Clinical trials: Dose-escalation
B-cell Acute Lymphoblastic Leukemia
CD19x22 Chimeric Antigen Receptor T-cell Therapy (CAR T) in Pediatric B-ALL
This study will evaluate the safety and tolerability of administering a novel bispecific CD19/CD22-directed CAR T cell product (CD19x22) for the treatment of relapsed or refractory pediatric B-ALL.
- Trial name
- NCT06559189
- Testing
- CD19x22 CAR T
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of Colorado, Denver
Hemophilia B
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time.
- Trial name
- NCT01233440
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein, Plasma derived FIX [pdFIX]
- Where
- 20 sites in Austria, France and 4 more
- Volunteers
- 25 people
- Sponsor
- CSL Behring
Safety, Immunogenicity
EMaBS TB Vaccine Study
The study will commence with a dose-escalation and age de-escalation study in healthy adults and adolescents from the previous Entebbe Mother and Baby Study (EMaBS) in Entebbe, Uganda, focusing on ChAdOx1 85A, to provide safety…
- Trial name
- NCT03681860
- Testing
- ChAdOx185A, MVA85A, BCG
- Where
- 2 sites in Uganda, United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- University of Oxford
High-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia (CMML)
A Study of CBX-250 in Participants With Relapsed or Refractory Myeloid Leukemias
Study CBX-250-001 is a Phase 1, open-label, dose-escalation study of CBX-250 in participants with relapsed/refractory AML, HR-MDS, CMML, and CML. Participants aged ≥ 12 years are planned to be enrolled.
- Conditions
- High-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia (CMML), AML - Acute Myeloid Leukemia, Chronic Myeloid Leukemia
- Trial name
- CROSSCHECK-001 (NCT06994676)
- Testing
- CBX-250
- Where
- 11 sites in United States
- Volunteers
- 72 people
- Sponsor
- Crossbow Therapeutics, Inc.
AML, Childhood
A Phase 1 Trial of CD33xCD3 BsAb in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
Pediatric patients (<21 years at study entry) with relapsed or refractory acute myeloid leukemia (AML) will be treated with CD33*CD3 a bispecific antibody to investigate the safety and tolerability of the drug.
- Trial name
- NCT05077423
- Testing
- CD33*CD3 BsAb
- Where
- 13 sites in United States
- Volunteers
- 3 people
- Sponsor
- Y-mAbs Therapeutics
Advanced Cancer
Cisplatin HAI Study in Patients With Advanced Cancer and Dominant Liver Involvement
The goal of this clinical research study is find the highest safe dose of cisplatin that can be given with liposomal doxorubicin in the treatment of advanced cancer involving the liver.
- Trial name
- NCT00507962
- Testing
- Cisplatin, Liposomal Doxorubicin
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- M.D. Anderson Cancer Center
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
Diarrhoea
A Study on the Safety and Immune Responses to the GVGH altSonflex1-2-3 Vaccine Against Shigellosis in Adults, Children, and Infants
The aim of the current clinical study was to evaluate, for the first time in humans (FTIH), the safety and immunogenicity of the altSonflex1-2-3 candidate vaccine against S. sonnei and S. flexneri serotypes 1b, 2a, and 3a.
- Trial name
- NCT05073003
- Testing
- AltSonflex1-2-3 High Dose, AltSonflex1-2-3 Medium Dose, AltSonflex1-2-3 Low Dose
- Where
- 2 sites in Belgium, Kenya
- Volunteers
- 551 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Status Asthmaticus
Treating Exacerbations of Asthma With Oral Montelukast in Children
This research will establish a mg/kg dose for a future RCT to determine the efficacy of high-dose oral montelukast for children with moderate and severe acute asthma exacerbations.
- Trial name
- TEAM (NCT05819541)
- Testing
- Montelukast Oral Granules, Albuterol, Corticosteroid
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Vanderbilt University Medical Center
Congenital Adrenal Hyperplasia
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02349503
- Testing
- NBI-77860
- Where
- 2 sites in United States
- Sponsor
- Neurocrine Biosciences
Advanced Cancer, Solid Tumor
Vemurafenib in Combination With Everolimus or Temsirolimus With Advanced Cancer
The goal of this clinical research study is to find the highest tolerable dose of vemurafenib that can be given in combination with either everolimus or temsirolimus. The safety of these drug combinations will also be studied.
- Trial name
- NCT01596140
- Testing
- Vemurafenib, Everolimus, Temsirolimus
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- M.D. Anderson Cancer Center
Stargardt Disease
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021.
- Trial name
- AURORA (NCT07439887)
- Testing
- RTx-021
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Ray Therapeutics, Inc.
X-Linked Myotubular Myopathy
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07052929
- Testing
- ASP2957, Methylprednisolone, Prednisolone
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Plasmodium Falciparum Infection, Malaria
Anti-malaria MAb in Malian Children
The purpose of this study is to evaluate the safety and tolerability of onetime subcutaneous (SC) or intravenous (IV) administration of monoclonal antibody (MAb) L9LS in healthy Malian adults and one-time SC administration of…
- Trial name
- L9LS (NCT05304611)
- Testing
- L9LS (VRC-MALMAB0114-00-AB) Subcutaneous injection, L9LS (VRC-MALMAB0114-00-AB) intravenous injection
- Where
- 2 sites in Mali
- Volunteers
- 365 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Dexmedetomidine, Congenital Heart Disease
Pharmacokinetic Study of Intranasal Dexmedetomidine in Pediatric Patients With Congenital Heart Disease
The main objectives of the study are to determine peak plasma drug concentration levels and corresponding time of dexmedetomidine following intranasal administration in children age ≥1 mo to ≤ 6 yr with congenital heart disease…
- Trial name
- NCT03417999
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital of Philadelphia
Poliomyelitis
Safety and Immunogenicity of a New Inactivated Poliovirus Vaccine in Infants
A new inactivated polio vaccine based on attenuated poliovirus strains was developed to transfer the technology to manufacturers in low- and middle-income countries.
- Trial name
- NCT01709071
- Testing
- IPV, Low dose Sabin-IPV, Low dose adjuvanted Sabin-IPV
- Where
- 7 sites in Poland
- Volunteers
- 140 people, healthy volunteers welcome
- Sponsor
- Pauline Verdijk
Toxoplasmosis, Cerebral, HIV Infections
A Study of Azithromycin Plus Pyrimethamine in the Treatment of a Brain Infection in Patients With AIDS
To evaluate the effectiveness and toxicity of oral azithromycin and pyrimethamine as acute therapy for toxoplasmic encephalitis in AIDS patients.
- Trial name
- NCT00000966
- Testing
- Azithromycin, Pyrimethamine, Leucovorin calcium
- Where
- 11 sites in United States
- Volunteers
- 45 people
- Sponsor
- Pfizer
Salmonella Infections
A Study to Evaluate Safety, Reactogenicity, and Immune Response of GVGH iNTS-TCV Vaccine Against Invasive Nontyphoidal Salmonella Disease and Typhoid Fever in Infants
The purpose of this study is to evaluate the safety, reactogenicity, and immune response induced by the GlaxoSmithKline Biologicals SA (GSK) Vaccines Institute for Global Health (GVGH) invasive nontyphoidal Salmonella-typhoid…
- Trial name
- NCT07286370
- Testing
- Low dose of iNTS-TCV, Full dose of iNTS-TCV, TYPHIBEV
- Where
- The Gambia
- Volunteers
- 537 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical