Clinical trials: Dose-escalation
Pneumonia, Pulmonary Tuberculosis
Interferon Gamma for Drug Resistant Tuberculosis
This study will determine what dose of recombinant interferon-gamma is safe and effective for treating multiple drug-resistant tuberculosis.
- Trial name
- NCT00001407
- Testing
- Interferon Gamma
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Classical Hodgkin Lymphoma, Non-Hodgkin Lymphoma
A First-in-human Trial of GEN3017 in Hodgkin Lymphoma and Non-Hodgkin Lymphoma
The purpose of this trial is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics (PK), pharmacodynamics (PD), and anti-tumor activity of GEN3017 as a monotherapy in participants with relapsed or refractory…
- Trial name
- NCT06018129
- Testing
- GEN3017
- Where
- 4 sites in United States, Australia
- Volunteers
- 9 people
- Sponsor
- Genmab
Healthy
A Study to Evaluate the Safety, Tolerability, Immunogenicity and Vaccine-like Viral Shedding of MEDI-534, Against Respiratory Syncytial Virus (RSV) and Parainfluenza Virus Type 3 (PIV3), in Healthy 6 to <24 Month-old Children and in 2 Month-old Infants
Primary objective of this study is to describe the safety and tolerability of multiple doses of MEDI-534 in children 6 to less than (<) 24 months of age and in infants 2 months of age.
- Trial name
- NCT00686075
- Testing
- MEDI-534, Cohort 1, MEDI-534, Cohort 2, MEDI-534, Cohort 3
- Where
- 100 sites in United States, Australia and 7 more
- Volunteers
- 1,338 people, healthy volunteers welcome
- Sponsor
- MedImmune LLC
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Synovial Sarcoma
FHD-609 in Subjects With Advanced Synovial Sarcoma or Advanced SMARCB1-Loss Tumors
This Phase 1, multicenter, open-label, dose escalation and expansion study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-609 given…
- Trial name
- NCT04965753
- Testing
- FHD-609
- Where
- 12 sites in United States, France and 2 more
- Volunteers
- 55 people
- Sponsor
- Foghorn Therapeutics Inc.
Healthy
A Phase I Clinical Trial With Whole Virus Inactivated Influenza H5N1 Vaccine in Healthy Adolescents and Adults
Influenza is an acute respiratory infection caused by influenza virus with high incidence and serious complications even causing death.
- Trial name
- NCT01676675
- Testing
- 7.5μg /0.5ml, 15.0μg /0.5ml, 30.0μg /0.5ml
- Where
- China
- Sponsor
- Jiangsu Province Centers for Disease Control and Prevention
OTOF Gene Mutation, DFNB9
A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.
- Conditions
- OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders
- Trial name
- AUDIOGENE (NCT06370351)
- Testing
- SENS-501 administration
- Where
- 2 sites in Australia, France
- Volunteers
- 12 people
- Sponsor
- Sensorion
Methylmalonic Acidemia, Propionic Acidemia
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA).
- Trial name
- HERO (NCT04732429)
- Testing
- HST5040
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 26 people
- Sponsor
- HemoShear Therapeutics
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.
Phenylketonuria (PKU)
AAV Gene Therapy Study for Subjects with PKU
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency.
- Trial name
- NCT04480567
- Testing
- BMN 307
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- BioMarin Pharmaceutical
Neuroblastoma, Rhabdomyosarcoma
Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients
This is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Lymphoma, Wilm's Tumor
- Trial name
- NCT01169584
- Testing
- Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Jennerex Biotherapeutics
Fabry Disease
Evaluate the Safety and Exploratory Efficacy of GC1119
The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
- Trial name
- NCT01653444
- Testing
- GC1119
- Where
- 3 sites in South Korea
- Volunteers
- 8 people
- Sponsor
- Green Cross Corporation
Type I Gaucher Disease
Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
- Trial name
- NCT01463215
- Testing
- Ambroxol
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Exsar Corporation
Neuroectodermal Tumors, Rhabdomyosarcoma
Trial of Afatinib in Pediatric Tumours
Open-label, dose escalation, monotherapy, basket trial with biomarker specific MTD expansion cohort/Phase II part. The trial will consist of 2 parts: 1. Dose finding part to determine the MTD 2.
- Trial name
- NCT02372006
- Testing
- afatinib
- Where
- 28 sites in United States, Australia and 10 more
- Volunteers
- 56 people
- Sponsor
- Boehringer Ingelheim
NF2 Deficiency
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors.
- Trial name
- NCT06834438
- Testing
- ST002
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Hodgkin's Lymphomas
Phase 1 Study of MDX-1401 in Patients With CD30-positive Refractory/Relapsed Hodgkin's Lymphoma
To establish the safety and tolerability profile of MDX-1401 in patients with relapsed or refractory Hodgkin's Lymphoma (HL).
- Trial name
- MDX1401-01 (NCT00634452)
- Testing
- MDX-1401
- Where
- 6 sites in United States
- Volunteers
- 22 people
- Sponsor
- Bristol-Myers Squibb
Intracerebral Hemorrhage, Stroke-Associated Pneumonia (SAP)
An Open-Label, Dose-Escalation Study on the Safety, Tolerability and Preliminary Efficacy for Preventing SAP by Intravenous Pump-Delivered Propranolol in Patients With ICH
This is an open-label, dose-escalation study on the safety, tolerability and preliminary efficacy for preventing stroke-associated pneumonia of propranolol in patients with intracerebral hemorrhage.
- Trial name
- PROCHASE-DoE (NCT07528235)
- Testing
- Intravenous Pump-Delivered Propranolol
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Tang-Du Hospital
Epilepsy
Evaluation of the Efficacy and Safety of E2007 (Perampanel) Given as Adjunctive Therapy in Subjects With Refractory Partial Seizures
The purpose of this study was to evaluate the safety and tolerability of perampanel (up to 12 mg/day) given as adjunctive treatment in subjects with refractory partial seizures and to evaluate the maintenance of effect of…
- Trial name
- NCT00735397
- Testing
- perampanel
- Where
- 239 sites in United States, Argentina and 37 more
- Volunteers
- 1,218 people
- Sponsor
- Eisai Inc.