AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine

NCT00326872 | Terminated Phase 2 Results

• 7 other identifiers: NCI-2009-00128MC047FCDR00004757617133N01CM17104N01CM62205P30CA015083
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 9

Brief Summary

This phase II trial is studying how well AZD2171 works in treating patients with neurofibromatosis type 1 and plexiform neurofibroma and/or neurofibroma near the spine. AZD2171 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

Conditions

Neurofibromatosis Type 1; Plexiform Neurofibroma; Spinal Cord Neurofibroma

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients With Tumor Response (Complete Response [CR] or Partial Response [PR])

  Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the volume of target lesions taking as reference the baseline volume.

  Baseline to end of treatment, maximum of 26 cycles (28 days/cycle).

Secondary Outcomes (5)

• Survival Time as Measured Using Kaplan-Meier Method

  From registration to death (due to any cause) max 51 months

• Time to Disease Progression as Measured Using Kaplan-Meier Method

  From registration to documentation of disease progression up to 26 cycles (28 days/cycle).

• Duration of Response as Assessed Using the Method of Kaplan-Meier

  From time of confirmed tumor objective response as CR or PR to the date of progression max 51 months

• Time to Treatment Failure as Assessed Using the Method of Kaplan-Meier

  From the date of randomization to the date at which the patient is removed from treatment due to progression, toxicity, or refusal up to 51 months.

• Reduction in Self Reported Worst Pain Per Cycle.

  At baseline, prior to each subsequent course (q 28+/- 3 days), and at end of treatment up to 51 months

Study Arms (1)

Treatment (cediranib maleate)

EXPERIMENTAL

Patients receive oral AZD2171 once daily on days 1-28. Treatment repeats every 28 days for 26 courses in the absence of disease progression or unacceptable toxicity. Patients with responding or stable disease may continue treatment beyond 26 courses in the absence of disease progression or unacceptable toxicity. Quality of life is assessed at baseline, prior to course 2, prior to course 4, and every 6 courses thereafter.

Drug: Cediranib Maleate

Interventions

Cediranib Maleate DRUG

Also known as: AZD2171, AZD2171 Maleate, Recentin

Treatment (cediranib maleate)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis\* of neurofibromatosis type 1 (NF1) and extensive plexiform and/or paraspinal neurofibromasproducing pain (not controlled by use of over-the-counter medications), progressive neurologic deficit, or significant neurologic consequenceswith continuous tumor growth
• Extensive paraspinal neurofibroma defined as a neurofibroma that involves multiple neural roots at ≥ 3 spinal levels with connection between the levels or extending laterally along the nerves
• Symptomatic neurofibromas at \< 3 spinal levels, but surgical treatment is not possible, allowed
• Meets ≥ 2 diagnostic criteria for NF1, including the following:
• Six or more café-au-lait spots (≥ 1.5 cm in postpubertal patients)
• Freckling in the axilla or groin
• Optic glioma
• Two or more Lisch nodules
• Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia orthinning of long-bone cortex)
• First-degree relative with NF1
• Patients with documented mutation in neurofibromin gene with onlysymptomatic plexiform and/or paraspinal neurofibroma who do not fulfill the above clinical criteria are eligible
• Measurable disease, defined as ≥ 1 lesion whose longest diameter can beaccurately measured as 8.0 cm\^3 with 3-dimensional (3D) MRI
• Skin lesions are consideredmeasurable (e.g., plexiform neurofibromas), but MRI imaging still required for 3D measurement
• Patients with symptomatic neurofibroma, in whom surgery is not feasible, who refuse surgery or are not goodsurgical candidates due to high risk of damage to vital structures or spinal cordinjury are eligible
• No evidence of progressive optic glioma, malignant glioma, malignant peripheralnerve sheath tumor, or other cancer requiring treatment with chemotherapy orradiotherapy

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (9)

University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States

Location

Howard University Hospital

Washington D.C., District of Columbia, 20060, United States

Location

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States

Location

Massachusetts General Hospital Cancer Center

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Interventions

cediranib

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Results Point of Contact

• Title: Dr. Dusica Babovic-Vuksanovic
• Organization: Mayo Clinic

dbabovic@mayo.edu

Study Officials

• Dusica Babovic-Vuksanovic

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2006
• First Posted: May 17, 2006
• Study Start: May 1, 2006
• Primary Completion: August 21, 2011
• Study Completion: May 31, 2016
• Last Updated: August 18, 2017
• Results First Posted: September 26, 2013

Record last verified: 2017-07

Locations

US (9)