Clinical trials: Phase 1
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency
- Trial name
- ENERGY (NCT05734196)
- Testing
- INZ-701
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Inozyme Pharma
Limb Girdle Muscular Dystrophy Type 2D/R3
BBM-L101 Gene Therapy in Participants With LGMD2D/R3
This phase 1 study will evaluate a single intravenous infusion of BBM-L101, an investigational AAV-based gene therapy designed to deliver a functional SGCA gene, in ambulatory children aged 4 to <13 years with genetically…
- Trial name
- NCT07874516
- Testing
- BBM-L101 Injection
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital
Duchenne Muscular Dystrophy
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD).
- Trial name
- IGNITE DMD (NCT03368742)
- Testing
- SGT-001
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Solid Biosciences Inc.
B-cell Acute Lymphoblastic Leukemia (B-ALL)
Study of AZD4512 Monotherapy or in Combination With Anticancer Agents in Participants With Acute Lymphoblastic Leukemia
The study is intended to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AZD4512 in patients with relapsed/refractory B-Cell acute lymphoblastic leukemia (r/r B-ALL).
- Trial name
- ALLight (NCT07109219)
- Testing
- AZD4512 monotherapy
- Where
- 27 sites in United States, Australia and 7 more
- Volunteers
- 83 people
- Sponsor
- AstraZeneca
Acute Lymphoblastic Leukemia, Leukemia, Lymphoblastic, Acute, Philadelphia-Positive
Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL)
Multi-center, open-label, single arm study of asciminib in participants aged ≥1 year to ≤30 years old with r/r Ph+ or ABL-class Ph-like ALL. This study will have 2 parts: Part 1 dose escalation and Part 2 dose expansion.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Lymphoblastic, Acute, Philadelphia-Positive, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
- Trial name
- NCT07387926
- Testing
- Asciminib Adult formulation, Asciminib Pediatric formulation, Dexamethasone
- Where
- 4 sites in Australia, Israel and 2 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Severe Hemophilia A
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors.
- Trial name
- NCT06703606
- Testing
- MARSTACIMAB
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Metastatic Malignant Neoplasm in the Lung, Metastatic Melanoma
Aerosolized Aldesleukin in Treating Patients With Lung Metastases
This phase I/II trial studies the side effects and best dose of aerosolized aldesleukin and to see how well it works in treating patients with cancer that has spread from the original tumor to the lungs.
- Conditions
- Metastatic Malignant Neoplasm in the Lung, Metastatic Melanoma, Metastatic Osteosarcoma, Metastatic Renal Cell Cancer
- Trial name
- NCT01590069
- Testing
- Aerosolized Aldesleukin, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- M.D. Anderson Cancer Center
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
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
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Malignant Brain Neoplasm, Recurrent Malignant Brain Neoplasm
CAR T Cells After Lymphodepletion for the Treatment of IL13Rα2 Positive Recurrent or Refractory Brain Tumors in Children
This phase I trial investigates the side effects of chemotherapy and cellular immunotherapy in treating children with IL13Ralpha2 positive brain tumors that have come back after a period of improvement (recurrent) or do not…
- Conditions
- Malignant Brain Neoplasm, Recurrent Malignant Brain Neoplasm, Refractory Malignant Brain Neoplasm
- Trial name
- NCT04510051
- Testing
- Cyclophosphamide, Fludarabine, IL13Ralpha2-specific Hinge-optimized 41BB-co-stimulatory CAR Truncated CD19-expressing Autologous T-Lymphocytes
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- City of Hope Medical Center
Knee, ACL
Restoration of Knee Extension After ACL Reconstruction
The purpose of this study is to determine whether the addition of a home-based high-intensity knee extension stretching device (Ermi Knee Extensionater) to standard postoperative physical therapy improves recovery of full knee…
- Trial name
- NCT07685561
- Testing
- Standard Physical Therapy, Ermi Knee Extensionater
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- University of Alabama at Birmingham
Refractory Acute Lymphoblastic Leukemia, Relapsed Acute Lymphoblastic Leukemia
Trial Treating Relapsed Acute Lymphoblastic Leukemia With Venetoclax and Navitoclax
This is a phase I/II clinical trial evaluating the activity of combination chemotherapy with venetoclax and navitoclax in children with relapsed or refractory acute lymphoblastic leukemia or lymphoma (rALL) and assessing the…
- Trial name
- NCT05192889
- Testing
- Venetoclax, Navitoclax, Dexamethasone
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- St. Jude Children's Research Hospital
Congenital Heart Disease, Cardiopulmonary Bypass
Influence of Personalized Lung Volume Optimization Maneuver on Lung Function and Cardiac Performance in Children
The goal of this randomized interventional clinical trial is to learn if a standardized lung volume optimization maneuver (LVOM) is beneficial in 1.
- Conditions
- Congenital Heart Disease, Cardiopulmonary Bypass, Cardiac Surgery, Mechanical Ventilation
- Trial name
- ILOCO (NCT07193719)
- Testing
- End-expiratory lung volume optimization maneuver with PEEP titration, Standard Care (in control arm)
- Where
- Germany
- Volunteers
- 80 people
- Sponsor
- Charite University, Berlin, Germany
Developmental and Epileptic Encephalopathies, Epileptic Spasms
Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies
Epileptic spasms (ES) are a predominantly infantile seizure type observed frequently in certain genetic disorders. Ketogenic diet (high ratio of fat to carbohydrate/protein) is an established non-medication treatment for…
- Conditions
- Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
- Trial name
- NCT06700811
- Testing
- Ketogenic diet
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Heather Olson
Osteosarcoma, Soft Tissue Sarcoma
Safety and Efficacy of DIT309 in Advanced Bone and Soft Tissue Sarcomas
This is a open-Label, dose-escalation study to evaluate the safety, tolerability and antitumor activity of DIT309 in subjects with advanced bone and soft tissue sarcomas.The study also plan to explore the Maximum Tolerated Dose…
- Trial name
- NCT07052383
- Testing
- DIT309 cell injection
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Tcelltech Inc.
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Navitoclax, Venetoclax, and Decitabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Previously Treated With Venetoclax
This phase Ib trial is to find the side effect and best dose of navitoclax when given together with venetoclax and decitabine in treating patients with acute myeloid leukemia that has come back (relapsed) or does not respond to…
- Trial name
- NCT05222984
- Testing
- Decitabine, Navitoclax, Venetoclax
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- City of Hope Medical Center
B-Cell Leukemia, Non-Hodgkin's Lymphoma
CLIC-2201 for the Treatment of Relapsed/Refractory B Cell Malignancies
This is a phase I dose-finding trial of an autologous CD22 targeting chimeric antigen receptor (CAR)-T cell product, called CLIC-2201, for participants with relapsed/refractory B cell malignancies.
- Conditions
- B-Cell Leukemia, Non-Hodgkin's Lymphoma, B-cell Acute Lymphoblastic Leukemia, Diffuse Large B Cell Lymphoma
- Trial name
- NCT06208735
- Testing
- CLIC-2201
- Where
- 7 sites in Canada
- Volunteers
- 24 people
- Sponsor
- British Columbia Cancer Agency
Acute Myeloid Leukemia (AML)
Niclosamide in Pediatric Patients With Relapsed and Refractory AML
Protocol is designed to evaluate a niclosamide dose escalation scale in combination with cytarabine as a therapeutic modality for pediatric subjects with relapsed/refractory acute myeloid leukemia.
- Trial name
- NCT05188170
- Testing
- Niclosamide
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Stanford University
Neuronal Ceroid Lipofuscinosis Type 2
A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease
This is a registrational, first-in-human, open-label, dose escalation and expansion study evaluating the safety, tolerability and effectiveness of TTX-381, administered by injection under the retina, for the treatment of eye…
- Trial name
- NCT05791864
- Testing
- TTX-381
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Tern Therapeutics, LLC
Cystinosis
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
- Trial name
- INCA (NCT05994534)
- Testing
- Cysteamine Bitartrate, N-Acetylcysteine Amide
- Where
- Australia
- Volunteers
- 8 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
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