Hopital Necker Enfants Malades
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Digestive System and Pediatric Nutrition
The Service de Gastro-entérologie et Nutrition Pédiatriques at Hôpital Necker Enfants Malades focuses on improving care for children with gastrointestinal disorders and nutritional challenges. Clinical research aims to evaluate new therapies, refine diagnostic tools, and enhance long‑term health outcomes for young patients.
- Investigating novel treatments for Crohn’s Disease and Ulcerative Colitis in pediatric populations
- Assessing nutritional interventions for intestinal malabsorption in preterm infants
- Exploring therapies to reduce proteinuria and preserve renal function in children with kidney‑related digestive complications
- Evaluating safety and efficacy of biologics such as mirikizumab and ixekizumab for inflammatory bowel disease
- Studying the impact of gut‑microbiome modulation on disease activity and growth parameters
These studies aim to bring effective, child‑friendly treatments to the bedside and support better growth, development, and quality of life for affected children.
Genetic and Congenital Growth Disorders
Research at the site addresses rare genetic conditions that affect skeletal development and metabolism. Trials are designed to test innovative gene‑based and peptide therapies that could alter disease trajectories and improve physical outcomes.
- Long‑term safety and growth benefits of TransCon CNP in children with achondroplasia
- Evaluating the efficacy of BMN 111 for height gain in achondroplastic patients
- Assessing infigratinib and other agents for bone growth in pediatric achondroplasia
- Investigating metabolic impacts of therapies for congenital adrenal hyperplasia
- Studying treatment approaches for rare vascular malformations affecting facial and oral structures
By targeting the underlying molecular pathways, these trials seek to provide lasting improvements in stature, endocrine balance, and overall health for children with congenital growth disorders.
Blood, Immune, and Hematologic Disorders
The department conducts extensive studies on pediatric blood and immune diseases, ranging from hemophilia to immune deficiencies. The primary goal is to introduce safer, more effective therapies and to reduce disease‑related complications.
- Evaluating gene‑therapy approaches for severe hemophilia A using AAV vectors
- Testing novel immunoglobulin‑targeting agents in children with autoimmune hemolytic anemia
- Assessing the safety of long‑acting antibodies for HIV remission in early infection
- Studying treatments for EBV‑related post‑transplant lymphoproliferative disease in kidney transplant recipients
- Investigating the efficacy of ravulizumab for transplant‑associated thrombotic microangiopathy
These investigations aim to enhance disease control, lower bleeding risk, and improve transplant outcomes for young patients.
Neurological and Neurodevelopmental Disorders
Pediatric neuro‑oncology and neurodevelopmental conditions are a key focus, with trials exploring targeted therapies and supportive care strategies to improve neurological function and survival.
- Assessing the efficacy of ONC201 in children with diffuse intrinsic pontine glioma and related midline gliomas
- Evaluating radiprodil for seizure reduction in GRIN‑related neurodevelopmental disorder
- Testing early rituximab infusion to prevent EBV infection in pediatric kidney transplant recipients
- Studying gene‑editing therapies for spinal muscular atrophy
- Exploring treatments for type 1 interferonopathies and related autoimmune encephalopathies
The research strives to provide disease‑modifying options that can preserve neurological development and extend survival for children with severe brain disorders.
Metabolic, Endocrine, and Rare Diseases
A broad spectrum of metabolic and rare genetic diseases are under investigation, with an emphasis on innovative drug candidates and precision medicine approaches.
- Testing povetacicept to reduce proteinuria in immunoglobulin A nephropathy
- Evaluating stiripentol for lowering urinary oxalate in primary hyperoxaluria
- Assessing mavorixafor to improve neutrophil counts in chronic neutropenia
- Investigating crinecerfont for adrenal steroid regulation in congenital adrenal hyperplasia
- Studying therapies for metabolic bone disease in children with PIK3CA‑related overgrowth spectrum
These studies aim to deliver targeted treatments that address the root causes of metabolic dysfunctions, offering hope for improved quality of life.
Site Overview
Activity Indicator
Last 6 months
Trial Flow
How this site's trials are distributed across therapeutic areas and active substances (IMPs), and how they're positioned within their overall lifecycle status. Flow thickness represents the number of unique trials.
Top 5 of 26 therapeutic areas and 15 of 205 IMPs, ranked by number of trials.
Compounds & Molecules Timeline
The time span of clinical-trial activity for the main active substances (IMPs) studied at this site over the last 8 years. Each bar runs from the earliest trial start to the most recent end; a substance with a trial still open has no recorded end and is shown as ongoing.
Top 40 of 194 compounds, ranked by number of trials.
Sponsor & Partnership
Top sponsors by active trials, and the ones that have come back for more than one study.
Sponsor Network
Top 61 by active trials.
- 1Pfizer Inc.5active
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Partnership History
Sponsors with repeated collaboration (≥ 2 trials).
- 1Pfizer Inc.10trials
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Sponsor Network Graph
How sponsors and therapeutic areas connect across this site's active trials: each link is at least one active trial shared between them, and bigger nodes mean more activity. Click a node to see its exact connections.
Country Benchmark
Compared to 978 other sites in France
Therapeutic Area Benchmark — France
Select a therapeutic area to compare against other sites in France.
Competitor Comparison
Similar sites in the same country running a comparable number of trials (within ±30%), scored on how closely their trial portfolios match this one. Higher percentages mean a more similar profile.
Country: France
Market Share
Country: France
Therapeutic Area Market Share
Select a therapeutic area to view its disease coverage breakdown.
Heat Maps & Advanced Analytics
How often specific conditions appear in trials across this site and comparable sites from the same country. Each row is a site, each column a condition, and each cell the number of trials on it — darker means more activity.
Other Trials in Hopital Necker Enfants Malades
lock Showing 1 of 154 trials — the rest is available on a paid plan.
| Trial | Sponsor | Phase | Status | Start Date | End Date |
|---|---|---|---|---|---|
| A Multicenter Randomized Non‑Inferiority Trial of Weekly Somatrogon versus Daily Somatropin in Pediatric Patients with Small‑for‑Gestational‑Age or Idiopathic Short Stature | Schneider Children's Medical Center Of Israel | Phase III | Not Yet Recruiting | — | — |
IMP list
lock Showing 1 of 205 compounds — the rest is available on a paid plan.
| IMP | Trials | Status | First start | Last start |
|---|---|---|---|---|
| Alpelisib | 5 | Unknown | 2021-04-19 | 2025-12-11 |
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