Neurofibromatosis 1Phase 2NCT00846430

Medical Treatment of "High-Risk" Neurofibromas

A Phase 2 study testing Peg-Interferon alpha-2b, Celecoxib (Celebrex) and Temozolomide (temodar) in people with Neurofibromatosis 1, run by Corewell Health West at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 2 to 30 years
Conditions the study is for: Neurofibromatosis 1
What is being tested?: Peg-Interferon alpha-2b, Celecoxib (Celebrex) and Temozolomide (temodar)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 1 siteUnited States
Study dates: Oct 2008to May 2017
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 30 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

"High-Risk" Plexiform Neurofibromas associated with a diagnosis of NF1
2-30 years old (minimum bodyweight of 10 kilograms)
Adequate renal function

Who cannot join

Previously untreated active optic glioma
History of any previous allergy to study medications
History of ischemic vascular disease
Pregnancy / Breast feeding

Trial locations and contacts

1 site in 1 country.

United States1 site

Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503

Other trials in United States

About this study

Official title: Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies

Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction and/or become life-threatening. Presently, there is no effective medical therapy to offer patients with "high-risk" plexiform neurofibromas, and surgery does not provide lasting help. This study will evaluate the effectiveness of two treatment combinations in patients with "high-risk" plexiform neurofibromas.

Read the detailed description

The study's design involves treating eligible patients with a combination of celecoxib and pegylated interferon alpha-2b. If the patients have at least a partial response after six months, they may continue on the same treatment for up to two years. If the patient experiences less than a partial response, or has progressive disease after six months of therapy, then vincristine and temozolomide will be added to the celecoxib and interferon alpha-2b backbone. Response to treatment will be assessed after a minimum of six months, presuming the patient has not experienced progressive disease. Total duration of therapy on study is two years for any individual treatment plan.

Conditions: Neurofibromatosis 1
Being tested: Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar), Vincristine Sulfate (Oncovin)
Keywords: Neurofibromas, Neurofibromatosis 1, Celecoxib, Celebrex, Peg-Interferon alpha-2b, PEG-Intron, Temozolomide, Temodar, Vincristine Sulfate, Oncovin
Medical subject headings: Neurofibromatosis 1, Neurofibroma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Peg-Interferon alpha-2bDrug
age and weight dependant
Celecoxib (Celebrex)Drug
age and weight dependant
Temozolomide (temodar)Drug
age and weight dependant
Vincristine Sulfate (Oncovin)Drug
age and weight dependant

Study groups in NCT00846430
Study group	Role	What they receive
Open-Label Intervention	Experimental	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. Receives: Drug: Peg-Interferon alpha-2b; Drug: Celecoxib (Celebrex); Drug: Temozolomide (temodar); Drug: Vincristine Sulfate (Oncovin)

What is being measured

The primary outcome is the main question the study is designed to answer.

Improvement of Symptoms and Pain
Subjects will be evaluated for pain and Quality of Life scores
Measured: Monthly physical exam first three months and then every three months after, for up to 36 months
At Least 50% Shrinkage in Tumor Measurements by Physical Examination
Measured: Monthly physical exam first three months and then every three months after, for up to 36 months
Response by MRI Measurements
partial response by RICST criteria is defined as >50% tumor shrinkage
Measured: evaluated 6, 12 and 24 months compared to baseline

Secondary outcomes (1)

No Reported Psychological Toxicity Based Upon Psychological Evaluations
Psychological toxicity defined as suicidal ideation
Measured: Psychological evaluation at 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 2008May 2017

First posted18 Feb 2009 (estimated)
Study startOct 2008
Primary completionApr 2017
Study completionMay 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00846430
Sponsor's protocol ID: 2008-260
Other IDs: 2008-260
Status last verified: Aug 2023
First submitted: 16 Jan 2009
Last updated: 30 Aug 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms

All Neurofibromatosis 1 trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
ClinicalTrials.gov ID: NCT00846430
Acronym / sponsor ID: 2008-260
Conditions: Neurofibromatosis 1
Keywords: Neurofibromas, Neurofibromatosis 1, Celecoxib, Celebrex, Peg-Interferon alpha-2b, PEG-Intron, Temozolomide, Temodar
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: Corewell Health WestUniversity, hospital or other
Study start: Oct 2008 (actual)
Primary completion: Apr 2017 (actual)
Study completion: May 2017 (actual)
First posted: 18 Feb 2009 (estimated)
Results: Posted 30 Aug 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Open-Label Intervention	Experimental	Drug: Peg-Interferon alpha-2b, Drug: Celecoxib (Celebrex), Drug: Temozolomide (temodar), Drug: Vincristine Sulfate (Oncovin)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.

Who may qualify

Ages: 2 to 30 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (3 listed)

"High-Risk" Plexiform Neurofibromas associated with a diagnosis of NF1
2-30 years old (minimum bodyweight of 10 kilograms)
Adequate renal function

Key exclusion criteria (4 listed)

Previously untreated active optic glioma
History of any previous allergy to study medications
History of ischemic vascular disease
Pregnancy / Breast feeding

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Peg-Interferon alpha-2bage and weight dependant	Drug	Open-Label Intervention
Celecoxib (Celebrex)age and weight dependant	Drug	Open-Label Intervention
Temozolomide (temodar)age and weight dependant	Drug	Open-Label Intervention
Vincristine Sulfate (Oncovin)age and weight dependant	Drug	Open-Label Intervention

Primary outcome

Improvement of Symptoms and Pain
Time frame: 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
At Least 50% Shrinkage in Tumor Measurements by Physical Examination
Time frame: Monthly physical exam first three months and then every three months after, for up to 36 months
Response by MRI Measurements
Time frame: evaluated 6, 12 and 24 months compared to baseline
partial response by RICST criteria is defined as >50% tumor shrinkage

1 secondary outcome

No Reported Psychological Toxicity Based Upon Psychological Evaluations
Time frame: Psychological evaluation at 24 months

Results and safety

Results posted to the registry on 30 Aug 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Open-Label Intervention	9	9

Primary outcome: Improvement of Symptoms and Pain

Time frame: Monthly physical exam first three months and then every three months after, for up to 36 months. Count of participants. Unit: Participants

Measure	Open-Label Intervention n = 9
Value	9

Primary outcome: At Least 50% Shrinkage in Tumor Measurements by Physical Examination

Time frame: Monthly physical exam first three months and then every three months after, for up to 36 months. Count of participants. Unit: Participants

Measure	Open-Label Intervention n = 9
Value	7

Primary outcome: Response by MRI Measurements

Time frame: evaluated 6, 12 and 24 months compared to baseline. Number. Unit: participants

Measure	Open-Label Intervention n = 9
Partial Response at 6 months	0
Complete Response at 6 months	1
Less than Partial Response at 6 months	8
Partial Response at 12 months	2
Complete Response at 12 months	1
Less than Partial Response at 12 months	6
Partial Response at 24 months	4
Complete Response at 24 months	1

1 secondary outcome is reported in the full results.

Adverse events

Time frame: collected over 5 years. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Open-Label Intervention	1 of 9 (11.1%)	1 of 9 (11.1%)	0 of 9

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Neurofibromatosis 1

Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Albert S Cornelius, MD principal investigator

Helen DeVos Children's Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Peg-Interferon alpha-2b, Celecoxib (Celebrex) and Temozolomide (temodar) in people with Neurofibromatosis 1, run by Corewell Health West at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Improvement of Symptoms and Pain (time frame: Monthly physical exam first three months and then every three months after, for up to 36 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00846430.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Aug 2023.