Clinical trials: enrolling by invitation
- Status Enrolling by invitation
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Atopic Dermatitis, Eczema
A Study to Assess the Long-Term Safety and Efficacy of Lebrikizumab (LY3650150) in Participants 6 Months to <18 Years of Age With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to assess the long-term safety and efficacy of lebrikizumab in participants 6 Months to <18 years of age with moderate-to-severe atopic dermatitis.
- Trial name
- ADorable-2 (NCT05735483)
- Testing
- Lebrikizumab
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 338 people
- Sponsor
- Eli Lilly and Company
Hematologic Malignancies, Solid Tumors
Follow-Up for Study Participants Treated With an Allogeneic CAR T Cell Product
An observational, long-term follow up (LTFU) study of participants who received an allogeneic CAR T product in a prior clinical study.
- Trial name
- NCT06925685
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Allogene Therapeutics
Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment…
- Trial name
- ENDURE (NCT06270719)
- Testing
- Delandistrogene Moxeparvovec, Standard of Care
- Where
- 29 sites in United States
- Volunteers
- 500 people
- Sponsor
- Sarepta Therapeutics, Inc.
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101.
- Trial name
- NCT06657859
- Testing
- GLM101
- Where
- 15 sites in United States, Czechia and 6 more
- Volunteers
- 90 people
- Sponsor
- Glycomine, Inc.
Deep Vein Thrombosis
ClotTriever® Thrombectomy System Use-Result Survey in Japan
The objective of this survey is to determine the safety and efficacy of the ClotTriever Thrombectomy System under daily clinical practice after marketing approval in Japan.
- Trial name
- NCT06871683
- Testing
- ClotTriever® Thrombectomy System
- Where
- 15 sites in Japan
- Volunteers
- 100 people
- Sponsor
- Inari Medical
Female Athletes, Healthy Women
A Descriptive Analysis of the Physiological and Behavioral Profiles of Elite Female Athletes
In May 2026, the Gatorade Brand launched the "Body of Science" (BoS), a multi-year research initiative aimed at closing the gender gap in sports science.
- Trial name
- NCT07883278
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- PepsiCo Global R&D
Heart Diseases, Heart Failure
Global Utilization And Registry Database for Improved heArt preservatioN
The primary objective of this study is to collect real-world clinical performance data to assess the clinical outcomes of patients receiving heart transplants using donor hearts transported via the SherpaPak CTS System.
- Conditions
- Heart Diseases, Heart Failure, Heart Defects, Congenital
- Trial name
- GUARDIAN (NCT04141605)
- Testing
- SherpaPak Cardiac Transport System
- Where
- 22 sites in United States, Austria and 2 more
- Volunteers
- 4,000 people
- Sponsor
- Paragonix Technologies
Systemic Lupus Erythematosus
A Study to Evaluate the Safety and Tolerability of Dapirolizumab Pegol in Study Participants With Systemic Lupus Erythematosus
The purpose of this study is to evaluate long-term safety and tolerability of dapirolizumab pegol treatment.
- Trial name
- NCT04976322
- Testing
- Dapirolizumab pegol
- Where
- 123 sites in United States, Argentina and 22 more
- Volunteers
- 760 people
- Sponsor
- UCB Biopharma SRL
Developmental and Epileptic Encephalopathy
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
This (DEEp OLE Study) is a multicentre, open-label study to investigate the long-term safety, efficacy, tolerability, and pharmacokinetics (PK) of LP352 in the treatment of seizures in children and adults with DEE who completed…
- Trial name
- NCT06908226
- Testing
- LP352
- Where
- 88 sites in United States, Australia and 15 more
- Volunteers
- 324 people
- Sponsor
- Longboard Pharmaceuticals
Prader-Willi Syndrome
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with PWS treated with VYKAT XR.
- Trial name
- NCT07450664
- Testing
- VYKAT XR
- Where
- 25 sites in United States
- Volunteers
- 200 people
- Sponsor
- Soleno Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
ADHD
An Open-Label Study to Assess the Long-Term Safety of Solriamfetol in Children and Adolescents With ADHD
This study is a Phase 3, multi-center, open-label trial to evaluate the long-term safety of solriamfetol in children and adolescents with ADHD.
- Trial name
- NCT07773883
- Testing
- Solriamfetol
- Where
- 7 sites in United States
- Volunteers
- 300 people
- Sponsor
- Axsome Therapeutics, Inc.
Craniofacial Defects
Long-term Safety and Performance of CMF Porous Titanium Implants
The goal of this retrospective study is to evaluate the clinical characteristics, efficiency, safety, and long-term performance of craniomaxillofacial (CMF) Porous Titanium Implants within the standard of care across children (0…
- Trial name
- CMF SafeTi Fit (NCT06910033)
- Testing
- Craniomaxillofacial (CMF) porous titanium implant
- Where
- 2 sites in Denmark, France
- Volunteers
- 200 people
- Sponsor
- Materialise
Open-Angle Glaucoma
A Study of Outcomes Following Bio-Reinforced Canaloplasty in Eyes With Open-Angle Glaucoma
The goal of this study is to learn about outcomes after bio-reinforced canaloplasty surgery in patients with OAG. The key question the study aims to answer is: How much does the surgical procedure lower pressure inside the eye…
- Trial name
- BOOST (NCT07520175)
- Testing
- AlloSpan Canal Platform Bio-Reinforced Canaloplasty
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- Iantrek, Inc.
Temporomandibular Joint Disorders
Long-term Safety and Performance of Materialise's Patient-specific TMJ System.
This is a long-term (10 years), retrospective-prospective, observational, single-arm, monocenter study of patients to be treated with the Materialise TMJ Total Arthroplasty System for TMJ replacement.
- Trial name
- NCT06951178
- Testing
- TMJ Total Arthroplasty System (TMJ System)
- Where
- Denmark
- Volunteers
- 30 people
- Sponsor
- Materialise
Erythropoietic Protoporphyria
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
- Trial name
- NCT05883748
- Testing
- DISC-1459
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 230 people
- Sponsor
- Disc Medicine, Inc
Multiple System Atrophy
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the…
- Trial name
- NCT07729852
- Testing
- ATH434
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Alterity Therapeutics
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Participants are free to decide if they want to take part in this study or not. The study will be conducted to collect information about the influence of adherence to growth hormone therapy with Norditropin® in children and…
- Trial name
- NCT03972345
- Testing
- Norditropin® FlexPro®
- Where
- Germany
- Volunteers
- 750 people
- Sponsor
- Novo Nordisk A/S
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
The goal of this real world data study is to evaluate the clinical utility of remote patient monitoring solution (using Byteflies' EpiCare@Home with reduced EEG montage and other vital signs) with Dravet and LGS patients (ages 3…
- Trial name
- TETRIS (NCT07225231)
- Testing
- Byteflies Remote patient monitoring solution (with reduced EEG)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Byteflies