Juvenile Pilocytic AstrocytomasPhase 2NCT02343224
Pegylated Interferon ALFA-2b in Children With Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas
A Phase 2 study testing Pegylated interferon alpha-2b in people with Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas, run by Emory University at 1 site in United States.
Trial summary
This is a phase II study of the drug, pegylated interferon alfa-2b (PEG-Intron), used to treat brain tumors in a pediatric population. Researchers want to see if treatment with PEG-Intron will stop tumor growth for patients with juvenile pilocytic astrocytomas or optic pathway gliomas. Read more about this study
- Who is it for?
- Ages 3 to 25 years
- Conditions the study is for
- Juvenile Pilocytic Astrocytomas, Optic Pathway Gliomas
- What is being tested?
- Pegylated interferon alpha-2b
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2014to 11 Nov 2020
- 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 3 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be older than 3 years and less than or equal to 25 years of age at the time of enrollment
- Patients with neurofibromatosis are eligible
- Histologic confirmation is not required for this if the patient has neurofibromatosis type 1 (NF-1) with MRI findings consistent with optic pathway glioma or JPA. Any other tumors will need histological confirmation, either at the time of diagnosis or at the time of recurrence. The histological diagnosis includes World Health Organization (WHO) grade I JPA
- Patients must have measurable residual disease, defined as tumor that is measurable in two or three perpendicular diameters on MRI. For a lesion to be considered measurable, it must be at least twice the slice thickness on MRI (i.e visible on more than one slice)
- All patients must have a brain MRI with and without contrast (gadolinium) within 30 days prior to study enrollment. All patients with history of spinal or leptomeningeal disease and those patients with symptoms suspicious of spinal disease, must have a spine MRI with contrast (gadolinium) performed within 30 days prior to study enrollment. Lumbar puncture is necessary if there is evidence of tumor dissemination on the MRI of spine
- Performance Level: Karnofsky > or equal to 50% for patients > 10 years of age or Lansky > or equal to 50 for patients < 10 years of age
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- Patients must have recovered (to Common Toxicity Criteria (CTC) v.5.0 ≤ Grade 1 unless indicated below) from the acute toxic effects of all prior chemotherapy, immunotherapy prior to entering this study, with the exception of alopecia, weight changes and Grade I or II lymphopenia
- Must not have received myelosuppressive chemotherapy within 3 weeks of enrollment onto this study (6 weeks if prior nitrosourea)
- At least 7 days must have elapsed since the completion of therapy with other biologic agents. For other biologic agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
- At least 3 half-lives of the antibody after the last dose of a monoclonal antibody. Specifically for bevacizumab 36 days after the last dose
- At least 3 weeks from the last surgical resection, prior to start study drug
- At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines
- Patients must have had their last fraction of cranial or craniospinal Radiation ≥ 24 months prior to study entry
- Patients who have received polyinosinic-polycytidylic acid-polylysine-carboxymethylcellulose (Poly-ICLC) are eligible for this trial if all acute Poly-ICLC -related toxicity has resolved
- Patients must not have received Pegylated interferon previously
- Must not have received growth factor within 2 weeks of entry into this study
- Patients who are receiving corticosteroids must be on a stable or decreasing dose for at least 1 week prior enrollment in the study
- Adequate organ, hematological, renal, and pulmonary function
- If history of depression or psychiatric illness, has to be well controlled with antidepressants and/or under psychiatrist/psychologist care
Who cannot join
- Patients who are receiving concurrent chemotherapy, or who are currently receiving other investigational chemotherapeutic agents or concurrently receiving radiation
- Patients with a known hypersensitivity to interferon-alpha
- Prior use of Pegylated interferon or interferon
- Less than 2 years since completion of radiation therapy
- Pregnant or breast-feeding females are excluded
- Patients with clinically significant unrelated systemic illness
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- Dental braces or prosthesis that interferes with magnetic resonance (MR) imaging
- History of noncompliance to medical regimens
- Patients unwilling to or unable to comply with the protocol
- Patients with a positive history of Hepatitis B or Hepatitis C
- Male patient whose sexual partner(s) are women of childbearing potential who are not willing to use adequate contraception, during the study and for 8 weeks after the end of treatment
- Patients should not receive immunization with attenuated live vaccines within one week of study entry or during study period
- Patient with diagnosis of Diffuse Intrinsic Pontine Glioma
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: A Phase II Study Of Pegylated Interferon ALFA-2b in Children With Recurrent or Refractory and Radiographically or Clinically Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas
This is a phase II study of the drug, pegylated interferon alfa-2b (PEG-Intron), used to treat brain tumors in a pediatric population. Researchers want to see if treatment with PEG-Intron will stop tumor growth for patients with juvenile pilocytic astrocytomas or optic pathway gliomas.
The purposes of this study are:
- To learn more about the response to pegylated interferon
- To learn more about the side effects of pegylated interferon
- To learn more about MRI images in patients with Juvenile Pilocytic Astrocytomas or Optic Pathway Gliomas.
- To learn more about quality of life in patients treated with pegylated interferon
Read the detailed description
Low grade gliomas are the most common pediatric central nervous system malignancies and can occur in different parts of the brain. Patients who undergo gross total resection, usually those with hemispheric tumors, have an excellent prognosis with surgical resection alone. Patients for whom gross total resection is not achievable have a significant risk of disease progression. Therefore, these patients benefit from adjuvant therapy. Multiple chemotherapy regimens have shown some efficacy in residual tumor, but more than 50% of patients experience recurrences. Radiation has been shown to be an effective therapy in the treatment of these tumors. Because of concerns regarding radiation toxicity especially in young children, and progression despite chemotherapy, novel approaches are needed. This protocol represents an attempt to measure the efficacy and safety of use of pegylated interferon for patients with recurrent, refractory Juvenile Pilocytic Astrocytomas (JPA) or optic pathway gliomas. It provides a different approach to the commonly used treatment modalities. The objectives of this study are to determine the response of children with chemotherapy-refractory progressive JPA or optic pathway gliomas (OPG) to weekly pegylated interferon alpha-2b. The secondary objectives include to better identify the toxicities of weekly pegylated interferon alpha-2b (PEG-Intron™) in pediatric patients with unresectable, refractory, recurrent JPAs or optic pathway gliomas, to evaluate various magnetic resonance imaging techniques for noninvasive monitoring of metabolic and biologic changes in the tumors and to evaluate the quality of life for patients with recurrent, refractory JPAs who receive therapy with pegylated interferon alpha-2b (PEG-Intron™).
The primary end point is to determine the response rate. A two-stage design has been selected to evaluate the response rate. If the treatment demonstrates at least a 25% response rate, the researchers would consider it a promising regimen for further study. A response rate less than 5% is considered evidence of unpromising regimen. Seventeen evaluable pediatric patients with JPA or OPG will be accrued. If at least 3 responders are seen among the 17 patients, this will be considered evidence of a promising response rate for further evaluation.
- Being tested
- Pegylated interferon alpha-2b
- Medical subject headings
- Astrocytoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pegylated interferon alpha-2bDrug
Also called Peg-Intron
PEG-Intron 1mcg/kg/dose weekly through an injection under the skin on the same day each week
| Study group | Role | What they receive |
|---|---|---|
| Pegylated interferon alpha-2b | Experimental | Subjects will receive PEG-Intron based on their weight (1 mcg/kg/dose) once a week Receives: Drug: Pegylated interferon alpha-2b |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Children Responding to Treatment
The objective response of study participants was categorized as complete response, partial response, or stable disease. Determination of tumor response or progression is based on the pre-study baseline scan performed closest to time of study entry. The overall response assessment takes into account response in both target and non-target lesion, and the appearance of new lesions. Complete response is defined as the disappearance of all target lesions and non-target lesions, and no new lesions. Partial response is defined as ≥ 65% decrease in the sum of the products of the three perpendicular diameters of all target lesions, and no development of new lesions. Stable disease is defined as neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for partial response, nor sufficient increase in a single target lesion to qualify for progressive disease, as well as no new lesions.
Measured: Month 12
Secondary outcomes (2)
Number of Participants Meeting Event Free Survival Criteria
Event free survival is the time to treatment failure from the time of study enrollment to tumor progression, tumor recurrence, death from any cause or occurrence of a second malignant neoplasm.
Measured: Month 12, Month 24
Number of Participants Meeting Overall Survival Criteria
Overall survival is measured as the time from study enrollment to death from any cause.
Measured: Month 12, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2015 (estimated)
- Study startNov 2014
- Primary completion11 Nov 2020
- Study completion11 Nov 2020
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 Juvenile Pilocytic AstrocytomasStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- 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)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- CURE Childhood Cancer, Inc.
- Responsible party
- Principal investigator, Dolly Aguilera, Emory University
- Investigators
- Dolly Aguilera, MD, Principal investigator, Children's Healthcare of Atlanta/Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02343224
- Sponsor's protocol ID
- IRB00074563
- Status last verified
- Jan 2022
- First submitted
- 8 Jan 2015
- Last updated
- 24 Jan 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Of Pegylated Interferon ALFA-2b in Children With Recurrent or Refractory and Radiographically or Clinically Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas
- ClinicalTrials.gov ID
- NCT02343224
- Acronym / sponsor ID
- IRB00074563
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- CURE Childhood Cancer, Inc.
- Study start
- Nov 2014
- Primary completion
- 11 Nov 2020 (actual)
- Study completion
- 11 Nov 2020 (actual)
- First posted
- 21 Jan 2015 (estimated)
- Results
- Posted 24 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase II study of the drug, pegylated interferon alfa-2b (PEG-Intron), used to treat brain tumors in a pediatric population. Researchers want to see if treatment with PEG-Intron will stop tumor growth for patients with juvenile pilocytic astrocytomas or optic pathway gliomas.
The purposes of this study are:
- To learn more about the response to pegylated interferon
- To learn more about the side effects of pegylated interferon
- To learn more about MRI images in patients with Juvenile Pilocytic Astrocytomas or Optic Pathway Gliomas.
- To learn more about quality of life in patients treated with pegylated interferon
Detailed description
Low grade gliomas are the most common pediatric central nervous system malignancies and can occur in different parts of the brain. Patients who undergo gross total resection, usually those with hemispheric tumors, have an excellent prognosis with surgical resection alone. Patients for whom gross total resection is not achievable have a significant risk of disease progression. Therefore, these patients benefit from adjuvant therapy. Multiple chemotherapy regimens have shown some efficacy in residual tumor, but more than 50% of patients experience recurrences. Radiation has been shown to be an effective therapy in the treatment of these tumors. Because of concerns regarding radiation toxicity especially in young children, and progression despite chemotherapy, novel approaches are needed. This protocol represents an attempt to measure the efficacy and safety of use of pegylated interferon for patients with recurrent, refractory Juvenile Pilocytic Astrocytomas (JPA) or optic pathway gliomas. It provides a different approach to the commonly used treatment modalities. The objectives of this study are to determine the response of children with chemotherapy-refractory progressive JPA or optic pathway gliomas (OPG) to weekly pegylated interferon alpha-2b. The secondary objectives include to better identify the toxicities of weekly pegylated interferon alpha-2b (PEG-Intron™) in pediatric patients with unresectable, refractory, recurrent JPAs or optic pathway gliomas, to evaluate various magnetic resonance imaging techniques for noninvasive monitoring of metabolic and biologic changes in the tumors and to evaluate the quality of life for patients with recurrent, refractory JPAs who receive therapy with pegylated interferon alpha-2b (PEG-Intron™).
The primary end point is to determine the response rate. A two-stage design has been selected to evaluate the response rate. If the treatment demonstrates at least a 25% response rate, the researchers would consider it a promising regimen for further study. A response rate less than 5% is considered evidence of unpromising regimen. Seventeen evaluable pediatric patients with JPA or OPG will be accrued. If at least 3 responders are seen among the 17 patients, this will be considered evidence of a promising response rate for further evaluation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pegylated interferon alpha-2b | Experimental | Drug: Pegylated interferon alpha-2bSubjects will receive PEG-Intron based on their weight (1 mcg/kg/dose) once a week |
Who may qualify
- Ages
- 3 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Patients must be older than 3 years and less than or equal to 25 years of age at the time of enrollment
- Patients with neurofibromatosis are eligible
- Histologic confirmation is not required for this if the patient has neurofibromatosis type 1 (NF-1) with MRI findings consistent with optic pathway glioma or JPA. Any other tumors will need histological confirmation, either at the time of diagnosis or at the time of recurrence. The histological diagnosis includes World Health Organization (WHO) grade I JPA
- Patients must have measurable residual disease, defined as tumor that is measurable in two or three perpendicular diameters on MRI. For a lesion to be considered measurable, it must be at least twice the slice thickness on MRI (i.e visible on more than one slice)
- All patients must have a brain MRI with and without contrast (gadolinium) within 30 days prior to study enrollment. All patients with history of spinal or leptomeningeal disease and those patients with symptoms suspicious of spinal disease, must have a spine MRI with contrast (gadolinium) performed within 30 days prior to study enrollment. Lumbar puncture is necessary if there is evidence of tumor dissemination on the MRI of spine
Key exclusion criteria (13 listed)
- Patients who are receiving concurrent chemotherapy, or who are currently receiving other investigational chemotherapeutic agents or concurrently receiving radiation
- Patients with a known hypersensitivity to interferon-alpha
- Prior use of Pegylated interferon or interferon
- Less than 2 years since completion of radiation therapy
- Pregnant or breast-feeding females are excluded
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 |
|---|---|---|
| Pegylated interferon alpha-2bAlso: Peg-IntronPEG-Intron 1mcg/kg/dose weekly through an injection under the skin on the same day each week | Drug | Pegylated interferon alpha-2b |
Primary outcome
Number of Children Responding to Treatment
Time frame: Month 12
The objective response of study participants was categorized as complete response, partial response, or stable disease. Determination of tumor response or progression is based on the pre-study baseline scan performed closest to time of study entry. The overall response assessment takes into account response in both target and non-target lesion, and the appearance of new lesions. Complete response is defined as the disappearance of all target lesions and non-target lesions, and no new lesions. Partial response is defined as ≥ 65% decrease in the sum of the products of the three perpendicular diameters of all target lesions, and no development of new lesions. Stable disease is defined as neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for partial response, nor sufficient increase in a single target lesion to qualify for progressive disease, as well as no new lesions.
2 secondary outcomes
Number of Participants Meeting Event Free Survival Criteria
Time frame: Month 12, Month 24
Number of Participants Meeting Overall Survival Criteria
Time frame: Month 12, Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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.
Dolly Aguilera, MD principal investigator
Children's Healthcare of Atlanta/Emory University
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 Pegylated interferon alpha-2b in people with Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas, run by Emory University 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 19 inclusion and 13 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 24 Jan 2022. 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 24 Jan 2022). 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?
Number of Children Responding to Treatment (time frame: Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Jan 2022.