OK432 (Picibanil) in the Treatment of Lymphatic Malformations
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|ClinicalTrials.gov Identifier: NCT03427619|
Recruitment Status : Completed
First Posted : February 9, 2018
Results First Posted : October 28, 2021
Last Update Posted : November 23, 2021
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.
|Condition or disease||Intervention/treatment||Phase|
|Lymphatic Malformations||Drug: OK432||Phase 2|
Lymphatic malformations are uncommon tumors that represent localized malformations in the development of the lymphatic system. They typically present in children under 2 years of age and in almost 50% of the cases, are diagnosed at birth. There is neither a racial nor a sexual tendency. The malformations can occur anywhere on the body, but typically they are in the head/neck area.
Morbidity can be significant. Besides the obvious cosmetic deformity caused by these tumors, there is risk of infection and airway compromise and even obstruction. However, effective therapeutic options are limited. Small lesions can be observed, although spontaneous resolution is unlikely. For larger lesions, surgery has been the traditional form of therapy. In the head and neck, in particular, lymphangiomas typically wrap themselves around major neurovascular structures, making total excision removal difficult, if not impossible, and thus the likelihood of recurrence is quite high. Because of these surgical limitations, alternate therapies have been considered; including cryotherapy, diathermy, and chemical sclerotherapy.
The investigators experience with using the drug for macrocystic disease(large cysts) since 1992 in the United States has been very promising compared to traditional surgery. Recurrence rate to date, has been very minimal as well. (<2%)
After the conclusion of the Phase 2 randomized study, all new subjects who presented with an LM and were eligible for treatment were treated under an open-label protocol for continued access to OK-432. This multicenter, open label study enrolled subjects between September 2005 and November 2017.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||275 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||All eligible participants receive the actual drug.|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Multicenter, Open Label Study to Evaluate the Efficacy and Safety of OK-432 Immunotherapy in Individuals With Lymphatic Malformations|
|Actual Study Start Date :||October 5, 2005|
|Actual Primary Completion Date :||April 30, 2018|
|Actual Study Completion Date :||April 30, 2018|
Experimental: OK432 (Picibanil)
There is no control in this study. All participants will receive the actual drug -OK432. With each injection they may receive 0.01 to 0.05mg/mL 6-12 weeks apart up to 4 injections total.
OK432 will be injected at dosage of 0.01 to 0.05 mg/mL 6-12 weeks apart up to 4 injections total.
Other Name: Picibanil
- Number of Participants With Clinical Success at 1 to 6 Months Post-Therapy as Assessed by Imaging [ Time Frame: 1 to 6 Months Post-Therapy ]Clinical success was defined as having either a complete (90% 100%) or substantial (60% 89%) reduction in lymphatic malformation (LM) volume after treatment. Response was determined using post treatment imaging studies at approximately 1 to 6 months after completion of treatment
- Number of Participants With Clinical Response 1 to 6 Months Post-Therapy as Assessed by Imaging [ Time Frame: 1 to 6 Months Post-Therapy ]Number of participants who demonstrated a complete (90%-100% reduction in LM volume), substantial (60%-89% reduction in LM volume), intermediate (20%-59% reduction in LM volume), or no (< 20% reduction in LM volume) response 1 to 6 months post-therapy as assessed by imaging
- Number of Participants With Investigator-Evaluated Overall Response [ Time Frame: 1 to 6 Months Post-Therapy ]Investigator evaluated post-therapy clinical response based on physical exam and/or ultrasound was categorized as "Clinical Improvement" or "No Change" in the size of the cyst.
- Change From Baseline in Lesion Volume [ Time Frame: Baseline and 1 to 6 Months Post-Therapy ]Percent change from baseline in lesion volume - pre-therapy to post therapy assessed by imaging.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03427619
|United States, California|
|Rady Children's Hospital & Health Center San Diego|
|San Diego, California, United States, 92123|
|United States, Colorado|
|The Children's Hospital of Denver|
|Denver, Colorado, United States, 80218|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Florida|
|All Children's Hospital|
|Saint Petersburg, Florida, United States, 33701|
|United States, Iowa|
|Richard Smith, MD|
|Iowa City, Iowa, United States, 52242|
|United States, Michigan|
|Spectrum Health-SHMG Ear, Nose, & Throat|
|Grand Rapids, Michigan, United States, 49546|
|United States, Minnesota|
|Children's Hospitals & Clinics of Minnesota - Minneapolis|
|Minneapolis, Minnesota, United States, 55404|
|United States, New York|
|SUNY Health Science Center|
|Syracuse, New York, United States, 13210|
|United States, Oregon|
|Oregon Health Sciences University|
|Portland, Oregon, United States, 97239|
|United States, Tennessee|
|Vanderbilt University Hospital|
|Nashville, Tennessee, United States, 37232|
|United States, Texas|
|Children's ENT of Houston|
|Houston, Texas, United States, 77030|
|United States, Virginia|
|Children's Hospital of the Kings Daughter|
|Norfolk, Virginia, United States, 23507|
|United States, Wisconsin|
|University of Wisconsin Hospital & Clinic|
|Madison, Wisconsin, United States, 53279|
|Children's Hospital of Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Principal Investigator:||Richard JH Smith, MD||University of Iowa|