GliomaPhase 1ASCRNCT01661400

Anti-Angiogenic Therapy Post Transplant (ASCR) for Pediatric Solid Tumors

A Phase 1 study testing Metronomic Cyclophosphamide and Thalidomide in people with Glioma and Neuroectodermal Tumors, Primitive, run by Washington University School of Medicine 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

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Trial summary

Who is it for?: Ages 6 months to 21 years
Conditions the study is for: Glioma, Neuroectodermal Tumors, Primitive, Wilms Tumor
What is being tested?: Metronomic Cyclophosphamide and Thalidomide
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 14 participants
Where: 1 siteUnited States
Study dates: 26 Oct 2012to 27 Dec 2023
Phase: Phase 1
Are there results?: None posted in the registry

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 6 months to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Patient must have an original diagnosis, benefited by autologous transplantation, confirmed by biopsy* of high-grade glial tumor, low-grade glial tumor, ependymoma, medulloblastoma, primitive neuro-ectodermal tumor (PNET), Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, retinoblastoma, or miscellaneous poor-prognosis solid tumors. Lymphomas and other lymphoid malignancies will not be studied in this protocol.
Brain stem glioma patients who have progressed after radiation therapy do not require histologic confirmation. Brain stem gliomas are defined as intrinsic tumors of the pons causing diffuse enlargement. These patients are diagnosed on clinical and radiographic appearance of the lesion and the biopsy requirement will be waived for this group.
Patient must be ≥ 6 months of age and ≤ 21 years of age at the time of study entry.
Patient must have a Karnofsky performance status or Lansky* play status ≥ 50
For purpose of determining performance scores, wheelchair-bound patients will be considered ambulatory.
Patient must have an adequate supply of stem cells for transplant harvested prior to study enrollment, with adequate supply defined as 3 x 10^6 CD34+ cells/kg for peripheral blood stem cells (PBSC). Cell mobilization method will be left up to the treating physician's discretion and may include mobilization growth factor alone or mobilization after chemotherapy. If patient is unable to mobilize the proper amount of peripheral stem cells, bone marrow may be harvested as the source of hematopoietic stem cells. In this instance, 3 x 10^8 mononuclear cells/kg will be considered adequate. If necessary, a combination of peripheral stem cells and bone marrow can be used.

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Prior radiation therapy and/or chemotherapy, including cyclophosphamide, are permitted.
Prior anti-angiogenic therapy, including thalidomide and oral cyclophosphamide, is permitted.
If on corticosteroids for mass effect and/or edema related to the tumor, patient must be on a stable or decreasing dose for at least 2 weeks prior to study entry.
Patient must have a life expectancy > 3 months.
Patient must have an adequate bone marrow reserve as defined by:
Hemoglobin ≥ 8.0 g/dl and
Peripheral absolute neutrophil count (ANC) ≥ 750/mm3
Patient must have adequate cardiac function tested within 4 weeks of study enrollment as defined by:
Shortening fraction of ≥ 27% by echocardiogram or
Ejection fraction of ≥ 50% by radionuclide angiogram
Patient must have adequate pulmonary function tested within 4 weeks of study enrollment as defined by:
Pulse oximetry > 94% on room air or O2 by nasal cannula and
No evidence of dyspnea at rest.
Patient must have adequate hepatic function as defined by:
Total bilirubin ≤ 1.5x upper limit of normal (ULN) for age and
SGOT (AST) or SGPT (ALT) ≤ 2.5 x ULN (SGOT ≤ 4x ULN if on Zantac)
Patient must have adequate renal function as defined by:
Serum creatinine < 1.5 mg/dl
Glomerular filtration rate (GFR), calculated via I-125 iothalamate clearance, 24-hour creatinine clearance, or Schwartz formula*, ≥ 70 mL/min and ≥ 50 mL/min/1.73 m2 done within 4 weeks of study entry
The Schwartz formula is an estimated glomerular filtration rate in children based upon serum creatinine and height. Height (Ht) should be measured in cm and serum creatinine (Cr) in mg/dL. Proportionality constant (k) is 0.55 for children and adolescent girls and 0.7 for adolescent boys aged 13-21. This constant is based upon a series of evaluations performed by Schwartz. Formula: GFR= (k x Ht)/Cr
Enrollment in the Celgene THALOMID REMSTM Program:
If enrolled in Arm III of this study, patient must be registered at the Celgene THALOMID REMSTM Program prior to day +30 post-ASCR.
If enrolled in Arm III of this study, patient must be willing to practice birth control as outlined in the THALOMID REMSTM Program from the beginning of the thalidomide treatment until at least 4 weeks following discontinuation of thalidomide therapy. Two reliable forms of contraception must be used simultaneously unless continuous abstinence from heterosexual sexual contact is chosen. Contraceptive methods must include at least one highly effective method (e.g. oral contraceptive pills, injections, hormonal patches, IUD, or implants), AND one additional effective barrier method (e.g. latex condom, diaphragm, cervical cap).
If hormonal or IUD contraception is medically contraindicated, another highly effective method or two barrier methods must be used at the same time.
Pregnancy surveillance:
Patient must have a negative in office pregnancy test sensitive to within 50 mIU/mL (serum or urine) within 24 hours prior to beginning thalidomide even if continuous abstinence is the preferred method of birth control. ii. A pregnancy test must be performed weekly during the first 4 weeks of therapy and repeated monthly for patients with regular menses or every 2 weeks for patients with irregular menses iii. Negative pregnancy tests are valid for only 7 days. iv. If irregular bleeding or skipped menses, pregnancy test should be performed and pregnancy counseling given. v. If pregnancy occurs during treatment, thalidomide must be immediately discontinued. Any suspected lethal exposure must be reported immediately to Celgene Customer Care Center at 1-888-423-5346, and the patient referred to an OB/GYN experienced in reproductive toxicity for further evaluation and counseling.
Patient (or legally authorized representative) must be able to understand and willing to sign a written informed consent document.

Who cannot join

Patient must not have any active, uncontrolled cardiac, hepatic, renal, or psychiatric disease defined as ≥ grade 3 based on NCI Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
Patient must not be receiving any other investigational agents.
Patient must not have any active infection or concurrent illness obscuring toxicity or dangerously altering drug metabolism.
Patient must not have any thromboembolic event (deep vein thrombosis or pulmonary embolism) less than 3 weeks prior to enrollment.
Patient must not have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to any of the agents used in the study.
Patient must not be pregnant or breastfeeding.

Trial locations and contacts

1 site in 1 country.

United States1 site

Washington University School of Medicine
St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: Anti-Angiogenic Therapy After Autologous Stem Cell Rescue (ASCR) for Pediatric Solid Tumors

The purpose of this research study is to determine whether taking either of two low dose drugs that would prevent new blood vessels from growing after stem cell transplant is feasible, and what the side effects of taking each of these drugs after autologous transplant might be. The reason the investigators are looking at these drugs is because one of the things that allows tumors to grow quickly is their ability to stimulate the growth of new blood vessels. By suppressing the growth of new blood vessels after stem cell transplant, the investigators hope to prevent the tumors from coming back or continuing to grow.

Conditions: Glioma, Neuroectodermal Tumors, Primitive, Wilms Tumor, Rhabdomyosarcoma, Sarcoma, Ewing, Osteosarcoma
Being tested: Metronomic Cyclophosphamide, Thalidomide
Keywords: Glioma includes ependymoma and medulloblastoma
Medical subject headings: Glioma, Neuroectodermal Tumors, Primitive, Wilms Tumor, Rhabdomyosarcoma, Sarcoma, Ewing, Osteosarcoma, Retinoblastoma, Medulloblastoma

What participants receive

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

Metronomic CyclophosphamideDrug
Also called Cytoxan®, CPM
ThalidomideDrug
Also called Thalomid®

Study groups in NCT01661400
Study group	Role	What they receive
Control	No intervention	No intervention
Metronomic Cyclophosphamide	Experimental	Cyclophosphamide will be given PO once daily at 2.5 mg/kg/day for children < 40kg or 100 mg daily for children > 40kg beginning Day + 30 (30 days post transplant) and continue until at least Day +86 Receives: Drug: Metronomic Cyclophosphamide
Thalidomide	Experimental	Thalidomide will be initiated at 3mg/kg PO daily beginning Day + 30 (30 days post transplant) and continue until Day +86 Receives: Drug: Thalidomide

What is being measured

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

Safety as measured by absence of grade 4 or 5 non-hematological or grade 5 hematological toxicity
Measured: Through 1 year post-transplant
Incidence of major transplant related toxicities (Grades IV and IV)
Measured: Within the first year of transplant

Secondary outcomes (1)

Best overall response
Assessed by Response Evaluation Criteria in Solid Tumors (RECIST) guideline version 1.1.
Measured: 86 days

Timeline

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

26 Oct 201227 Dec 2023

First posted9 Aug 2012 (estimated)
Study start26 Oct 2012
Primary completion27 Dec 2023
Study completion27 Dec 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for GliomaStudies of the same condition, open and under way.
More trials run by Washington University School of MedicineThe 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 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 14 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01661400
Sponsor's protocol ID: 201209088
Acronym: ASCR
Status last verified: Jan 2024
First submitted: 30 Jul 2012
Last updated: 31 Jan 2024
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: 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

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RecruitingPhase 3
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All Glioma trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Anti-Angiogenic Therapy After Autologous Stem Cell Rescue (ASCR) for Pediatric Solid Tumors
ClinicalTrials.gov ID: NCT01661400
Acronym / sponsor ID: ASCR / 201209088
Conditions: Glioma, Neuroectodermal Tumors, Primitive, Wilms Tumor, Rhabdomyosarcoma, Sarcoma, Ewing, Osteosarcoma
Keywords: Glioma includes ependymoma and medulloblastoma
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 14 participants (actual)
Lead sponsor: Washington University School of MedicineUniversity, hospital or other
Study start: 26 Oct 2012 (actual)
Primary completion: 27 Dec 2023 (actual)
Study completion: 27 Dec 2023 (actual)
First posted: 9 Aug 2012 (estimated)
Results: Not posted in the source record

Clinical summary

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

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Control	No intervention	–No intervention
Metronomic Cyclophosphamide	Experimental	Drug: Metronomic CyclophosphamideCyclophosphamide will be given PO once daily at 2.5 mg/kg/day for children < 40kg or 100 mg daily for children > 40kg beginning Day + 30 (30 days post transplant) and continue until at least Day +86
Thalidomide	Experimental	Drug: ThalidomideThalidomide will be initiated at 3mg/kg PO daily beginning Day + 30 (30 days post transplant) and continue until Day +86

Who may qualify

Ages: 6 months to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (33 listed)

Patient must have an original diagnosis, benefited by autologous transplantation, confirmed by biopsy* of high-grade glial tumor, low-grade glial tumor, ependymoma, medulloblastoma, primitive neuro-ectodermal tumor (PNET), Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, retinoblastoma, or miscellaneous poor-prognosis solid tumors. Lymphomas and other lymphoid malignancies will not be studied in this protocol.
Patient must be ≥ 6 months of age and ≤ 21 years of age at the time of study entry.
Patient must have a Karnofsky performance status or Lansky* play status ≥ 50
Patient must have an adequate supply of stem cells for transplant harvested prior to study enrollment, with adequate supply defined as 3 x 10^6 CD34+ cells/kg for peripheral blood stem cells (PBSC). Cell mobilization method will be left up to the treating physician's discretion and may include mobilization growth factor alone or mobilization after chemotherapy. If patient is unable to mobilize the proper amount of peripheral stem cells, bone marrow may be harvested as the source of hematopoietic stem cells. In this instance, 3 x 10^8 mononuclear cells/kg will be considered adequate. If necessary, a combination of peripheral stem cells and bone marrow can be used.
Prior radiation therapy and/or chemotherapy, including cyclophosphamide, are permitted.

Key exclusion criteria (6 listed)

Patient must not have any active, uncontrolled cardiac, hepatic, renal, or psychiatric disease defined as ≥ grade 3 based on NCI Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
Patient must not be receiving any other investigational agents.
Patient must not have any active infection or concurrent illness obscuring toxicity or dangerously altering drug metabolism.
Patient must not have any thromboembolic event (deep vein thrombosis or pulmonary embolism) less than 3 weeks prior to enrollment.
Patient must not have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to any of the agents used in the study.

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
Metronomic CyclophosphamideAlso: Cytoxan®, CPM	Drug	Metronomic Cyclophosphamide
ThalidomideAlso: Thalomid®	Drug	Thalidomide

Primary outcome

Safety as measured by absence of grade 4 or 5 non-hematological or grade 5 hematological toxicity
Time frame: Through 1 year post-transplant
Incidence of major transplant related toxicities (Grades IV and IV)
Time frame: Within the first year of transplant

1 secondary outcome

Best overall response
Time frame: 86 days

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2024.
Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Glioma

Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Active, not recruitingPhase 3
EF-41/KEYNOTE D58: Phase 3 Study of Optune Concomitant With Temozolomide Plus Pembrolizumab in Newly Diagnosed Glioblastoma
Glioblastoma
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 3
Beginning Radiation Immediately With GammaTile at GBM Excision Versus Standard of Care
Glioblastoma
RecruitingPhase 1/2
Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
Recurrent Glioblastoma, IDH-Wildtype, Recurrent MGMT-Methylated Glioblastoma

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.

Andrew Cluster, M.D. principal investigator

Washington University School of Medicine

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

Questions about this trial

What is this clinical trial studying?

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 33 inclusion and 6 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 31 Jan 2024. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Safety as measured by absence of grade 4 or 5 non-hematological or grade 5 hematological toxicity (time frame: Through 1 year post-transplant). The study lists 2 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 NCT01661400.

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 31 Jan 2024.