Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

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Brief Title

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

Official Title

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T

Brief Summary

      This trial is evaluating the use of oral Everolimus to determine if there is a reduction in
      the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6
      month period. The evaluation will be done by 3D photography measuring volume with the LIFEVIZ
      Micro system.
    

Detailed Description

      Qualifying subjects will have a diagnosis of Neurofibromatosis 1 and have disfiguring
      cutaneous lesions that can be measured by photography. The subjects will have photographs of
      the target lesions,biopsies of the lesions,and safety blood laboratory tests at 3 time points
      (baseline, 3 months and 6 months which is end of treatment). Everolimus will be taken orally
      for 6 months. Subjects will visit the clinic monthly for an exam and adverse event
      evaluation. Laboratory testing will be done at these visits if determined necessary by the
      PI.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

3D Photographic Measurement of Surface Volume

Secondary Outcome

 Number of Participants With Grade 3-4 Adverse Events

Condition

Neurofibromatosis 1

Intervention

Everolimus

Study Arms / Comparison Groups

 Intervention
Description:  This is a single arm intervention using Everolimus

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

24

Start Date

February 2015

Completion Date

March 2016

Primary Completion Date

March 2016

Eligibility Criteria

        Inclusion Criteria:

          1. Patient is at least 18 years of age at the time of enrollment

          2. Informed consent

          3. Compliance with trial requirements (photography, lab draws, medication schedules, and
             study visits)

          4. Diagnosis of NF 1 and has cutaneous neurofibromas that are located in in a region
             amenable to photography

          5. Females of child bearing potential must not be pregnant as confirmed by a negative
             pregnancy test (blood beta-hCG level) prior to study enrollment and must agree to use
             appropriate contraceptive methods for the duration of the trial

          6. Patient must have adequate liver function as shown by, total bilirubin /- 3 years, patients with a history of non compliance to medical regimens or who
        are considered potentially unreliable or will not be able to complete the entire study,
        patients who are currently part of or have participated in any clinical investigation with
        an investigational drug within 1 month prior to dosing, pregnant or nursing (lactating)
        women, women of child-bearing potential (WOCBP), defined as all women physiologically
        capable of becoming pregnant, unless they are using highly effective methods of
        contraception during dosing of study treatment. highly effective contraception methods,male
        patients whose sexual partner(s) are WOCBP who are not willing to use adequate
        contraception, during the study and for 8 weeks after the end of treatment.

        -
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Mary Kay Koenig, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02332902

Organization ID

HSC-MS-14-0758


Responsible Party

Principal Investigator

Study Sponsor

The University of Texas Health Science Center, Houston

Collaborators

 Texas Neurofibromatosis Foundation

Study Sponsor

Mary Kay Koenig, MD, Principal Investigator, The University of Texas Health Science Center, Houston


Verification Date

May 2017