreich Ataxia
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of…
- Trial name
- NCT06447025
- Testing
- CTI-1601
- Where
- 10 sites in United States
- Volunteers
- 85 people
- Sponsor
- Larimar Therapeutics, Inc.
Generalized Myasthenia Gravis
A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).
- Trial name
- roMyG (NCT06149559)
- Testing
- rozanolixizumab
- Where
- 9 sites in Italy, Japan and 2 more
- Volunteers
- 14 people
- Sponsor
- UCB Biopharma SRL
Port Wine Stains
Rapamycin Dose-Ranging Efficacy Study in Port Wine Stains
The goal of this clinical trial is to learn if the use of rapamycin cream can be used as a treatment together with pulsed dyed laser in treating port wine stain birthmarks.
- Trial name
- NCT07436611
- Testing
- Rapamycin cream, Pulsed dyed laser
- Where
- 2 sites in United States, Spain
- Volunteers
- 30 people
- Sponsor
- AFT Pharmaceuticals, Ltd.
Blepharitis
A Phase 2, Controlled, Double Blind, Randomized and Multicenter Study to Compare Efficacy and Safety of a Novel Topical Therapy (APT-001, Spinosad 1.8%) in Patients With Blepharitis.
The purpose of this study is to establish the clinical efficacy of APT-001 topical therapy 1.8% in patients with blepharitis as compared to its vehicle control, and to establish that the therapeutic is safe and generally well…
- Trial name
- VISTA-1 (NCT06720896)
- Testing
- APT-001 topical ophthalmic ointment (spinosad), Vehicle control for APT-001 topical ophthalmic ointment
- Where
- 2 sites in Guatemala
- Volunteers
- 66 people
- Sponsor
- Aperta Biosciences, LLC
Mucopolysaccharidosis II
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous…
- Trial name
- NCT06075537
- Testing
- tividenofusp alfa
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 99 people
- Sponsor
- Denali Therapeutics Inc.
Agitation
Dexmedetomidine Transdermal Systems (DMTS) Treatment for Agitation Associated With Dementia of the Alzheimer's Type
The primary objective of this study is to evaluate the efficacy of DMTS on frequency and severity of agitation associated with dementia of the Alzheimer's type, compared with placebo.
- Trial name
- NCT06052254
- Testing
- 12 cm2 - 2 Active DMTS Patches
- Where
- 8 sites in United States
- Volunteers
- 150 people
- Sponsor
- Teikoku Pharma USA, Inc.
Ulcerative Colitis
A Study to Evaluate Etrasimod Treatment in Adolescents With Ulcerative Colitis
The purpose of this study is to determine the safety, efficacy, and pharmacokinetics (PK) of etrasimod for the treatment of moderately to severely active ulcerative colitis in adolescents (≥ 12 years up to < 18 years of age).
- Trial name
- NCT05287126
- Testing
- Etrasimod
- Where
- 47 sites in United States, Japan and 3 more
- Volunteers
- 36 people
- Sponsor
- Pfizer
Human Immunodeficiency Virus (HIV) Infection
Doravirine (DOR) in Human Immunodeficiency Virus (HIV)-Infected Children Aged 4 Weeks to <12 Years and <45 kg (MK-1439-066)
This is a single-group, open-label, multi-site study in pediatric participants with human immunodeficiency virus type 1 (HIV-1) infection, aged 4 weeks to <12 years and weighing <45 kg, who are treatment-naive (TN) or have been…
- Trial name
- NCT04375800
- Testing
- Doravirine, 2 NRTIs, DOR/3TC/TDF
- Where
- 26 sites in United States, Colombia and 6 more
- Volunteers
- 84 people
- Sponsor
- Merck Sharp & Dohme LLC
Pertussis (Whooping Cough), Diphtheria
A Phase II Clinical Trial of Absorbed Acellular Pertussis (Two-Component) Diphtheria-Tetanus Combined Vaccine (For Adults and Adolescents)
the immunogenicity and safety of different dosages of adsorbed acellular pertussis (two-component) diphtheria-tetanus combined vaccine
- Conditions
- Pertussis (Whooping Cough), Diphtheria, Tetanus
- Trial name
- NCT07894055
- Testing
- Tdap vaccine
- Where
- China
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Changchun BCHT Biotechnology Co.
Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB
- Trial name
- HMBeacon (NCT07575308)
- Testing
- ALN-6400
- Where
- 21 sites in United States, Netherlands and 3 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Diarrhoea
A Study on the Immune Response and Safety of a Multicomponent Shigella Vaccine in Preventing Shigellosis in Infants
This study evaluates the immune response and safety of a multicomponent, 2-dose Shigella vaccine in preventing shigellosis in African infants.
- Trial name
- NCT06663436
- Testing
- altSonflex1-2-3 Dose A, altSonflex1-2-3 Dose B, altSonflex1-2-3 Dose C
- Where
- Kenya
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
The goal of this clinical trial is to learn if sonrotoclax (BGB-11417) is safe and may help treat children and adolescents with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has come back after treatment…
- Conditions
- Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, B-cell Acute Lymphoblastic Leukemia, Pediatric Cancer
- Trial name
- NCT07770698
- Testing
- sonrotoclax, Azacitidine, Inotuzumab ozogamicin
- Volunteers
- 30 people
- Sponsor
- BeOne Medicines
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Colitis, Ulcerative
A Study of How the Medicine Called "Etrasimod" Works in Children With the Gut Disease Called Ulcerative Colitis
The purpose of this study is to determine the safety, efficacy, and pharmacokinetics (PK) of etrasimod for the treatment of moderately to severely active ulcerative colitis in pediatrics participants (≥ 2 years up to < 12 years…
- Trial name
- ELEVATE-UCkids (NCT07470879)
- Testing
- Etrasimod
- Where
- 46 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- Pfizer
B-cell Acute Lymphoblastic Leukemia, Large B-cell Lymphoma
Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19)
This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19-positive hematologic malignancies.
- Conditions
- B-cell Acute Lymphoblastic Leukemia, Large B-cell Lymphoma, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
- Trial name
- NCT06533579
- Testing
- Dose Level 1, VNX-101, Dose Level 2, VNX-101, Dose Level 3, VNX-101
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Vironexis Biotherapeutics Inc.
Proteinuric Kidney Disease
Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.
- Trial name
- AMPLITUDE (NCT05312879)
- Testing
- VX-147
- Where
- 314 sites in United States, Belgium and 11 more
- Volunteers
- 522 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
High-Risk Neuroblastoma
A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients
Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years.
- Trial name
- SHINE (NCT07549321)
- Testing
- hu1418K322A + Temozolomide + Irinotecan
- Where
- 18 sites in United States, Canada and 5 more
- Volunteers
- 144 people
- Sponsor
- Renaissance Pharma Ltd.
Pruritus, Chronic Kidney Diseases
Safety and Tolerability of Difelikefalin in Adolescents on Haemodialysis With Moderate-to-Severe Pruritus
Rationale: People with long term kidney disease who are on haemodialysis (a procedure for removing waste products from the blood) commonly develop a condition that makes their skin very itchy.
- Trial name
- NCT06593392
- Testing
- Difelikefalin
- Where
- 18 sites in China, Greece and 5 more
- Volunteers
- 18 people
- Sponsor
- Vifor Fresenius Medical Care Renal Pharma
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