Brain EdemaPhase 1/2NCT01369121
Tolerability Study of Xerecept® in Pediatric Patients
A Phase 1/2 study testing XERECEPT in people with Brain Edema and Brain Tumor, run by Celtic Pharma Development Services at 2 sites in United States.
Trial summary
This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Brain Edema, Brain Tumor
- What is being tested?
- XERECEPT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- Jun 2010to Mar 2013
- Phase
- Phase 1/2
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have histologically confirmed primary, recurrent or metastatic cerebral tumors and require chronic steroid dosing to manage symptoms of peritumoral brain edema (No histological confirmation is required of optic pathway tumors or brain stem gliomas)
- Have been taking dexamethasone (or equivalent) at a dose of ≥1 mg/day for a minimum of 14 days and have had a stable dose for at least 7 days.
- Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)
- Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.
- Karnofsky/Lansky performance status ≥ 40
- Life expectancy of at least 6 months
- Female subjects must not be pregnant or breast-feeding and, if of childbearing potential, must use adequate methods of contraception during the study
- 10. Ability to provide written informed consent or, if unable to provide, have a legal guardian or representative provide written informed
Who cannot join
- Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
- Subject and/or parent/guardian is unwilling or unable to comply with this protocol
- Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
- Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Dana-Farber Cancer Institute Pediatric Oncology
Boston, Massachusetts, 02115
Children's Memorial Hospital, Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60614
About this study
Official title: A Phase I/II Open Label Individual Dose Titration Trial of the Human Corticotropin- Releasing Factor (hCRF), Corticorelin Acetate Injection (Xerecept®), to Determine the Tolerability of Xerecept® in a Pediatric Population
This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials.
Read the detailed description
Steroid-related side effects are much more severe in a pediatric population than they are in adults. The side effects appear more quickly and are often apparent in as few as 14 days. Dexamethasone side effects in children include rounding of the face, acne, increased body hair, muscle weakness, osteoporosis, mood changes, weight gain, fluid retention, glucose instability, high blood pressure, increased susceptibility to infection, stunted growth and aseptic necrosis of the hip joints14-16.
Although extensive studies have been performed with Xerecept® in adults, no studies have been performed with Xerecept® in pediatric subjects. Therefore, this study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. The study will also explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
- Conditions
- Brain Edema, Brain Tumor
- Being tested
- XERECEPT
- Keywords
- peritumoral brain edema, edema, malignant brain tumor, brain tumor, dexamethasone, Decadron
- Medical subject headings
- Brain Edema, Brain Neoplasms, Edema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XERECEPTDrug
Also called hCRF, Corticorelin acetate injection
BID dosing, subcutaneous for 1 year
| Study group | Role | What they receive |
|---|---|---|
| Xerecept | Experimental | All patients will receive hCRF (XERECEPT) Receives: Drug: XERECEPT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose
To define the maximum tolerated dose (MTD) of Xerecept® in pediatric patients; the MTD will be defined as the dose at which 1 or fewer of six patients experiences a dose limiting adverse event of any kind.
Measured: 2 months
Secondary outcomes (5)
Dexamethasone Dosing
To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
Measured: 1 Year
Incidence and severity of specified Steroid-Related Side Effects
To explore the clinical benefit associated with a reduction in steroid dosing over the course of the study by comparing the incidence and severity of pre-specified steroid-related side effects in all patients.
Measured: 1 year
Number of patients with adverse events
Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients
Measured: 1 Year
Change from baseline in clinical chemistry, hematology and urinalysis measures
Standard clinical chemistry, hematology and urinalysis measures will be analyzed at baseline and monthly during the study; changes from baseline will be measured over the course of the study
Measured: 1 Year
PedsQL™ Quality of Life Inventory Scores
Child self report and parent proxy reports conducted at each study visit to assess changes in health-related quality of life over the course of the study
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2011 (estimated)
- Study startJun 2010
- Primary completionOct 2012
- Study completionMar 2013
Why it stopped: Single subject remained on study - transferred to compassionate use
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain EdemaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celtic Pharma Development Services
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stewart Goldman, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01369121
- Sponsor's protocol ID
- CPDS 1001
- Status last verified
- Jul 2013
- First submitted
- 7 Jun 2011
- Last updated
- 30 Jul 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open Label Individual Dose Titration Trial of the Human Corticotropin- Releasing Factor (hCRF), Corticorelin Acetate Injection (Xerecept®), to Determine the Tolerability of Xerecept® in a Pediatric Population
- ClinicalTrials.gov ID
- NCT01369121
- Acronym / sponsor ID
- CPDS 1001
- Conditions
- Brain Edema, Brain Tumor
- Keywords
- peritumoral brain edema, edema, malignant brain tumor, brain tumor, dexamethasone, Decadron
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Single subject remained on study - transferred to compassionate use
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Celtic Pharma Development ServicesIndustry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Oct 2012 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 8 Jun 2011 (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.
This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials.
Detailed description
Steroid-related side effects are much more severe in a pediatric population than they are in adults. The side effects appear more quickly and are often apparent in as few as 14 days. Dexamethasone side effects in children include rounding of the face, acne, increased body hair, muscle weakness, osteoporosis, mood changes, weight gain, fluid retention, glucose instability, high blood pressure, increased susceptibility to infection, stunted growth and aseptic necrosis of the hip joints14-16.
Although extensive studies have been performed with Xerecept® in adults, no studies have been performed with Xerecept® in pediatric subjects. Therefore, this study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. The study will also explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Xerecept | Experimental | Drug: XERECEPTAll patients will receive hCRF (XERECEPT) |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Have histologically confirmed primary, recurrent or metastatic cerebral tumors and require chronic steroid dosing to manage symptoms of peritumoral brain edema (No histological confirmation is required of optic pathway tumors or brain stem gliomas)
- Have been taking dexamethasone (or equivalent) at a dose of ≥1 mg/day for a minimum of 14 days and have had a stable dose for at least 7 days.
- Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)
- Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.
- Karnofsky/Lansky performance status ≥ 40
Key exclusion criteria (4 listed)
- Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
- Subject and/or parent/guardian is unwilling or unable to comply with this protocol
- Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
- Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).
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 |
|---|---|---|
| XERECEPTAlso: hCRF, Corticorelin acetate injectionBID dosing, subcutaneous for 1 year | Drug | Xerecept |
Primary outcome
Maximum Tolerated Dose
Time frame: 2 months
To define the maximum tolerated dose (MTD) of Xerecept® in pediatric patients; the MTD will be defined as the dose at which 1 or fewer of six patients experiences a dose limiting adverse event of any kind.
5 secondary outcomes
Dexamethasone Dosing
Time frame: 1 Year
Incidence and severity of specified Steroid-Related Side Effects
Time frame: 1 year
Number of patients with adverse events
Time frame: 1 Year
Change from baseline in clinical chemistry, hematology and urinalysis measures
Time frame: 1 Year
PedsQL™ Quality of Life Inventory Scores
Time frame: 1 Year
Results and safety
The study was stopped early (reason given: Single subject remained on study - transferred to compassionate use). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2013.
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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.
Stewart Goldman, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing XERECEPT in people with Brain Edema and Brain Tumor, run by Celtic Pharma Development Services at 2 sites 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 8 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: stopped early, as of the sponsor's last update on 30 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined 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.
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?
The study was stopped early (reason given: Single subject remained on study - transferred to compassionate use). Results are not available in the source record.
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?
Maximum Tolerated Dose (time frame: 2 months). 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 30 Jul 2013.