Study of Imatinib in Children With Neurofibromatosis and Airway Tumors

NCT ID: NCT03688568

Last Updated: 2021-02-12

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-09-01
• Study Completion Date: 2021-09-01

Brief Summary

The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD

Conditions

Neurofibroma, Plexiform

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Imatinib Mesylate Arm

Imatinib Mesylate, given daily orally, 55 mg PO BID, if tolerated for 2 weeks increase to 110 mg/m2 BID, and further increase to 165 and final dosage to 220 mg/m2 bid if tolerated. Can continue for 12 months.

Group Type: EXPERIMENTAL

Imatinib Mesylate

Intervention Type: DRUG

Imatinib given orally as dose escalation treatment.

Interventions

Imatinib Mesylate

Imatinib given orally as dose escalation treatment.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Patients aged: > 6 months and < 12 years of age.

2. Diagnosis of neurofibromatosis type 1 (NF1).

3. Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing.

4. Patients must have measurable (> 1.5 cm in two dimensions or able to assess a minimum of 3 slices) 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 or crushed pills in a soft food such as pudding or apple sauce; or have other GI access such as a G-tube.

8. Written, voluntary informed consent/assent.

Exclusion Criteria

1. Patient has received any other investigational agents within 14 days of first day of study drug dosing.

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

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

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

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

8. Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C) prior to study entry.

9. Patient previously received radiotherapy to > 25 % of the bone marrow

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

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

12. 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.

13. Patient has an unstable airway requiring more urgent intervention or deemed unable to travel due to unstable airway by referring MD.

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Indiana University

OTHER

Sponsor Role: lead

Responsible Party

Kent A Robertson

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Riley Hospital for Children - Indiana University

Indianapolis, Indiana, United States

Countries

United States

Other Identifiers

1R01FD004830-01A2

Identifier Type: FDA

Identifier Source: secondary_id

View Link

1505569560

Identifier Type: -

Identifier Source: org_study_id