Cyproheptadine in Preventing Weight Loss in Children Receiving Chemotherapy for Cancer
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ClinicalTrials.gov Identifier: NCT01132547 |
Recruitment Status
:
Terminated
(The study was terminated due to slow accrual.)
First Posted
: May 28, 2010
Results First Posted
: July 2, 2015
Last Update Posted
: July 2, 2015
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RATIONALE: Cyproheptadine hydrochloride may prevent weight loss caused by cancer or cancer treatment. It is not yet known whether cyproheptadine is more effective than a placebo in preventing weight loss in young patients receiving chemotherapy for cancer.
PURPOSE: This randomized phase III trial is studying cyproheptadine hydrochloride to see how well it works in preventing weight loss in young patients receiving chemotherapy for cancer.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cancer | Drug: cyproheptadine hydrochloride Other: placebo | Phase 3 |
OBJECTIVES:
Primary
- To determine the effect of cyproheptadine hydrochloride in the prevention of cancer- or treatment-related weight loss (defined as ≥ 5% reduction in weight from baseline measurement) in children who are initiating a course of moderately or highly emetic chemotherapy.
Secondary
- To investigate the effect of cyproheptadine HCl on the change in weight for age scores after 8 weeks of study drug administration in comparison to placebo.
- Investigate the relationship between the secondary outcome variables (prealbumin, triceps skin fold, mid-upper arm circumference, and weight loss)from baseline to end of treatment in each group (treatment and placebo) separately.
OUTLINE: This is a multicenter study. Patients are stratified according to enrolling center and steroid use with cancer treatment (yes vs no). Study agent can start anytime up to and including day 28 after the first dose of chemotherapy.
- Arm I: Patients receive oral cyproheptadine hydrochloride twice daily for 8 weeks.
- Arm II: Patients receive an oral placebo twice daily for 8 weeks.
Patients undergo weight and height measurements at baseline and at each follow-up visit in weeks 4 and 8 to evaluate the effect of cyproheptadine hydrochloride and duration of response. Patients or parents complete medicine logs at each follow-up visit in weeks 4 and 8 to evaluate drug compliance and tolerance. Patients also undergo measures of nutrition; and measures of body composition, lean body mass, and fat percentage using standardized equipment and procedures for measuring triceps skin fold and mid-arm muscle circumference at baseline and at the end of the study.
Patients undergo blood sample collection at baseline and at the end of the study for biomarker studies. Samples are analyzed for pre-albumin levels.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 22 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Supportive Care |
Official Title: | Prevention of Cancer/Treatment-Related Weight Loss in Children at High Nutritional Risk |
Study Start Date : | June 2010 |
Actual Primary Completion Date : | January 2014 |
Actual Study Completion Date : | January 2014 |

Arm | Intervention/treatment |
---|---|
Experimental: Arm I cyproheptadine hydrochloride
Patients receive oral cyproheptadine hydrochloride twice daily for 8 weeks.
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Drug: cyproheptadine hydrochloride
Given orally
Other Name: cyproheptadine HCl
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Placebo Comparator: Arm II placebo
Patients receive an oral placebo twice daily for 8 weeks.
|
Other: placebo
Given orally
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- Participant With Weight Loss ≥ 5% at the 8- Week Assessment When Compared to Baseline [ Time Frame: 8 weeks ]
- Severity of Weight Loss [ Time Frame: Baseline and 8 weeks ]Change from Baseline in Weight Z score
- Pattern of Weight in the Study Population [ Time Frame: Baseline and 8 weeks ]Change from Baseline in Weight

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Ages Eligible for Study: | 2 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
INCLUSION CRITERIA:
- ≥ 2 years and ≤ 21 years of age at the time of study entry
- Scheduled to receive chemotherapy for:
- Newly diagnosed:
- Non-rhabdo soft tissue sarcomas, scheduled to receive chemotherapy, as well as intermediate or high-risk rhabdomyosarcoma, any stage osteosarcoma and any stage Ewing's sarcoma
- Intermediate or high-risk neuroblastoma
- Wilms' tumor (Stage III/IV)
- Hepatoblastoma (Stage III/IV)
- Germ cell tumors (Stage III/IV)
- Brain tumors, including medulloblastoma, PNET and ependymomas
- AML
- Relapsed/recurrent disease (any patient)
- Able to register and randomize within 28 days of starting chemotherapy (registration /randomization and start of study agent may occur at anytime up to and including Day 28 after the initiation of chemotherapy)
EXCLUSION CRITERIA:
- ≥ 29 days after starting chemotherapy
- Documented history of unintended weight loss ≥ 5% presumed secondary to cancer within 3 months of study entry
- Currently taking cyproheptadine HCl (or have taken cyproheptadine HCl within 3 weeks of study registration)
- History of anorexia nervosa or bulimia
- Taking other appetite-stimulating medications, i.e. dronabinol (Marinol) during the past three weeks.
- Initiation of other appetite enhancing agents, including steroids prescribed for the intent of weight gain, i.e. Megace. Note: Other forms of nutrition therapies, e.g. appetite-stimulating medications, TPN or enteral tube feedings are not allowed during this study.
- Children receiving steroids for >7 days as part of their cancer treatment regimen are excluded from participation. However, intermittent steroid use in an antiemetic regimen is allowed during the study
- Receiving monoamine oxidase (MAO) inhibitors, procarbazine, fluoxetine (Prozac), or paroxetine (Paxil)
- Diagnosed with glaucoma, cystic fibrosis, inflammatory bowel disease, or GI/GU obstruction
- Allergy to cyproheptadine HCl
- Females of childbearing age must not be pregnant.
- Female patients who are lactating must agree to stop breast-feeding.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01132547
United States, California | |
Miller Children's Hospital | |
Long Beach, California, United States, 90806 | |
United States, Connecticut | |
Connecticut Children's Medical Center | |
Hartford, Connecticut, United States, 06106 | |
United States, Delaware | |
A.I. duPont Hospital for Children | |
Wilmington, Delaware, United States, 19803 | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Florida | |
Children's Hospital of Southwest Florida at Lee Memorial | |
Fort Myers, Florida, United States, 33908 | |
Nemours Children's Clinic - Jacksonville | |
Jacksonville, Florida, United States, 32207-8482 | |
Arnold Palmer Hospital for Children | |
Orlando, Florida, United States, 32806 | |
Nemours Children's Clinic - Orlando | |
Orlando, Florida, United States, 32806 | |
Nemours Children's Hospital Pensacola | |
Pensacola, Florida, United States, 32504 | |
United States, Hawaii | |
Kapiolani Medical Center for Women and Children | |
Honolulu, Hawaii, United States, 96826 | |
United States, Louisiana | |
Ochsner Clinic Foundation | |
New Orleans, Louisiana, United States, 70121 | |
United States, New York | |
Columbia University Medical Center | |
New York, New York, United States, 10032 | |
United States, Texas | |
CHRISTUS Santa Rosa Children's Hospital | |
San Antonio, Texas, United States, 78207 | |
United States, Virginia | |
Children's Hospital of The King's Daughters | |
Norfolk, Virginia, United States, 23507 |
Principal Investigator: | Jeffrey P. Krischer, PhD | University of South Florida |
Responsible Party: | University of South Florida |
ClinicalTrials.gov Identifier: | NCT01132547 History of Changes |
Other Study ID Numbers: |
SCUSF 0703 SCUSF-0703 ( Other Identifier: SunCoast CCOP Research Base ) 5U10CA081920-11 ( U.S. NIH Grant/Contract ) |
First Posted: | May 28, 2010 Key Record Dates |
Results First Posted: | July 2, 2015 |
Last Update Posted: | July 2, 2015 |
Last Verified: | June 2015 |
Keywords provided by University of South Florida:
recurrent childhood subependymal giant cell astrocytoma untreated childhood subependymal giant cell astrocytoma cachexia weight changes nausea and vomiting alveolar childhood rhabdomyosarcoma anaplastic osteosarcoma childhood alveolar soft-part sarcoma childhood angiosarcoma childhood epithelioid sarcoma childhood fibrosarcoma childhood gliosarcoma childhood leiomyosarcoma childhood liposarcoma childhood neurofibrosarcoma |
childhood synovial sarcoma chondrosarcoma chondrosarcomatous osteosarcoma clear cell sarcoma of the kidney embryonal childhood rhabdomyosarcoma embryonal-botryoid childhood rhabdomyosarcoma endometrial stromal sarcoma extraosseous Ewing sarcoma peripheral primitive neuroectodermal tumor fibrosarcomatous osteosarcoma localized Ewing sarcoma localized osteosarcoma mast cell sarcoma metastatic childhood soft tissue sarcoma metastatic Ewing sarcoma |
Additional relevant MeSH terms:
Weight Loss Body Weight Changes Body Weight Signs and Symptoms Cyproheptadine Antipruritics Dermatologic Agents Gastrointestinal Agents Histamine H1 Antagonists |
Histamine Antagonists Histamine Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Serotonin Antagonists Serotonin Agents Anti-Allergic Agents |