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Phase 2 N=24 Treatment

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

Neurofibromatosis 1

Enrolled (actual)
24
Serious AEs
0.0%
Results posted
Jun 2017
Primary outcome: Primary: 3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion — 32.54; 33.93 mm^3

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Everolimus (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
The University of Texas Health Science Center, Houston
Primary completion
Mar 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion
32.54; 33.93
SECONDARY
Number of Participants With Grade 3-4 Adverse Events

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.

Eligibility Criteria

Inclusion Criteria

  • Patient is at least 18 years of age at the time of enrollment
  • Informed consent
  • Compliance with trial requirements (photography, lab draws, medication schedules, and study visits)
  • Diagnosis of NF 1 and has cutaneous neurofibromas that are located in in a region amenable to photography
  • 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
  • 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.

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View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02332902). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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