Growth Failure Clinical Trial
Official title:
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency (XSCID): A Phase 2 Study Assessing Safety and Clinical Response to Treatment With Insulin-like Growth Factor-1 (IGF-1)
This study will evaluate the safety and effectiveness of insulin-like growth factor-1
(IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID). Those who
have XSCID lack white blood cells that protect their bodies from invasion by all types of
germs. IGF-1 is the main hormone responsible for the body's growth and metabolism. As a
medication, IGF-1 is Increlex[(Trademark)] (mecasermin),
Patients ages 2 to 20 who have not yet begun puberty, have a diagnosis of XSCID, and are
shorter than the 3rd percentile for their age may be eligible for this study. This study
will last about 3 years, and patients' visits will be scheduled at 3-month intervals.
Patients will have a physical history and exam, X-rays, electrocardiogram, blood tests, and
body measurements.
Patients will take estradiol orally for 2 days, to help avoid false results of growth
hormone (GH) levels in blood samples. Then provocation testing is done, with two tests back
to back. It determines blood levels of GH and the body's response to testing with drugs
called arginine and clonidine. Patients are admitted to the pediatric inpatient unit and
will have an intravenous (IV) line placed in the arm. Arginine is given by IV over 30
minutes, and blood samples are taken. Right after arginine testing, the clonidine tablet is
given. The IGF-1 generation test is then done to see if the body makes IGF-1 as a product in
response to injections of GH for 5 consecutive days. This test does not require that
patients are inpatients, but after Day 8, patients must be admitted to the pediatric unit to
have blood sampling, start Increlex injections, and start close monitoring of blood sugar
levels. They will learn how to do a self-injection and follow other advice. They will
complete records about the injection site, symptoms, and side effects-keeping records for at
least the first 2 days after going home, with each dose change, and as needed. Patients
stick their fingertip and place a small drop of blood on a blood sugar monitoring strip. The
strip is put into a glucometer-a small hand-held device to measure the blood sugar level.
Patients will be instructed to always have a source of sugar available in case blood sugar
is too low.
Status | Terminated |
Enrollment | 6 |
Est. completion date | December 2012 |
Est. primary completion date | December 2012 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 2 Years to 20 Years |
Eligibility |
- INCLUSION CRITERIA: Participants Must: - Have a diagnosis of XSCID - Be between 2 years to 20 years old and have not completed puberty - Consent to permit blood and/or tissue samples for storage - Demonstrate short stature: height below the 3 rd percentile for chronological age - Have a primary care physician at home - Demonstrate growth failure, defined as growth velocity (measured as linear growth) that is less than 5% to 10% of that expected for children of the same age group, over the past 12 months - Willingness to remain hospitalized for several days - Provide evidence of serum IGF-1 level performed within the preceding 6 months and the results fall below normal limits for age EXCLUSION CRITERIA: Participants Must NOT: - Have fusion of epiphyseal plates - Demonstrate any history of anaphylactic reaction or hypersensitivity to mecasermin or any component of the drug's formulation - Have any active or suspected neoplasia - Demonstrate signs of intracranial hypertension as evidenced by papilledema upon examination by fundoscopy - Have any condition that, in the investigator's opinion, places the patient at undue risk by participating in the study - Be unwilling to undergo testing or procedures associated with this protocol |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Allergy and Infectious Diseases (NIAID) |
United States,
Buckley RH, Schiff RI, Schiff SE, Markert ML, Williams LW, Harville TO, Roberts JL, Puck JM. Human severe combined immunodeficiency: genetic, phenotypic, and functional diversity in one hundred eight infants. J Pediatr. 1997 Mar;130(3):378-87. — View Citation
Leonard WJ, Shores EW, Love PE. Role of the common cytokine receptor gamma chain in cytokine signaling and lymphoid development. Immunol Rev. 1995 Dec;148:97-114. Review. — View Citation
Puck JM, Pepper AE, Henthorn PS, Candotti F, Isakov J, Whitwam T, Conley ME, Fischer RE, Rosenblatt HM, Small TN, Buckley RH. Mutation analysis of IL2RG in human X-linked severe combined immunodeficiency. Blood. 1997 Mar 15;89(6):1968-77. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Safety of Study Drug | Rates of adverse events related to study drug | 1 year | Yes |
Primary | Change in Growth Rate on Study Drug | Growth rate on intervention is compared with growth rate before intervention for each participant. | During intervention, up to 2 years | No |
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