International PPB/DICER1 Registry

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
Children's Hospitals and Clinics of Minnesota
Study ID
NCT03382158
Status
Recruiting
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Conditions

  • Ciliary Body Medulloepithelioma
  • Cystic Nephroma
  • DICER1 Syndrome
  • Embryonal Rhabdomyosarcoma
  • Embryonal Rhabdomyosarcoma of Cervix
  • Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)
  • Embryonal Rhabdomyosarcoma of Vagina (Diagnosis)
  • Gynandroblastoma
  • Nasal Chondromesenchymal Hamartoma
  • Neuroblastoma
  • Nodular Hyperplasia of Thyroid
  • Ovarian Sarcoma
  • Pineoblastoma
  • Pituitary Cancer
  • Pleuropulmonary Blastoma
  • Renal Sarcoma
  • Sertoli-Leydig Cell Tumor
  • Thyroid Carcinoma
  • Wilms Tumor

Eligibility Criteria

Sex
ALL
Age
0 Minutes - 100 Years
Healthy Volunteers
Accepted

Study Details

Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.

Key Dates

Start date
Dec 6, 2016
Status verified
Jan 2025
Primary completion
Dec 6, 2030
Completion
Dec 6, 2035

Study Design

Enrollment
3,400 participants (estimated)

Arms

  • Arm: Type I PPB
    Type I PPB is an early manifestation of this malignant disease, cured in some cases by surgery. Surgical guidelines are presented. It is unknown whether adjuvant chemotherapy improves cure rates for individuals with Type I PPB. If the treating physicians select adjuvant chemotherapy treatment, chemotherapy options include a 22-week regimen: 4 courses of vincristine, actinomycin D and cyclophosphamide (VAC) followed by 3 courses of vincristine and actinomycin D (VA). Therapy decisions are the responsibility of the treating institution.
  • Arm: Types II and III PPB
    Types II and III PPB are aggressive sarcomas. Surgery and chemotherapy are necessary in all cases. Surgical guidelines are presented. Many children with Types II or III PPB receive a single-arm multi-agent chemotherapy neo-adjuvant/adjuvant regimen of IVADo (ifosfamide, vincristine, actinomycin, doxorubicin) for 36 weeks. Second and possible 3rd look surgery may be considered for local control. Radiation therapy may be considered. Specific therapy decisions are the responsibility of the treating institution.
  • Arm: Type Ir PPB
    Type Ir (regressed) PPB is a unique, purely cystic tumor which lacks a primitive cell component. The International PPB/DICER1 Registry will enroll and follow participants with Type Ir PPB, regardless of age.
  • Arm: DICER1 Gene or Cond Assoc with DICER1
    PPB and the associated conditions found in PPB families suggest a familial tendency to formation of tumors. The International PPB/DICER1 Registry for PPB, DICER1 and Associated Conditions study will enroll and follow participants who have the DICER1 gene mutations or conditions associated with PPB or DICER1.

Primary Outcome Measure

Event-free survival [ Time Frame: 7 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's MinnesotaMinneapolisMinnesota55404
Paige HR Mallinger, MS
[email protected]
612-813-7115
Anne K Harris, MPH
[email protected]
6128137121
Kris Ann P Schultz, MD (PRINCIPAL_INVESTIGATOR)

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