Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04888936 · ClinicalTrials.gov registry record
Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies
A clinical trial of Noonan Syndrome and Legius Syndrome, sponsored by National Cancer Institute (NCI).
- Recruiting
- Registry status
- 500
- Enrollment target
- 2
- Study locations
NCT04888936: Recruiting study of Noonan Syndrome and Legius Syndrome, sponsored by National Cancer Institute (NCI).
NCT04888936 is a study of Noonan Syndrome and Legius Syndrome that is actively recruiting participants, run by National Cancer Institute (NCI). The registered enrollment target is 500 participants, above the 293-participant average among 4 other Noonan Syndrome trials with a reported enrollment target (71% higher). The trial reports 2 study locations across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04888936, a study of Noonan Syndrome and Legius Syndrome, is actively recruiting participants, sponsored by National Cancer Institute (NCI).
- RECRUITING
- Registry status
- 500 participants
- Enrollment target
- 2
- Study locations
Study Summary
Background: RASopathies are a group of conditions caused by a genetic change. People with a RASopathy may have developmental issues, cognitive disability, poor growth, and birth defects. They may also have an increased risk for developing cancer. Researchers want to learn more. Objective: To learn more about RASopathies, how genes and environmental factors contribute to cancer development in people with RASopathies, and the best way to find these cancers and other conditions early or prevent them. Eligibility: People of any age who have or may have a RASopathy, and their family members. Design: Participants will complete questionnaires about their personal and family medical history. Their medical records will be reviewed. Participants will give blood and urine samples. They will give a saliva or cheek cell sample. Some samples will be used for genetic testing. Participants may have a skin biopsy. Participants may have a physical exam by the RASopathies study team. They may also have exams by additional specialists, such as dentists; urologists; ear, nose, and throat doctors; and neurologists. Participants may have computed tomography of the face and mouth. They may have an ultrasound of the abdomen. They may have a bone density scan. They may have skeletal and/or spine x-rays. They may have magnetic resonance imaging of the brain, low back, chest, and/or heart. They may be photographed. Participants may have other tests, such as sleep, brain and heart electrical activity, speech and swallow, metabolism, hearing, eye, and colon function tests. Participants may sign separate consent forms for some tests. Participation will last indefinitely. Participants may be contacted once in a while by phone or mail. They may have follow-up visits.
Primary Outcome
To establish a longitudinal cohort of participants with a clinical diagnosis of a RASopathy and/or a pathogenic germline variation in a Ras/MAPK pathway gene (excluding NF1).
Conditions Studied
Study Locations (2)
Maryland
- National Institutes of Health Clinical Center - Bethesda
- National Cancer Institute - Shady Grove - Rockville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 500 participants |
| Start Date | 2022-04-25 |
| Est. Completion | 2035-01-31 |
What NCT04888936 shows while recruiting
NCT04888936 is an observational study that tracks outcomes without assigning an intervention. The registered 500 participants enrollment target is mid-sized for trials with a published cap, above the 293-participant average among 4 other Noonan Syndrome trials with a reported enrollment target (71% higher).
The record links to 6 conditions, with Noonan Syndrome appearing as the primary indexed condition, and to 0 interventions.
NCT04888936 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT04888936 about?
NCT04888936 is a clinical study titled "Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies". Background: RASopathies are a group of conditions caused by a genetic change. People with a RASopathy may have developmental issues, cognitive disability, poor growth, and birth defects. They may also have an increased risk for developing cancer. Researchers want to learn more. Objective: To learn...
What is the current status of trial NCT04888936?
This trial is currently recruiting. The enrollment target is 500 participants. The study started on 2022-04-25. Estimated completion is 2035-01-31.
What conditions does trial NCT04888936 study?
This clinical trial studies the following conditions: Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Capillary Arteriovenous Malformation Syndrome.
Who is sponsoring clinical trial NCT04888936?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04888936 being conducted?
This trial has 2 study locations across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Noonan Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
RECRUITING · NA
-
RASopathy Biorepository
RECRUITING
-
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
RECRUITING · Phase 2
-
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
ACTIVE NOT RECRUITING · Phase 3
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00001230 · 500 participants
Host Response to Infection and Treatment in Filarial Diseases
- NCT00001529 · 500 participants
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
- NCT00697411 · 500 participants
Study of Selected X-Linked Disorders: Aicardi Syndrome
- NCT00903110 · 500 participants
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI