Eosinophilic EsophagitisPhase 2/3NCT00538434
Efficacy and Safety Study of Reslizumab to Treat Eosinophilic Esophagitis in Subjects Aged 5 to 18 Years
A Phase 2/3 study testing Reslizumab and Saline in people with Eosinophilic Esophagitis, run by Ception Therapeutics at 37 sites in 2 countries.
Trial summary
This trial will study three doses of reslizumab versus placebo in children with eosinophilic esophagitis (EE). The objectives of the trial will be to study the effectiveness of reslizumab in improving the clinical signs and symptoms and reducing esophageal eosinophils as well as assessing the safety profile compared… Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Eosinophilic Esophagitis
- What is being tested?
- Reslizumab and Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 227 participants
- Where
- 37 sitesin 2 countries
- Study dates
- Mar 2008to Oct 2009
- Phase
- Phase 2/3
- 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 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- written informed consent obtained
- male or female patients aged 5 to 18 years at time of screening
- of non-childbearing potential, of childbearing potential and willing to use specific barrier methods outlined in the protocol
- confirmed active EE (at Screening or within six weeks prior to Baseline Visit) as defined by esophageal mucosal eosinophils greater than or equal to 24 per high power field (hpf; 400X magnification)
- within the week prior to dosing, patient has one of the following symptoms of moderate (or worse) severity: vomiting, regurgitation (acid taste or feeling material movement upward), abdominal, chest pain/heartburn (burning or pain behind the sternum), or difficulty swallowing
- been on a therapeutic dose of proton pump inhibitors (PPIs; with or without histamine H2 receptor antagonists)for at least four weeks without resolution of symptoms, or by negative pH probe (with or without having failed a course of PPIs)
Who cannot join
- another disorder that causes esophageal eosinophilia (e.g., hypereosinophilic syndrome [HES],Churg Strauss vasculitis, eosinophilic gastroenteritis [EG], or a parasitic infection)
- history of abnormal gastric or duodenal biopsy or documented gastrointestinal [GI] disorders (e.g., celiac disease, Crohn's disease or Helicobacter pylori infection)
- history of the following GI surgeries: fundoplication, gastric surgery or surgery for intestinal atresia
- use of systemic immunosuppressive or immunomodulating agents (anti-immunoglobulin E [IgE] monoclinal antibody [mAb], methotrexate, cyclosporin, interferon alpha [α], or anti tumor necrosis factor [TNF] mAb) within six months prior to study entry
- received attenuated live attenuated vaccines (e.g., measles, mumps, rubella [MMR], bacillus Calmette-Guerin [BCG], varicella, FluMist or polio) within three months prior to study entry
- use of swallowed inhaled corticosteroids for the treatment of EE within one month prior to study entry. Note: Inhaled and nasal corticosteroids for the treatment of asthma and allergies, respectively, are permitted provided that the dose remains the same during the study
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- a stricture on endoscopy that prevents passage of the endoscope
- participation in any investigational drug or device study within 30 days prior to study entry
- female subjects who are pregnant or nursing
- concurrent infection or disease that may preclude assessment of EE
- concurrent immunodeficiency (human immunodeficiency [HIV], or acquired immunodeficiency syndrome [AIDS] or congenital immunodeficiency)
Trial locations and contacts
37 sites in 2 countries.
37 sites in 2 countries
United States34 sites
Children's Center for Digestive Health Care
Atlanta, Georgia, 30342
Denver Childrens At Aurora, Colorado
Aurora, Colorado, 80045
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215
The Children's Hospital of Alabama
Birmingham, Alabama, 35233
Tuft's Floating Hospital
Boston, Massachusetts, 02111
1st Allergy and Clinical Research Center
Centennial, Colorado, 80112
University of Chicago
Chicago, Illinois, 60637
Children'S Memorial Hospital Division of Gastroenterology Hepatology & Nutrition
Chicago, Illinois, 66014
Cincinnati Children's
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwest Medical Center
Dallas, Texas, 75390
Pediatric Allergy and Immunology of Duke Medical Center
Durham, North Carolina, 27720
Greenville Health System
Greenville, South Carolina, 29615
Kaiser Permanente Hospital- Pediatric Gastroenterology
Hayward, California, 94545
Riley Hospital for Children
Indianapolis, Indiana, 46202
Las Vegas Pediatric Gastroenterology Associates
Las Vegas, Nevada, 89109
Arkansas Children's Hospital/University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72202
South Jersey Pediatric Gastroenterology
Mays Landing, New Jersey, 08330
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Mount Sinai School of Medicine, Pediatrics
New York, New York, 10029
Creighton University Medical Center
Omaha, Nebraska, 68131
Children'S Hospital of Orange County Pediatric Subspecialty Faculty Division of Allergy and Asthma
Orange, California, 92868
Pediatric Allergy/Immunology
Palo Alto, California, 94305
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Minnesota Gastroenterology
Plymouth, Minnesota, 55446
Virginia Commonwealth University
Richmond, Virginia, 23219
Carilion Medical Center for Children
Roanoke, Virginia, 24013
University of Utah School of Medicine
Salt Lake City, Utah, 84113
Children's Hospital of San Diego
San Diego, California, 92123
Saint Louis University
St Louis, Missouri, 63104
State University of New York (SUNY)
Syracuse, New York, 13210
University of Arizona Dept. of Pediatrics
Tucson, Arizona, 85724
Center for Digestive Allergic and Immunologic Diseases
Williamsville, New York, 14221
Thomas Jefferson University Medical College
Wilmington, Delaware, 19803
Canada3 sites
Pediatric Allergy and Immunology
Edmonton, Alberta, T6G 2C8
Pediatric Allergy & Immunology
Edmonton, Alberta, T6G2C8
University of Montreal
Montreal, Quebec, H3T 1C5
About this study
Official title: An Efficacy and Safety Study of Reslizumab (CTx55700) in the Treatment of Eosinophilic Esophagitis in Subjects Aged 5 to 18 Years
This trial will study three doses of reslizumab versus placebo in children with eosinophilic esophagitis (EE). The objectives of the trial will be to study the effectiveness of reslizumab in improving the clinical signs and symptoms and reducing esophageal eosinophils as well as assessing the safety profile compared to placebo.
- Conditions
- Eosinophilic Esophagitis
- Being tested
- Reslizumab, Saline
- Keywords
- Eosinophilic Esophagitis, GERD, EE, Reslizumab, IL-5, Interleukin-5, Cinquil
- Medical subject headings
- Eosinophilic Esophagitis, Gastroesophageal Reflux
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReslizumabBiological
Also called CTx55700, Cinquil™, CEP-38072
SalineOther
| Study group | Role | What they receive |
|---|---|---|
| Reslizumab 1 mg/kg | Experimental | reslizumab 1 mg/kg intravenous (IV) on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles Receives: Biological: Reslizumab |
| Reslizumab 2 mg/kg | Experimental | reslizumab 2 mg/kg IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles Receives: Biological: Reslizumab |
| Reslizumab 3 mg/kg | Experimental | reslizumab 3 mg/kg IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles Receives: Biological: Reslizumab |
| Placebo | Placebo comparator | saline placebo IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles Receives: Other: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Percent Change From Baseline to End of Treatment in Peak Esophageal Eosinophil (EE) Levels
Participants underwent esophagogastroduodenoscopy (EGD) with biopsy (2 biopsies each at proximal and distal esophageal locations, plus any inflamed or abnormal areas) per standard clinical practice for the determination of esophageal eosinophils.
Measured: Baseline, End of Treatment (up to 15 weeks [+/- 4 days])
Mean Change From Baseline in Physician's Esophageal Eosinophil (EE) Global Assessment At The End-of-Treatment Visit (or at Early Withdrawal)
The investigator completed the Physician's EE Global Assessment based upon the participant's reporting of symptoms, weight, dietary status, and overall well-being. The assessment rated severity on a five-point scale (0=none to 4=very severe), taking into account physical findings, vital signs, the Subject's Predominant EE Symptom Assessment, the subject's diary data, and dietary questions. The Subject's Predominant EE Symptom was the EE symptom (vomiting/regurgitation, abdominal/chest pain, or dysphagia) that had the greatest negative impact on the subject based on patient diary data as of the baseline visit.
The full range for change from baseline values is -4 (very severe at baseline, none at end of study) to 4 (none at baseline, severe at end of study). Negative change from baseline scores in the Physician's EE Global Assessment indicate improvement in EE status.
Measured: Baseline (Day 1, pre-treatment), End of Treatment (Week 15, 3 weeks [± 4 days] after the last dose of study drug, or at early withdrawal)
Secondary outcomes (2)
Mean Change From Baseline to End of Treatment in EE Predominant Symptom Assessment
Participants rated the severity of each EE symptom (vomiting/regurgitation, abdominal/chest pain, and dysphagia) based on the previous week's daily diary as none (=0), mild, moderate, severe, or very severe (=4). The predominant symptom was selected at the baseline visit and remained the same throughout the trial. The predominant symptom was defined as the EE symptom that had the greatest negative impact on the participant. The full range for change from baseline values is -4 (very severe at baseline, none at end of study) to 4 (none at baseline, severe at end of study). Negative change from baseline scores in the Patient's EE Predominant Symptom Assessment indicate improvement in the selected symptom.
Measured: Baseline (Day 1, pre-treatment), End of Treatment (Week 15, 3 weeks [± 4 days] after the last dose of study drug, or at early withdrawal)
Mean Percent Change From Baseline to End of Treatment in the Child Health Questionnaire (CHQ)
CHQ is a quality-of-life (QoL), observer-rated (the parent in this study) instrument designed to assess the general health and well-being of pediatric subjects aged 5 to 18 years. The instrument comprises 50 items that cover 14 unique physical and psychological concepts. Each item was scored separately following different scales and timeframes for response. Proprietary scoring algorithms are used. This outcome reports the two CHQ Summary Scores (Physical Summary Score and the Psychosocial Summary Scores) which are indexed to a 0 (poorest quality of life) to 100 (best quality of life) scores. The two summary scores are subsequently combined (via proprietary algorithm) to create the Global Health Summary Score (also on a 0-100 scale). Percent change from baseline values range from 100% (poorest QoL at baseline, best QoL at end of treatment) to -100% (best QoL at baseline, poorest QoL at end of treatment). Higher percent change from baseline values indicate improved QoL.
Measured: Baseline, End of Treatment (up to 15 weeks +/- 4 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2007 (estimated)
- Study startMar 2008
- Primary completionOct 2009
- Study completionOct 2009
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Eosinophilic EsophagitisStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 227 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ception Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sponsor's Medical Expert, MD, Study director, Cephalon (Ception)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00538434
- Sponsor's protocol ID
- Res-05-0002
- Status last verified
- Jul 2016
- First submitted
- 1 Oct 2007
- Last updated
- 2 Sep 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Spergel JM, Rothenberg ME, Collins MH, Furuta GT, Markowitz JE, Fuchs G 3rd, O'Gorman MA, Abonia JP, Young J, Henkel T, Wilkins HJ, Liacouras CA. Reslizumab in children and adolescents with eosinophilic esophagitis: results of a double-blind, randomized, placebo-controlled trial. J Allergy Clin Immunol. 2012 Feb;129(2):456-63, 463.e1-3. doi: 10.1016/j.jaci.2011.11.044. Epub 2011 Dec 28.
PubMed 22206777
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Efficacy and Safety Study of Reslizumab (CTx55700) in the Treatment of Eosinophilic Esophagitis in Subjects Aged 5 to 18 Years
- ClinicalTrials.gov ID
- NCT00538434
- Acronym / sponsor ID
- Res-05-0002
- Conditions
- Eosinophilic Esophagitis
- Keywords
- Eosinophilic Esophagitis, GERD, EE, Reslizumab, IL-5, Interleukin-5, Cinquil
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 227 participants (actual)
- Lead sponsor
- Ception TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Mar 2008
- Primary completion
- Oct 2009 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 2 Oct 2007 (estimated)
- Results
- Posted 2 Sep 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial will study three doses of reslizumab versus placebo in children with eosinophilic esophagitis (EE). The objectives of the trial will be to study the effectiveness of reslizumab in improving the clinical signs and symptoms and reducing esophageal eosinophils as well as assessing the safety profile compared to placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Reslizumab 1 mg/kg | Experimental | Biological: Reslizumabreslizumab 1 mg/kg intravenous (IV) on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles |
| Reslizumab 2 mg/kg | Experimental | Biological: Reslizumabreslizumab 2 mg/kg IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles |
| Reslizumab 3 mg/kg | Experimental | Biological: Reslizumabreslizumab 3 mg/kg IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles |
| Placebo | Placebo comparator | Other: Salinesaline placebo IV on Day 0 of each 28-day (+/-7 days) cycle, for up to 4 cycles |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- written informed consent obtained
- male or female patients aged 5 to 18 years at time of screening
- of non-childbearing potential, of childbearing potential and willing to use specific barrier methods outlined in the protocol
- confirmed active EE (at Screening or within six weeks prior to Baseline Visit) as defined by esophageal mucosal eosinophils greater than or equal to 24 per high power field (hpf; 400X magnification)
- within the week prior to dosing, patient has one of the following symptoms of moderate (or worse) severity: vomiting, regurgitation (acid taste or feeling material movement upward), abdominal, chest pain/heartburn (burning or pain behind the sternum), or difficulty swallowing
Key exclusion criteria (11 listed)
- another disorder that causes esophageal eosinophilia (e.g., hypereosinophilic syndrome [HES],Churg Strauss vasculitis, eosinophilic gastroenteritis [EG], or a parasitic infection)
- history of abnormal gastric or duodenal biopsy or documented gastrointestinal [GI] disorders (e.g., celiac disease, Crohn's disease or Helicobacter pylori infection)
- history of the following GI surgeries: fundoplication, gastric surgery or surgery for intestinal atresia
- use of systemic immunosuppressive or immunomodulating agents (anti-immunoglobulin E [IgE] monoclinal antibody [mAb], methotrexate, cyclosporin, interferon alpha [α], or anti tumor necrosis factor [TNF] mAb) within six months prior to study entry
- received attenuated live attenuated vaccines (e.g., measles, mumps, rubella [MMR], bacillus Calmette-Guerin [BCG], varicella, FluMist or polio) within three months prior to study entry
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 |
|---|---|---|
| ReslizumabAlso: CTx55700, Cinquil™, CEP-38072 | Biological | Reslizumab 1 mg/kg, Reslizumab 2 mg/kg, Reslizumab 3 mg/kg |
| Saline | Other | Placebo |
Primary outcome
Mean Percent Change From Baseline to End of Treatment in Peak Esophageal Eosinophil (EE) Levels
Time frame: Baseline, End of Treatment (up to 15 weeks [+/- 4 days])
Participants underwent esophagogastroduodenoscopy (EGD) with biopsy (2 biopsies each at proximal and distal esophageal locations, plus any inflamed or abnormal areas) per standard clinical practice for the determination of esophageal eosinophils.
Mean Change From Baseline in Physician's Esophageal Eosinophil (EE) Global Assessment At The End-of-Treatment Visit (or at Early Withdrawal)
Time frame: Baseline (Day 1, pre-treatment), End of Treatment (Week 15, 3 weeks [± 4 days] after the last dose of study drug, or at early withdrawal)
The investigator completed the Physician's EE Global Assessment based upon the participant's reporting of symptoms, weight, dietary status, and overall well-being. The assessment rated severity on a five-point scale (0=none to 4=very severe), taking into account physical findings, vital signs, the Subject's Predominant EE Symptom Assessment, the subject's diary data, and dietary questions. The Subject's Predominant EE Symptom was the EE symptom (vomiting/regurgitation, abdominal/chest pain, or dysphagia) that had the greatest negative impact on the subject based on patient diary data as of the baseline visit. The full range for change from baseline values is -4 (very severe at baseline, none at end of study) to 4 (none at baseline, severe at end of study). Negative change from baseline scores in the Physician's EE Global Assessment indicate improvement in EE status.
2 secondary outcomes
Mean Change From Baseline to End of Treatment in EE Predominant Symptom Assessment
Time frame: Baseline (Day 1, pre-treatment), End of Treatment (Week 15, 3 weeks [± 4 days] after the last dose of study drug, or at early withdrawal)
Mean Percent Change From Baseline to End of Treatment in the Child Health Questionnaire (CHQ)
Time frame: Baseline, End of Treatment (up to 15 weeks +/- 4 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 22206777Spergel JM, Rothenberg ME, Collins MH, Furuta GT, Markowitz JE, Fuchs G 3rd, O'Gorman MA, Abonia JP, Young J, Henkel T, Wilkins HJ, Liacouras CA. Reslizumab in children and adolescents with eosinophilic esophagitis: results of a double-blind, randomized, placebo-controlled trial. J Allergy Clin Immunol. 2012 Feb;129(2):456-63, 463.e1-3. doi: 10.1016/j.jaci.2011.11.044. Epub 2011 Dec 28.
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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.
Sponsor's Medical Expert, MD study director
Cephalon (Ception)
37 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Reslizumab and Saline in people with Eosinophilic Esophagitis, run by Ception Therapeutics at 37 sites in 2 countries.
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 6 inclusion and 11 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 2 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Canada. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 2 Sep 2016). 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?
Mean Percent Change From Baseline to End of Treatment in Peak Esophageal Eosinophil (EE) Levels (time frame: Baseline, End of Treatment (up to 15 weeks [+/- 4 days])). 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 NCT00538434, which also lists 1 linked publication.
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 2 Sep 2016.