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OK432 (Picibanil) in the Treatment of Lymphatic Malformations

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ClinicalTrials.gov Identifier: NCT03427619
Recruitment Status : Completed
First Posted : February 9, 2018
Last Update Posted : December 6, 2018
Sponsor:
Information provided by (Responsible Party):
Richard JH Smith, University of Iowa

Brief 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.

Condition or disease Intervention/treatment Phase
Lymphatic Malformations Drug: OK432 Phase 4

Detailed Description:

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%)


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 701 participants
Intervention Model: Single Group Assignment
Intervention Model Description: All eligible participants receive the actual drug.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: OK432 (Picibanil) in the Treatment of Lymphatic Malformations
Actual Study Start Date : January 1, 1998
Actual Primary Completion Date : October 22, 2018
Actual Study Completion Date : October 22, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: OK432 (Picibanil)
There is no control in this study. All participants will receive the actual drug -OK432. With each injection they may receive .1mg-.2mg 6-12 weeks apart up to 4 injections total.
Drug: OK432
OK432 will be injected at dosage of .1mg-.2mg 6-12 weeks apart up to 4 injections total.
Other Name: Picibanil




Primary Outcome Measures :
  1. Determine the response rate of OK432 in children with symptomatic Lymphatic Malformations [ Time Frame: through study completion --approximately the next 5 years ]
    Response rate will be assessed radio-graphically after the final injection using the Clinical Staging Classification System and de Serres staging system.Images will be used and orthogonal measurements of LM size will be used to calculate volume/size in cubic centimeters (cc). Response rate to OK-432 immunotherapy will be recorded as percentage reduction in volume and graded as complete (90%-100% reduction in volume), substantial (60%-89% reduction in volume), incomplete (20%-59% reduction in volume), or none (0%-19% reduction in volume). Clinical success is defined as a complete or substantial response.



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Ages Eligible for Study:   6 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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)

Information from the National Library of Medicine

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


Locations
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United States, Iowa
Richard Smith, MD
Iowa City, Iowa, United States, 52242
Sponsors and Collaborators
Richard JH Smith
Investigators
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Principal Investigator: Richard JH Smith, MD University of Iowa

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Responsible Party: Richard JH Smith, Principal Investigator, University of Iowa
ClinicalTrials.gov Identifier: NCT03427619     History of Changes
Other Study ID Numbers: 201107741
First Posted: February 9, 2018    Key Record Dates
Last Update Posted: December 6, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Congenital Abnormalities
Lymphangioma
Lymphatic Abnormalities
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Lymphatic Diseases
Picibanil
Antineoplastic Agents