Trial Outcomes & Findings for A Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations (NCT NCT01290484)
NCT ID: NCT01290484
Last Updated: 2015-06-19
Results Overview
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).
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
7 participants
Primary outcome timeframe
Baseline, 20 weeks
Results posted on
2015-06-19
Participant Flow
Participant milestones
| Measure |
Sildenafil
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).
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|---|---|
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Overall Study
STARTED
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7
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Overall Study
COMPLETED
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7
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations
Baseline characteristics by cohort
| Measure |
Sildenafil
n=7 Participants
Male and female subjects between the ages of 6 months and 10 years, weighing at least 8 kg and had been given a diagnosis of a lymphatic malformation of at least 3 cm based on clinical and radiologic criteria. Macrocystic, microcystic, or mixed lymphatic malformations involving any location on the body were included. Lymphatic malformations associated with an incomplete response to previous treatments, a risk of functional or aesthetic impairment, or local complications were included.
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|---|---|
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Age, Continuous
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51.3 months
n=5 Participants
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Sex: Female, Male
Female
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3 Participants
n=5 Participants
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Sex: Female, Male
Male
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4 Participants
n=5 Participants
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Region of Enrollment
United States
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7 participants
n=5 Participants
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Weight
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17.0 Kilograms
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline, 20 weeksParticipants 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).
Outcome measures
| Measure |
Sildenafil
n=7 Participants
The primary outcome was the effect of sildenafil on lymphatic malformation volume. Response to sildenafil was characterized by any decrease in lymphatic malformation volume. Lymphatic malformations volumes were assessed blindly by MRI volume segmentation analysis at baseline and after 20 weeks of sildenafil. 4 subjects had a lymphatic malformation volume decrease.
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|---|---|
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Change in Volume of Lymphatic Malformation
Subject 1
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3.7 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 2
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-4.3 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 3
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-1.0 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 4
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-31.7 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 5
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-21.7 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 6
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1.1 percentage of volume change
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Change in Volume of Lymphatic Malformation
Subject 7
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29.6 percentage of volume change
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Adverse Events
Sildenafil
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Sildenafil
n=7 participants at risk
Adverse events reported while one sildenafil were minimal. All subjects tolerated the prescribed medication dose. One subject developed an upper respiratory tract infection and experienced temporary hearing loss due to fluid accumulation. This was resolved completely and she experienced no further hearing loss while on sildenafil. Four parents requested to have the child continue sildenafil after study completion.
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|---|---|
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Gastrointestinal disorders
Nausea/indigestion
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57.1%
4/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Respiratory, thoracic and mediastinal disorders
Rhinorrhea
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42.9%
3/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Respiratory, thoracic and mediastinal disorders
Cough
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42.9%
3/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Psychiatric disorders
Mild insomnia
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42.9%
3/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Investigations
Fever
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28.6%
2/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Gastrointestinal disorders
Emesis
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28.6%
2/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Gastrointestinal disorders
Diarrhea
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28.6%
2/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Skin and subcutaneous tissue disorders
Flushing
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Blood and lymphatic system disorders
Epistaxis
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Reproductive system and breast disorders
Prolonged erections
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Infections and infestations
Temporary hearing illness
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Skin and subcutaneous tissue disorders
Photosensitivity
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Blood and lymphatic system disorders
Bleeding into lymphangioma circumscriptum
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14.3%
1/7 • 20 weeks on sildenafil and 12 weeks after sildenafil
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).
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place