Lymphatic MalformationNCT02399527
Lymphatic Anomalies Registry for the Assessment of Outcome Data
An observational study of Lymphatic Malformation and Generalized Lymphatic Anomaly (GLA), run by Boston Children's Hospital at 1 site in United States.
Trial summary
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical therapies and procedures is limited.The goal of this project is to better understand these… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly
- How many people
- 1,000 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to Jun 2035
- How long will I take part?
- 15 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Both domestic and international patients with complex lymphatic anomalies, as described above, are eligible to participate. Travel to Boston is not required for participation. Eligible patients are identified through active referral in the Vascular Anomalies Center at Boston Children's Hospital. Physicians and patients may also refer eligible patients directly to the Lymphatic Anomalies Registry. Patients may indicate interest in participation themselves by contacting the registry team through the "Contact Us" link on www.lymphaticregistry.org.
Who may be able to join
- Clinical diagnosis of complex vascular tumor, malformation or overgrowth syndrome with significant lymphatic component
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Meghan O'Hare, CPNP, 617-355-5226, Lymphaticregistry@childrens.harvard.edu
Kelsey Deemer, 617-355-5226, Lymphaticregistry@childrens.harvard.edu
About this study
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical therapies and procedures is limited.The goal of this project is to better understand these diseases and improve the care of theses rare patients. To do this, the investigators are conducting an observational study of patients with lymphatic anomalies, including an annual follow-up questionnaire to gather prospective data on mortality, morbidity, treatments, and functionality as well as quality of life.
Read the detailed description
The purpose of the Lymphatic Anomalies Registry, created at Boston Children's Hospital, is to create a database to help current and future patients diagnosed with a lymphatic anomaly. The ultimate goal of the registry is to better understand and predict responses to therapies and risk factors for complications. Although the Lymphatic Anomalies Registry exists at Boston Children's Hospital, patients can be entered into the registry regardless of whether or not they visit Boston Children's Hospital, thus increasing the program's accessibility. The Lymphatic Anomalies Registry includes patients who have vascular anomalies with a lymphatic component across various diagnoses. From the patient's perspective, participation in the Lymphatic Anomalies Registry means taking part in a short, thirty minute interview, and providing the registry with access to medical records. The interview is conducted verbally with study staff of the Lymphatic Anomalies Registry, and can take place either at the hospital, or over the phone. During the interview, the registry will inquire about the patient's diagnosis, disease features, medical therapies, and procedures. Interested prospective patients will receive an introductory packet from the registry with information on how to proceed in the registry process. All obtained patient information is housed on a secure, HIPPA compliant, internal database, managed by Boston Children's Hospital staff. Patient information entered into the external database is de-identified. The research teams will also obtain a medical record release form to request the patient's medical record for review in our study.
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome, Gorham-Stout Disease ("Disappearing Bone Disease"), Blue Rubber Bleb Nevus Syndrome
- Keywords
- Vascular Anomalies, Lymphatic Anomalies, Lymphatic Malformation
- Medical subject headings
- Lymphatic Abnormalities, Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi, Osteolysis, Essential, Blue rubber bleb nevus syndrome, Kaposiform Hemangioendothelioma, Tufted angioma, Klippel-Trenaunay-Weber Syndrome, Vascular Malformations
What is being measured
The primary outcome is the main question the study is designed to answer.
To characterize the heterogeneity of lymphatic disorders, including demographics, presentation, and complications.
Measured: 15 years
To identify factors that are prognostic of the occurrence of complications, including effusions, coagulopathy, ectatic draining veins, prior infections, visceral involvement, bone involvement, and development of cardiopulmonary symptoms.
Measured: 15 years
To identify factors prognostic of poor outcome and use them to develop "staging" of lymphatic anomalies.
Measured: 15 years
To describe the natural history of lymphatic anomalies, including morbidity and mortality.
Measured: 15 years
To describe the therapies (medical and procedural), adverse events and responses to therapy in patients with lymphatic anomalies.
Measured: 15 years
To pilot quality of life, functional assessment and pain scoring tools in this patient population.
Measured: 15 years
Secondary outcomes (2)
To estimate the proportion of time that patients with lymphatic anomalies have affected offspring.
Measured: 15 years
To assess for correlations of pregnancy complications or medications taken during pregnancy with the development of lymphatic anomalies.
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2015 (estimated)
- Study startJun 2013
- Primary completionJun 2035 (estimated)
- Study completionJun 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Lymphatic MalformationStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Lymphatic Malformation Institute
- Responsible party
- Principal investigator, Marilyn Liang, Boston Children's Hospital
- Investigators
- Marilyn Liang, M.D., Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02399527
- Sponsor's protocol ID
- P00007182
- Status last verified
- Apr 2026
- First submitted
- 17 Mar 2015
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Rankin H, Zwicker K, Trenor CC 3rd. Caution is recommended prior to sildenafil use in vascular anomalies. Pediatr Blood Cancer. 2015 Nov;62(11):2015-7. doi: 10.1002/pbc.25600. Epub 2015 May 15.
PubMed 25982365Croteau SE, Kozakewich HP, Perez-Atayde AR, Fishman SJ, Alomari AI, Chaudry G, Mulliken JB, Trenor CC 3rd. Kaposiform lymphangiomatosis: a distinct aggressive lymphatic anomaly. J Pediatr. 2014 Feb;164(2):383-8. doi: 10.1016/j.jpeds.2013.10.013. Epub 2013 Nov 16.
PubMed 24252784Strychowsky JE, Rahbar R, O'Hare MJ, Irace AL, Padua H, Trenor CC 3rd. Sirolimus as treatment for 19 patients with refractory cervicofacial lymphatic malformation. Laryngoscope. 2018 Jan;128(1):269-276. doi: 10.1002/lary.26780. Epub 2017 Aug 7.
PubMed 28782106