Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

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Brief Title

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

Official Title

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

Brief Summary

      This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1)
      patients with plexiform neurofibromas using new response assessment modalities with the
      secondary goals of assessing Gleevec toxicity, and characterizing markers of response. The
      rationale for this study arises from the response of human and murine NF1 cells to Gleevec®
      in vitro, the response of a NF1 patient treated with Gleevec® for airway compression by a
      plexiform neurofibroma with a dramatic response not previously seen in NF1 therapy, and the
      experience in 37 NF1 patients treated with Gleevec® in the initial pilot study. Gleevec will
      be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA > 1.8 m2
      or 55 mg/m2 twice daily for patients with BSA < 1.8 m2. For patients with a BSA > 1.8 m2 the
      dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum
      dose of 400 mg bid. For patients with a BSA < 1.8 m2 the dose will increase by increments of
      55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid. Treatment
      will continue for 6 months with an option to continue for 24 months if the patient is
      deriving a clinical benefit.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Disease Response


Condition

Neurofibromatosis

Intervention

Gleevec

Study Arms / Comparison Groups

 Gleevec
Description:  Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA > 1.8 m2 or 55 mg/m2 twice daily for patients with BSA < 1.8 m2. For patients with a BSA > 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA < 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

21

Start Date

February 2012

Completion Date

December 2016

Primary Completion Date

August 2016

Eligibility Criteria

        Inclusion criteria

          1. Patients > 3 years of age.

          2. Diagnosis of neurofibromatosis type 1 (NF1).

          3. Presence of clinically significant plexiform neurofibromas (biopsy proven if possible
             with tissue blocks available); defined as tumors that are potentially life threatening
             or are impinging on vital structures or significantly impair the quality of life from
             pain or other symptoms.

          4. Patients must have measurable disease by magnetic resonance imaging (MRI).

          5. Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of >
             2 months.

          6. Adequate end organ function, defined as the following:

             total bilirubin < 1.5 x ULN, SGOT and SGPT < 2.5 x UNL, creatinine < 1.5 x ULN, ANC >
             1.5 x 109/L, platelets > 100 x 109/L.

          7. Patients must be able to swallow whole pills.

          8. Female patients of childbearing potential must have negative pregnancy test within 7
             days before initiation of study drug dosing. Postmenopausal women must be amenorrheic
             for at least 12 months to be considered of non-childbearing potential. Male and female
             patients of reproductive potential must agree to employ an effective barrier method of
             birth control throughout the study and for up to 3 months following discontinuation of
             study drug.

          9. Written, voluntary informed consent.

        Exclusion criteria

          1. Patient has received any other investigational agents within 28 days of first day of
             study drug dosing, unless the disease is rapidly progressing.

          2. Patient is < 5 years free of another primary malignancy except: if the other primary
             malignancy is not currently clinically significant nor requiring active intervention,
             or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in
             situ. Existence of any other malignant disease is not allowed.

          3. Patient with Grade III/IV cardiac problems as defined by the New York Heart
             Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6
             months of study)

          4. Female patients who are pregnant or breast-feeding.

          5. Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes,
             chronic renal disease, or active uncontrolled infection).

          6. Patient has a known brain metastasis. Non-specific CNS changes on MRI/CT
             characteristic of NF1 are allowed, but not known CNS malignancies.

          7. Patient has known chronic liver disease (i.e., chronic active hepatitis, and
             cirrhosis).

          8. Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.

          9. Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C)
             prior to study entry, unless the disease is rapidly progressing.

         10. Patient previously received radiotherapy to greater than or equal to 25 % of the bone
             marrow

         11. Patient had a major surgery within 2 weeks prior to study entry.

         12. Patient with any significant history of non-compliance to medical regimens or with
             inability to grant reliable informed consent.

         13. Patients who have or anticipate receiving permanent (or semi-permanent) metallic
             structures attached to their body. (e.g., braces on teeth, body piercings), which
             their physicians believe will interfere with the MRI.
      

Gender

All

Ages

3 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Kent Robertson, MD PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01140360

Organization ID

NF/Gleevec DOD Trial

Secondary IDs

0908-09

Responsible Party

Sponsor-Investigator

Study Sponsor

Kent Robertson


Study Sponsor

Kent Robertson, MD PhD, Principal Investigator, Indiana University


Verification Date

June 2017