LymphangiomaPhase 1/2NCT01290484
A Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations
A Phase 1/2 study testing Sildenafil in people with Lymphangioma, run by Stanford University at 1 site in United States.
Trial summary
There is an unsatisfied medical need for a first-line treatment of lymphatic malformations with a good benefit/risk profile. Based on a patient experience in the institution, the investigators plan to verify whether or not the medication sildenafil has a beneficial effect on lymphatic malformations. Read more about this study
- Who is it for?
- Ages 6 months to 10 years
- Conditions the study is for
- Lymphangioma
- What is being tested?
- Sildenafil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2010to Dec 2013
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 months to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written informed consent(s) for study participation and (where applicable) the use of the participant's images are obtained according to national regulations from the participant's parent(s) or guardian(s) prior to performing any study procedures.
- The participant is 6 months to 10 years of age at inclusion.
- The participant weight is at least 8kg.
- A diagnosis of LM or mixed venous lymphatic malformation involving the skin and subcutaneous tissue and at least 3cm based on clinical and radiographic criteria.
- LMs may benefit from systemic therapy based on clinical criteria.
- Females must not be pregnant or breast-feeding.
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- If participant is a child, parent/guardian must be able to follow instructions and must be willing and able to ensure that the subject is present for all required study visits.
- Subject has no contraindication for use of sildenafil.
- LMs may involve any part of the body.
- Subject will have normal results on screening tests (eye exam, blood tests).
- Subject has no contraindication for MRI examinations, such as metal implants, etc.
- Subject must not be a smoker.
Who cannot join
- The participant has a medically unstable health status that may interfere with his/her ability to complete the study.
- The participant presents with one or more of the following medical conditions: hepatic impairment; severe renal impairment; lymphedema conditions such as Milroy disease, Meige lymphedema, Hennekam syndrome, Njolstad syndrome, Aagenaes syndrome, and Fabry disease; hypotension or at risk for hypotension; seizures or history of seizures; any significant cardiovascular risk factors and any condition which requires participants to use nitric oxide donors or nitrates in any form; underlying anatomic or vascular risk factors for developing non-arteritic anterior ischemic optic neuropathy (NAION) including low ocular cup to disc ratio, age over 10, diabetes, hypertension, coronary artery disease, hyperlipidemia, and smoking. (Participants with Down syndrome, Turner syndrome, and Noonan syndrome will be considered on a case-by-case basis).
- The participant has received at least one of the following medications contraindicated in association with sildenafil within 15 days of inclusion: Alprostadil, Azole antifungals, Clarithromycins, Conivaptan, Delavirdine, Erythromycins, Fluvoxamine, Grapefruit, Imatinib, Nefazodone, Nitrates/sodium thiosulfate, Non-selective and selective alpha blockers, Protease inhibitors, Rufinamide, Tadalafil, Telithromycin, Vardenafil, Yohimbe, Yohimbine, Amifostine, Lapatinib, Warfarin
- The participant requires concomitant use of potent cytochrome P450 3A4 inhibitors (such as ketoconazole, itraconazole, erythromycin, saquinavir) or concomitant use of ritonavir.
- The patient has had extensive prior surgery or sclerotherapy to treat LM such that scarring may interfere with the treatment effect of sildenafil.
- The participant has previously been administered treatment for LMs or surgical procedures have been performed to remove the index LMs.
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- Participant is currently pregnant or considering becoming pregnant in the next 20 weeks.
- The participant is known to have an allergy to sildenafil.
- Ulcerated or currently infected LMs with pain.
- Diagnosis of the soft tissue tumor as LM is not clinically certain.
- The participant is participating in another clinical study.
- The participant has a history of priapism or is diagnosed with sickle cell anemia or any other disorder which may predispose to priapism.
- The investigator may declare any subject ineligible for a valid medical reason.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: An Investigational Pilot Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations
There is an unsatisfied medical need for a first-line treatment of lymphatic malformations with a good benefit/risk profile. Based on a patient experience in the institution, the investigators plan to verify whether or not the medication sildenafil has a beneficial effect on lymphatic malformations. The investigators plan to do this by treating patients with lymphatic malformations with the medication sildenafil for a 20 week period. This is an investigator initiated study funded by an Innovations in Patient Care grant and a SPARK grant.
Read the detailed description
Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications. We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children. Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline. Sildenafil can reduce lymphatic malformation volume and symptoms in some children. ( J Am Acad Dermatol 2014;70:1050-7.)
- Conditions
- Lymphangioma
- Being tested
- Sildenafil
- Keywords
- Lymphatic malformation, vascular malformation
- Medical subject headings
- Lymphangioma, Lymphatic Abnormalities, Vascular Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SildenafilDrug
Also called Revatio, Viagra
Sildenafil oral tablet three times daily
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | Sildenafil oral tablet three times daily Receives: Drug: Sildenafil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Volume of Lymphatic Malformation
Participants were given sildenafil for 20 weeks. Participants weighing more than 20 kg were given 20 mg 3 times daily (60 mg/day). Participants weighing between 8 kg and 20 kg were given 10 mg 3 times daily (30 mg/day).
Measured: Baseline, 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2011 (estimated)
- Study startDec 2010
- Primary completionApr 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LymphangiomaStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alfred Lane, Stanford University
- Investigators
- Alfred T Lane, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01290484
- Sponsor's protocol ID
- SU-10202010-7129
- Other IDs
- UL1TR000093
- Status last verified
- May 2015
- First submitted
- 3 Feb 2011
- Last updated
- 19 Jun 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Danial C, Tichy AL, Tariq U, Swetman GL, Khuu P, Leung TH, Benjamin L, Teng J, Vasanawala SS, Lane AT. An open-label study to evaluate sildenafil for the treatment of lymphatic malformations. J Am Acad Dermatol. 2014 Jun;70(6):1050-7. doi: 10.1016/j.jaad.2014.02.005. Epub 2014 Mar 20.
PubMed 24656411
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigational Pilot Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations
- ClinicalTrials.gov ID
- NCT01290484
- Acronym / sponsor ID
- SU-10202010-7129
- Conditions
- Lymphangioma
- Keywords
- Lymphatic malformation, vascular malformation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Apr 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 7 Feb 2011 (estimated)
- Results
- Posted 12 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There is an unsatisfied medical need for a first-line treatment of lymphatic malformations with a good benefit/risk profile. Based on a patient experience in the institution, the investigators plan to verify whether or not the medication sildenafil has a beneficial effect on lymphatic malformations. The investigators plan to do this by treating patients with lymphatic malformations with the medication sildenafil for a 20 week period. This is an investigator initiated study funded by an Innovations in Patient Care grant and a SPARK grant.
Detailed description
Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications. We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children. Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline. Sildenafil can reduce lymphatic malformation volume and symptoms in some children. ( J Am Acad Dermatol 2014;70:1050-7.)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open Label | Experimental | Drug: SildenafilSildenafil oral tablet three times daily |
Who may qualify
- Ages
- 6 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Written informed consent(s) for study participation and (where applicable) the use of the participant's images are obtained according to national regulations from the participant's parent(s) or guardian(s) prior to performing any study procedures.
- The participant is 6 months to 10 years of age at inclusion.
- The participant weight is at least 8kg.
- A diagnosis of LM or mixed venous lymphatic malformation involving the skin and subcutaneous tissue and at least 3cm based on clinical and radiographic criteria.
- LMs may benefit from systemic therapy based on clinical criteria.
Key exclusion criteria (13 listed)
- The participant has a medically unstable health status that may interfere with his/her ability to complete the study.
- The participant presents with one or more of the following medical conditions: hepatic impairment; severe renal impairment; lymphedema conditions such as Milroy disease, Meige lymphedema, Hennekam syndrome, Njolstad syndrome, Aagenaes syndrome, and Fabry disease; hypotension or at risk for hypotension; seizures or history of seizures; any significant cardiovascular risk factors and any condition which requires participants to use nitric oxide donors or nitrates in any form; underlying anatomic or vascular risk factors for developing non-arteritic anterior ischemic optic neuropathy (NAION) including low ocular cup to disc ratio, age over 10, diabetes, hypertension, coronary artery disease, hyperlipidemia, and smoking. (Participants with Down syndrome, Turner syndrome, and Noonan syndrome will be considered on a case-by-case basis).
- The participant has received at least one of the following medications contraindicated in association with sildenafil within 15 days of inclusion: Alprostadil, Azole antifungals, Clarithromycins, Conivaptan, Delavirdine, Erythromycins, Fluvoxamine, Grapefruit, Imatinib, Nefazodone, Nitrates/sodium thiosulfate, Non-selective and selective alpha blockers, Protease inhibitors, Rufinamide, Tadalafil, Telithromycin, Vardenafil, Yohimbe, Yohimbine, Amifostine, Lapatinib, Warfarin
- The participant requires concomitant use of potent cytochrome P450 3A4 inhibitors (such as ketoconazole, itraconazole, erythromycin, saquinavir) or concomitant use of ritonavir.
- The patient has had extensive prior surgery or sclerotherapy to treat LM such that scarring may interfere with the treatment effect of sildenafil.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SildenafilAlso: Revatio, ViagraSildenafil oral tablet three times daily | Drug | Open Label |
Primary outcome
Change in Volume of Lymphatic Malformation
Time frame: Baseline, 20 weeks
Participants were given sildenafil for 20 weeks. Participants weighing more than 20 kg were given 20 mg 3 times daily (60 mg/day). Participants weighing between 8 kg and 20 kg were given 10 mg 3 times daily (30 mg/day).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jun 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 24656411Danial C, Tichy AL, Tariq U, Swetman GL, Khuu P, Leung TH, Benjamin L, Teng J, Vasanawala SS, Lane AT. An open-label study to evaluate sildenafil for the treatment of lymphatic malformations. J Am Acad Dermatol. 2014 Jun;70(6):1050-7. doi: 10.1016/j.jaad.2014.02.005. Epub 2014 Mar 20.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alfred T Lane, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sildenafil in people with Lymphangioma, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jun 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jun 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Volume of Lymphatic Malformation (time frame: Baseline, 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01290484, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jun 2015.