Clinical trials: terminated
Angelman Syndrome
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
- Trial name
- Aspire (NCT06617429)
- Testing
- GTX-102
- Where
- 28 sites in United States, Canada and 4 more
- Volunteers
- 129 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Angelman Syndrome
Long-term Extension of GTX-102 in Angelman Syndrome
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
- Trial name
- NCT06415344
- Testing
- GTX-102
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203.
- Trial name
- NCT06961344
- Testing
- zagociguat 15mg
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 37 people
- Sponsor
- Tisento Therapeutics
Angelman Syndrome
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
- Trial name
- Aurora (NCT07157254)
- Testing
- No intervention, GTX-102
- Where
- 21 sites in United States, Argentina and 6 more
- Volunteers
- 55 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Amblyopia
Evaluation of AmblyoFix for the Treatment of Unilateral Amblyopia
This is a prospective, randomized, parallel-group, assessor-masked, non-inferiority clinical trial designed to compare the efficacy, safety, and adherence of AmblyoFix binocular digital therapy with standard occlusion (patching)…
- Trial name
- NCT07240311
- Testing
- AmblyoFix binocular digital therapy, Occlusion therapy (Patching)
- Where
- Iran
- Volunteers
- 40 people
- Sponsor
- Eyesight Electronics
Insomnia
Registry to Collect Information on Pregnancy, Neonatal, and Infant Outcomes in Pregnant Women Exposed to QUVIVIQ®
This study will investigate pregnancy, neonatal, and infant outcomes in women exposed to QUVIVIQ during pregnancy compared to women unexposed to QUVIVIQ during pregnancy.
- Trial name
- NCT06498128
- Testing
- Daridorexant, Non-orexin receptor antagonist medications for insomnia, No insomnia medication
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Osteosarcoma, Osteosarcoma in Children
Effects of Maintenance Cabozantinib+BSC Versus BSC in Children and AYA With Osteosarcoma
The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through surgery.
- Conditions
- Osteosarcoma, Osteosarcoma in Children, Osteosarcoma in Adolescents and Young Adults
- Trial name
- CabOSTar (NCT06341712)
- Testing
- Cabozantinib, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Belgium and 8 more
- Volunteers
- 10 people
- Sponsor
- Ipsen
Juvenile Idiopathic Scoliosis, Adolescent Idiopathic Scoliosis
Study of the Braive Growth Modulation System for Progressive Pediatric Scoliosis
The purpose of this study was to establish probable benefits and evaluate the safety and preliminary effectiveness of the Braive™ GMS when used in the treatment of pediatric progressive scoliosis.
- Trial name
- BRAIVE IDE (NCT04929678)
- Testing
- Braive™ Growth Modulation System (Braive™ GMS)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 10 people
- Sponsor
- Medtronic Spinal and Biologics
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Soft Tissue Sarcoma, Primary Central Nervous System Carcinoma
Study of Autologous Tumor-Infiltrating Lymphocytes in Pediatric, Adolescent, and Young Adult Participants
This study is planned to test the safety and tolerability of the TIL regimen. The study will also test how well TIL fights cancer.
- Conditions
- Soft Tissue Sarcoma, Primary Central Nervous System Carcinoma, Melanoma, Rhabdomyosarcoma
- Trial name
- NCT06566092
- Testing
- LN-145/LN-144
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Iovance Biotherapeutics, Inc.
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
Acute Myeloid Leukemia (AML)
Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 Years of Age With Relapsed/Refractory CBFA2T3::GLIS2 AML
This trial will evaluate whether luveltamab tazevibulin is well tolerated and active against a rare form of AML carrying a particular genetic abnormality called CBFA2T3::GLIS2 that arises in infants and children.
- Trial name
- NCT06679582
- Testing
- Luveltamab tazevibulin
- Where
- 36 sites in United States, Austria and 7 more
- Volunteers
- 1 people
- Sponsor
- Sutro Biopharma, Inc.
Fever, Acute Pain
Using a Decision-making System to Support the Management of Fever and Acute Pain in Children
FEVER is a single-center, prospective, open-label, pre-market, pivotal, single-arm study aimed at demonstrating how the use of an algorithm integrated with a Class I Medical Device (Puntofarma®), which is made available through a…
- Trial name
- FEVER (NCT07344090)
- Testing
- Puntofarma® FEVER
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Qwince Innovation S.r.l.
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Antithrombin III Deficiency
Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate
To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.
- Trial name
- NCT00319228
- Testing
- Plasma-derived AT-III concentrate
- Volunteers
- 3 people
- Sponsor
- Instituto Grifols, S.A.
RSV Immunization
Co-administration Study of an Investigational Live-Attenuated Respiratory Syncytial Virus Vaccine in Infants and Toddlers
This study is a Phase III, randomized, single-blind, placebo-controlled, multi-center study to be conducted in healthy infants and toddlers which will be enrolled at approximately 6 months of age (Cohort 1) and approximately 12…
- Trial name
- CORAL (NCT06397768)
- Testing
- RSVt vaccine, Diphtheria, tetanus, pertussis, poliomyelitis , Haemophilus influenzae type b, and hepatitis B vaccine, Diphtheria, tetanus, pertussis, Haemophilus influenzae type b and poliomyelitis vaccine
- Where
- 72 sites in United States, Mexico and 1 more
- Volunteers
- 2,226 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Cataract
Evaluate the Safety of DEXYCU for the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract
A Phase 3/4, Prospective, Randomized, Active Treatment-Controlled, Parallel-Design, Multicenter Study to Evaluate the Safety of DEXYCUfor the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract
- Trial name
- NCT05191706
- Testing
- Dexamethasone, Prednisolone Acetate Ophthalmic
- Where
- 9 sites in United States
- Volunteers
- 17 people
- Sponsor
- EyePoint Pharmaceuticals, Inc.
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
- Trial name
- NCT06046820
- Testing
- INZ-701, Control Arm (Conventional Therapy)
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 27 people
- Sponsor
- Inozyme Pharma
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette…
- Trial name
- NCT06302439
- Testing
- No Intervention for this observational study
- Where
- 14 sites in United States, Canada and 7 more
- Volunteers
- 54 people
- Sponsor
- Inozyme Pharma
Relapsed/Refractory Acute Myeloid Leukemia
Safety and Efficacy of NK520 to Treat Pediatric Relapsed/Refractory Acute Myeloid Leukemia
This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection.
- Trial name
- NCT06541405
- Testing
- NK520
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Base Therapeutics (Shanghai) Co., Ltd.
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