Clinical trials: Phase 2
Generalized Myasthenia Gravis
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of…
- Trial name
- THYME (NCT06987539)
- Testing
- Inebilizumab
- Where
- 14 sites in United States, Argentina and 6 more
- Volunteers
- 15 people
- Sponsor
- Amgen
Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced), Alopecia Areata
Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
Background: Autoimmune polyendocrinopathy candidiasis ectodermal dystrophy (APECED) is a problem of the immune system. In people with APECED, the immune system makes a mistake and attacks the body.
- Trial name
- NCT05398809
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and…
- Trial name
- ENDEAVOR (NCT05419492)
- Testing
- ETX101
- Where
- 14 sites in United States, Australia and 1 more
- Volunteers
- 113 people
- Sponsor
- Encoded Therapeutics
Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia
Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
The purpose of this study is to understand the safety, tolerability, efficacy, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of orally administered AZD3632 in participants with advanced haematologic…
- Conditions
- Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia, Higher-risk Myelodysplastic Syndromes
- Trial name
- MOMENTUM (NCT07155226)
- Testing
- AZD3632, Posaconazole
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- AstraZeneca
Malignant Neoplasm
A Study of Belzutifan in Adolescent Participants With Solid Tumors (MK-9999-01E/LIGHTBEAM-U01)
Researchers are looking for new ways to treat adolescents with locally advanced, unresectable, or metastatic solid tumors. Participants were enrolled into pheochromocytoma/paraganglioma (PPGL), wild type gastrointestinal stromal…
- Trial name
- NCT07700758
- Testing
- Belzutifan
- Where
- 4 sites in United States, Belgium and 2 more
- Volunteers
- 15 people
- Sponsor
- Merck Sharp & Dohme LLC
Epinephrine, Hypersensitivity, Immediate
Pharmacokinetics and Safety of a Single Dose of Epinephrine Nasal Spray in Paediatric Allergy Patients
This Phase 2, open-label, single-arm study will evaluate the pharmacokinetics, pharmacodynamics, safety, and tolerability of a single dose of epinephrine nasal spray (ENS) in children and adolescents 2 to 17 years of age with a…
- Conditions
- Epinephrine, Hypersensitivity, Immediate, Anaphylaxis
- Trial name
- NCT07891780
- Testing
- Epinephrine Nasal Spray, 2.5 mg, Epinephrine Nasal Spray, 5.25 mg
- Where
- 2 sites in United States
- Volunteers
- 44 people
- Sponsor
- Hikma Pharmaceuticals USA Inc.
Diffuse Intrinsic Pontine Gliomas (DIPG), Progressive DIPG
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
- Conditions
- Diffuse Intrinsic Pontine Gliomas (DIPG), Progressive DIPG, Refractory DIPG, Recurrent DIPG
- Trial name
- NCT06838676
- Testing
- ACT001
- Where
- 20 sites in United States, Australia and 3 more
- Volunteers
- 60 people
- Sponsor
- Nationwide Children's Hospital
Wilms Tumor, Rhabdoid Tumor
A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors
The purpose of this study is to find out whether selinexor is an effective treatment for people who have a relapsed/refractory Wilms tumor, rhabdoid tumor, MPNST, BCOR-driven sarcoma, or another solid tumor that makes a higher…
- Conditions
- Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST
- Trial name
- NCT05985161
- Testing
- Selinexor
- Where
- 16 sites in United States
- Volunteers
- 45 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Cytomegalovirus Infections, Primary Immune Deficiency Disorder
Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV)
CMV cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults (CAYA) with refractory cytomegalovirus (CMV) infection post…
- Trial name
- NCT03266640
- Testing
- viral specific cytotoxic t-lymphocytes
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Non-Hodgkin Lymphoma, Burkitt Lymphoma
Obinutuzumab and ICE Chemotherapy in Refractory/Recurrent CD20+ Mature NHL
The purpose of this study is to determine the safety of administering obinutuzumab as a single agent alone and in combination with ifosfamide, carboplatin, and etoposide (ICE) chemotherapy and determine the response rate of this…
- Conditions
- Non-Hodgkin Lymphoma, Burkitt Lymphoma, Diffuse Large B-Cell Lymphoma, Primary Mediastinal B-cell Lymphoma
- Trial name
- O-ICE (NCT02393157)
- Testing
- Obinutuzumab, Liposomal ARA-C, Ifosfamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- New York Medical College
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Kidney Transplant
Advancing Transplantation Outcomes in Children
This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF).
- Trial name
- ADVANTage (NCT06055608)
- Testing
- Sirolimus, Belatacept, Mycophenolate Mofetil
- Where
- 21 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Malaria, Cerebral
DON in Pediatric Cerebral Malaria
The goal of this clinical trial is to evaluate the safety of a single intravenous dose of DON in healthy adults, adults with uncomplicated malaria, and children 12 months-14 years old with clinically defined Cerebral Malaria.
- Trial name
- NCT05478720
- Testing
- 6-diazo-5-oxo-L-norleucine (DON)
- Where
- 2 sites in Malawi
- Volunteers
- 152 people
- Sponsor
- Douglas Postels, MD, MS
Lassa Fever
A Lassa Fever Vaccine Trial in Adults and Children Residing in West Africa
A Phase 2 Randomized, Double-Blinded, Placebo-Controlled Clinical Trial to Evaluate the Safety, Tolerability, and Immunogenicity of rVSV∆G-LASV-GPC Vaccine in Adults and Children Residing in West Africa
- Trial name
- NCT05868733
- Testing
- Day 1 Dose (2×106 pfu), Day 1 Dose (1×107 pfu), Day 1 Dose (2×10^5 pfu)
- Where
- 3 sites in Ghana, Liberia and 1 more
- Volunteers
- 530 people, healthy volunteers welcome
- Sponsor
- International AIDS Vaccine Initiative
Diphtheria, Pertussis
A Phase I/II Clinical Trial of the Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine Adsorbed
This is a randomized, positive-controlled phase I/II clinical trial to evaluate the safety and preliminary immunogenicity of the Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine…
- Conditions
- Diphtheria, Pertussis, Tetanus, Poliomyelitis
- Trial name
- NCT07891104
- Testing
- Diphtheria, Tetanus, Acellular Component Pertussis and Sabin Inactivated Poliomyelitis Vaccine Adsorbed, Diphtheria, Tetanus and Acellular Component Pertussis Combined Vaccine, Diphtheria, Tetanus and Acellular Component Pertussis Combined Vaccine+Inactivated Poliomyelitis Vaccine Made From Sabin Strains
- Where
- 3 sites in China
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- Institute of Medical Biology, Chinese Academy of Medical Sciences
Adenovirus, Primary Immune Deficiency Disorder
Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with…
- Trial name
- NCT03266627
- Testing
- cytotoxic t-lymphocytes
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Graves Disease, Graves' Disease
Impact of Vitamin D Therapy on Thyroid Function and Antibody Levels in Pediatric Graves' Disease
The goal of this randomized pilot feasibility clinical trial is to determine the feasibility of implementing a protocol for a larger trial to assess the effects of high-dose vitamin D supplementation in pediatric patients (9-17…
- Conditions
- Graves Disease, Graves' Disease, Hyperthyroidism
- Trial name
- NCT07369063
- Testing
- Ergocalciferol 50,000 IU, Methimazole
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Northwell Health
LGCMN, Large and Giant Congenital Melanocytic Nevi
Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults
Background: Large and giant congenital melanocytic nevi (LGCMN) are dark birthmarks (moles). They can grow to 8 inches or more across.
- Conditions
- LGCMN, Large and Giant Congenital Melanocytic Nevi, NF1, Solid Tumor
- Trial name
- NCT07891286
- Testing
- Mirdametinib
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Cancer Institute (NCI)
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo