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Sildenafil for the Treatment of Lymphatic Malformations
Phase 2 trial testing Sildenafil 20 mg tablets in Lymphatic Malformations in 22 participants. Completed in 30 March 2021.
| Lead sponsor | Stanford University |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | supportive care |
| Enrollment | 22 |
| Start date | 23 May 2015 |
| Primary completion | 30 March 2021 |
| Estimated completion | 30 March 2021 |
| Sites | 3 locations across United States |
Stanford University
Adults 6 Months to 10, any sex, with Lymphatic Malformations or Lymphatic Diseases. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Participants will be followed for the duration of the study, an expected average of 20 weeks.
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 5.89 | ± 13.5 |
| Open-Label Sildenafil | -8.54 | ± 12.1 |
| Double-blind Sildenafil | -0.642 | ± 18.3 |
Subject's evaluation of the overall change in lymphatic malformation. Participants will be followed from baseline to 20 weeks. Patients rated change as no improvement, minimal improvement (1-25% change), fair improvement (25-50% change), good improvement (50-75% change), and excellent improvement (75-100% change).
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 1 | |
| Double-Blind Sildenafil | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 2 | |
| Double-Blind Sildenafil | 4 |
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 0 | |
| Double-Blind Sildenafil | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 2 | |
| Double-Blind Sildenafil | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Double-Blind Placebo | 0 | |
| Double-Blind Sildenafil | 0 |
Time frame: Up to 52 weeks in the Placebo then Open-Label Sildenafil group, and up to 32 weeks in the Sildenafil group. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Double-Blind Placebo | Open-Label Sildenafil | Double-Blind Sildenafil |
|---|---|---|---|---|
| Cold | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Headaches | Nervous system disorders | — | — | — |
| Fever | General disorders | — | — | — |
| Nausea/vomit | Gastrointestinal disorders | — | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Loose stool/increase bowel movement | Gastrointestinal disorders | — | — | — |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Abdominal pain/bloating | Gastrointestinal disorders | — | — | — |
| Constipation | Gastrointestinal disorders | — | — | — |
| Acute gastroenteritis/viral gastroenteritis | Gastrointestinal disorders | — | — | — |
| Intermittent flushing | Vascular disorders | — | — | — |
| Cellulitis | Infections and infestations | — | — | — |
| Ear pain | Ear and labyrinth disorders | — | — | — |
Data from ClinicalTrials.gov NCT02335242 adverse events section.
A Phase 2 study to evaluate safety and efficacy of sildenafil taken orally to improve or resolve lymphatic malformations in children. Subjects may receive either placebo or treatment in an oral dosage with an open label extension for subjects who received placebo. The study treatment assignment will be randomized in a double blind fashion. MRI examination will evaluate change in lesion volume due to treatment. Other safety and efficacy measures will be taken through the 32-week study duration. Funding Source - FDA OOPD
4 peer-reviewed publications reference this trial (live from Europe PMC):
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