Trial Outcomes & Findings for Medical Treatment of "High-Risk" Neurofibromas (NCT NCT00846430)

NCT ID: NCT00846430

Last Updated: 2023-08-30

Results Overview

Subjects will be evaluated for pain and Quality of Life scores

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 9 participants
• Primary outcome timeframe: Monthly physical exam first three months and then every three months after, for up to 36 months
• Results posted on: 2023-08-30

Participant Flow

Period 1: Baseline to 6 months", "Period 2: After 6 month evaluation for addition of vincristine and temozolomide", if accurate and appropriate

Participant milestones

Measure	Open-Label Intervention This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Open-Label Intervention n=9 Participants This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant
Age, Categorical <=18 years	9 Participants n=9 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=9 Participants
Age, Categorical >=65 years	0 Participants n=9 Participants
Sex: Female, Male Female	4 Participants n=9 Participants
Sex: Female, Male Male	5 Participants n=9 Participants
Region of Enrollment United States	9 participants n=9 Participants

PRIMARY outcome

Timeframe: Monthly physical exam first three months and then every three months after, for up to 36 months

Subjects will be evaluated for pain and Quality of Life scores

Outcome measures

Measure

Open-Label Intervention n=9 Participants This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant

Improvement of Symptoms and Pain

9 Participants

PRIMARY outcome

Timeframe: Monthly physical exam first three months and then every three months after, for up to 36 months

Outcome measures

Measure

Open-Label Intervention n=9 Participants This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant

At Least 50% Shrinkage in Tumor Measurements by Physical Examination

7 Participants

PRIMARY outcome

Timeframe: evaluated 6, 12 and 24 months compared to baseline

partial response by RICST criteria is defined as >50% tumor shrinkage

Outcome measures

Measure	Open-Label Intervention n=9 Participants This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant
Response by MRI Measurements Partial Response at 6 months	0 participants
Response by MRI Measurements Complete Response at 6 months	1 participants
Response by MRI Measurements Less than Partial Response at 6 months	8 participants
Response by MRI Measurements Partial Response at 12 months	2 participants
Response by MRI Measurements Complete Response at 12 months	1 participants
Response by MRI Measurements Less than Partial Response at 12 months	6 participants
Response by MRI Measurements Partial Response at 24 months	4 participants
Response by MRI Measurements Complete Response at 24 months	1 participants
Response by MRI Measurements Less than Partial Response at 24 months	1 participants
Response by MRI Measurements no longer participating at 24 months	3 participants

SECONDARY outcome

Timeframe: Psychological evaluation at 24 months

Psychological toxicity defined as suicidal ideation

Outcome measures

Measure

Open-Label Intervention n=9 Participants This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant

No Reported Psychological Toxicity Based Upon Psychological Evaluations

8 Participants

Adverse Events

Open-Label Intervention

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure

Open-Label Intervention n=9 participants at risk This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant

Psychiatric disorders Suicidal Ideation

11.1% 1/9 • Number of events 1 • collected over 5 years adverse events collected during provider appointments

Other adverse events

Measure

Open-Label Intervention n=9 participants at risk This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study. Peg-Interferon alpha-2b: age and weight dependant Celecoxib (Celebrex): age and weight dependant Temozolomide (temodar): age and weight dependant Vincristine Sulfate (Oncovin): age and weight dependant

Nervous system disorders Fatigue

11.1% 1/9 • Number of events 1 • collected over 5 years adverse events collected during provider appointments

Additional Information

Dr. Albert Cornelius

Corewell Health

Phone: 616-391-2086

Email: albert.cornelius@helendevoschildrens.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place