Clinical trials: Phase 2 Study
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Choriocarcinoma
Gamma-Secretase Inhibitor RO4929097 in Treating Young Patients With Relapsed or Refractory Solid Tumors, CNS Tumors, Lymphoma, or T-Cell Leukemia
This phase I/II clinical trial is studying the side effects and best dose of gamma-secretase inhibitor RO4929097 and to see how well it works in treating young patients with relapsed or refractory solid tumors, CNS tumors…
- Conditions
- Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Choriocarcinoma, Childhood Central Nervous System Germinoma, Childhood Central Nervous System Mixed Germ Cell Tumor
- Trial name
- NCT01088763
- Testing
- gamma-secretase/Notch signalling pathway inhibitor RO4929097, diagnostic laboratory biomarker analysis, pharmacological study
- Where
- 12 sites in United States, Canada
- Volunteers
- 129 people
- Sponsor
- National Cancer Institute (NCI)
Bloody Diarrhea, STEC, Unspecified
Phase 2/3 Study to Evaluate PK, Safety & Efficacy of INM004 in STEC Positive Pediatric Patients for Prevention of HUS
The investigational medicinal product (IMP), INM004, proposes to neutralize the toxin in the bloodstream to prevent the interaction of the Stx with the specific receptor, by means of a polyclonal antibody to be administered upon…
- Conditions
- Bloody Diarrhea, STEC, Unspecified, Hemolytic-Uremic Syndrome, Pediatric Kidney Disease
- Trial name
- NCT04132375
- Testing
- INM004
- Where
- 7 sites in Argentina
- Volunteers
- 11 people
- Sponsor
- Inmunova S.A.
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma
Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma
- Trial name
- NCT03213665
- Testing
- Tazemetostat
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Osteosarcoma
A Placebo-Controlled Study of Saracatinib (AZD0530) in Patients With Recurrent Osteosarcoma Localized to the Lung
The purpose of this study is to determine how long patients who undergo complete surgical removal of recurrent osteosarcoma in the lung will remain free of cancer after taking Saracatinib compared to patients taking placebo (a…
- Trial name
- NCT00752206
- Testing
- Saracatinib
- Where
- 18 sites in United States
- Volunteers
- 38 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
HIV/AIDS
REMUNE HIV/AIDS Vaccine Phase II Pediatric Safety & Efficacy Clinical Study
The primary objective is to compare \& evaluate between the treatment groups the changes in decline/reduction of HIV viral load \& increase changes in WBC white blood cell counts in the adult Remune dose vs the low dose Remune…
- Trial name
- NCT02291809
- Testing
- REMUNE
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Immune Response BioPharma, Inc.
AIDS-related Diffuse Large Cell Lymphoma, AIDS-related Diffuse Mixed Cell Lymphoma
Topotecan Hydrochloride in Treating Children With Meningeal Cancer That Has Not Responded to Previous Treatment
Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. This phase II trial is studying how well topotecan hydrochloride works in treating children with meningeal cancer that…
- Conditions
- AIDS-related Diffuse Large Cell Lymphoma, AIDS-related Diffuse Mixed Cell Lymphoma, AIDS-related Diffuse Small Cleaved Cell Lymphoma, AIDS-related Immunoblastic Large Cell Lymphoma
- Trial name
- NCT00005811
- Testing
- topotecan hydrochloride, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- National Cancer Institute (NCI)
Solid Tumors Harboring MET Amplification
A Study to Assess Adverse Events and Change in Disease Activity in Participants 12 Years of Age or Older With Locally Advanced or Metastatic Solid Tumors That Harbor MET Amplification Receiving Intravenously Infused Telisotuzumab Adizutecan
Cancer is a condition where cells in a specific part of body grow and reproduce uncontrollably. The purpose of this study is to assess adverse events and change in disease activity of telisotuzumab adizutecan.
- Trial name
- NCT07196644
- Testing
- Telisotuzumab Adizutecan
- Where
- 38 sites in United States, Israel and 3 more
- Volunteers
- 100 people
- Sponsor
- AbbVie
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to…
- Trial name
- CHORD (NCT05788536)
- Testing
- DB-OTO
- Where
- 17 sites in United States, Germany and 3 more
- Volunteers
- 36 people
- Sponsor
- Regeneron Pharmaceuticals
Mycobacterium Avium-intracellulare Infection, HIV Infections
A Phase II Safety and Efficacy Study of Clarithromycin in the Treatment of Disseminated M. Avium Complex (MAC) Infections in Patients With AIDS
This study is designed to evaluate the efficacy and safety of clarithromycin given orally at 1 of 3 doses to treat disseminated Mycobacterium avium complex infections (MAC) in patients with AIDS.
- Trial name
- NCT00000644
- Testing
- Clarithromycin
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Acute Lymphoblastic Leukemia, B-precursor, Non-Hodgkin Lymphoma, B-cell
T-cells Expressing Anti-CD19 CAR in Pediatric and Young Adults With B-cell Malignancies
This phase 1 / 2 study will evaluate the response of B-cell malignancies expressing CD19 to autologous T cells transduced with a second generation anti-CD19 chimeric antigen receptor in children and young adults.
- Trial name
- NCT02772198
- Testing
- CD19 CAR T cells
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- Sheba Medical Center
Type 1 Diabetes
Stem Cell Educator Therapy in Type 1 Diabetes
The translational potential to the clinical applications of cord blood stem cells has increased enormously in recent years, mainly because of its unique advantages including no risk to the donor, no ethical issues, low risk of…
- Trial name
- NCT01350219
- Testing
- Stem Cell Educator
- Where
- 4 sites in China, Spain
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Throne Biotechnologies Inc.
Childhood Infratentorial Ependymoma, Childhood Supratentorial Ependymoma
Study of Tumor Samples From Patients With Ependymoma Treated on the Children's Oncology Group ACNS0121 Trial
This research trial studies tumor samples from patients with ependymoma treated on the Children Oncology Group ACNS0121 trial. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn…
- Conditions
- Childhood Infratentorial Ependymoma, Childhood Supratentorial Ependymoma, Newly Diagnosed Childhood Ependymoma
- Trial name
- NCT01407744
- Testing
- laboratory biomarker analysis
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Children's Oncology Group
Infections, Streptococcal
Assess Reacto- and Immunogenicity of Pneumococcal Conjugate Vaccine When Given as Booster or a 2 Dose Catch up Schedule
This is a booster study in 2 groups of healthy children less than 3 years old to measure the reactogenicity, safety and immunogenicity of GSK Biologicals' pneumococcal conjugate vaccine, when given as a booster or as a two-dose…
- Trial name
- NCT00513409
- Testing
- Synflorix, Infanrix Hexa, Havrix
- Where
- Chile
- Volunteers
- 163 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
Gastric Adenocarcinoma, Esophageal Squamous Cell Carcinoma
Efficacy and Safety of Anlotinib Hydrochloride Combined With Nivolumab in the Treatment of Gastric and Esophageal Cancer
Patients with unresectable or metastatic gastric or esophageal cancer, with first-line treatment applied are to be recruited in the study.
- Trial name
- OASIS (NCT04503967)
- Testing
- Anlotinib Hydrochloride With Nivolumab
- Volunteers
- 48 people
- Sponsor
- Shanghai Zhongshan Hospital
Hidradenitis Suppurativa
A Phase 3 Study to Assess Efficacy Safety and Tolerability of Remibrutinib in Adult and Adolescent Patients With Moderate to Severe Hidradenitis Suppurativa (RECHARGE 1)
The purpose of this study is to establish the efficacy, safety, and tolerability of remibrutinib (LOU064) Dose A and Dose B compared to placebo in participants with moderate to severe hidradenitis suppurativa (HS).
- Trial name
- NCT06799000
- Testing
- Remibrutinib Dose A, Remibrutinib Dose B
- Where
- 139 sites in United States, Argentina and 18 more
- Volunteers
- 588 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Malignant Mesothelioma, Epithelial Mesothelioma
Erlotinib in Treating Patients With Malignant Mesothelioma of the Lung
Erlotinib may interfere with the growth of tumor cells and slow the growth of the tumor. This phase II trial is studying how well erlotinib works in treating patients with malignant mesothelioma of the lung
- Conditions
- Advanced Malignant Mesothelioma, Epithelial Mesothelioma, Recurrent Malignant Mesothelioma, Sarcomatous Mesothelioma
- Trial name
- NCT00039182
- Testing
- erlotinib hydrochloride, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Sarcoma, Ewing, Sarcoma
SM-88 Maintenance Therapy for Advanced Ewing's Sarcoma and as Salvage Therapy for Sarcoma
The primary objective is to evaluate the efficacy of SM-88, a combination metabolic cancer treatment, in two study cohorts: Clinically advanced Ewing's Sarcoma patients who have not progressed at the conclusion of systemic…
- Trial name
- HopES (NCT03778996)
- Testing
- Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Sarcoma Oncology Research Center, LLC
Hypochondroplasia
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to < 36 months over a 52-week period.
- Trial name
- NCT07126262
- Testing
- Vosoritide
- Where
- 26 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- BioMarin Pharmaceutical