Phase 2
N=275
OK432 (Picibanil) in the Treatment of Lymphatic Malformations
Lymphatic Malformations
Bottom Line
View on ClinicalTrials.gov: NCT03427619 ↗Enrolled (actual)
275
Serious AEs
4.0%
Results posted
Oct 2021
Primary outcome: Primary: Number of Participants With Clinical Success at 1 to 6 Months Post-Therapy as Assessed by Imaging — 57 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- OK432 (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Richard JH Smith
- Primary completion
- Apr 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Clinical Success at 1 to 6 Months Post-Therapy as Assessed by Imaging |
57 | — |
| SECONDARY Number of Participants With Clinical Response 1 to 6 Months Post-Therapy as Assessed by Imaging |
43; 14; 5; 16 | — |
| SECONDARY Number of Participants With Investigator-Evaluated Overall Response |
80 | — |
| SECONDARY Change From Baseline in Lesion Volume |
-44.92 | — |
Summary
Standard of care for Lymphatic Malformations has been surgical excision. We have been using OK432/Picibanil (generously supplied by Chugai Pharmaceuticals in Japan) since 1992 with great success for macrocystic disease.
The objective of the study was to provide OK-432 immunotherapy to subjects with macrocystic or mixed (> 50% macrocystic) lymphatic malformations (LMs) and investigate the efficacy and safety of OK 432 as a treatment option in subjects with LMs.
Eligibility Criteria
Inclusion Criteria
To be eligible to receive OK432 immunotherapy
- Patients must be ages 6 months to 17 years
- Patients must have a macrocystic Lymphatic Malformation
- Patients may have had surgical treatment for their Lymphatic Malformation
- Patients must have an imaging study to confirm the diagnosis of a macrocystic or mixed Lymphatic Malformation An MRI is preferred over a CT scan (an ultrasound may be used between injections if warranted, however an MRI or CT should be done pre and post treatment)
Exclusion Criteria
- Penicillin allergy
- Women who are pregnant or nursing
- Patients who present with a temperature of 100.5 degrees F or greater
- Patients with mixed hemangioma-lymphangioma lesions
- Patients with a history OR a family history of rheumatic heart disease or post-streptococcal glomerulonephritis
- Patients with hemodynamic instability and respiratory failure
- Patients with a history OR a family history of obsessive-compulsive, tic disorders, or PANDA (pediatric autoimmune neuro-psychiatric disorder associated with streptococcal infections)
- Patients who demonstrate abnormalities in the history, physical examination or laboratory analysis which may indicate significant hepatic, hematologic, or renal disease
- Patients who are not in "good general health" (including patients with congenital disorders, chronic diseases, immunologic dysfunction, transplant recipients)
Data sourced from ClinicalTrials.gov (NCT03427619). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.