Treatment of Rett Syndrome With Recombinant Human IGF-1 - Full Text View

Purpose

Investigators are recruiting children for a clinical trial using the medication recombinant human IGF-1 (a.k.a. mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). While IGF-1 is approved by the Food & Drug Administration (FDA) for certain use in children, it is considered an investigational drug in this trial because it has not previously been used to treat RTT. Information from this study will help determine if IGF-1 effectively treats RTT but will not necessarily lead to FDA approval of IGF-1 as a treatment for RTT.

Condition	Intervention	Phase
Rett Syndrome	Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1) Drug: Placebo	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment
Official Title:	Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)

Resource links provided by NLM:

Genetics Home Reference related topics: MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome Rett syndrome

MedlinePlus related topics: Anxiety Diabetes Medicines Rett Syndrome

Drug Information available for: Insulin human Insulin-like growth factor I Mecasermin Mecasermin rinfabate

U.S. FDA Resources

Further study details as provided by Children's Hospital Boston:

Primary Outcome Measures:

Rett Syndrome Behavior Questionnaire (RSBQ) [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Clinical Global Impression Scales [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Parent Global Impression Scales [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Parent Targeted Symptoms Visual Analog Scale [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Kerr Clinical Severity Scale [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Quantitative Measures of Respiration [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Mullen Scales of Early Learning (MSEL) [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Vineland Adaptive Behavior Scales II (VABS-II) [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Communication and Symbolic Behavior Scales Developmental Profile [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Aberrant Behavior Checklist (ABC) [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
Physical Examination [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Parent Current Clinical Report [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: Yes ]

Other Outcome Measures:

Concomitant medication log [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: Yes ]
Monitoring System of Side Effects (MOSES) [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: Yes ]
SF-36v2 ("Your Health and Well-Being") [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Vital signs [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Growth measurements [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Electrocardiogram [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Laboratory tests [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: Yes ]
Scoliosis x-ray [ Time Frame: At the beginning and end of study (twice over 50 week span) ] [ Designated as safety issue: Yes ]
Bone age x-ray [ Time Frame: At the beginning and end of study (twice over 50 week span) ] [ Designated as safety issue: Yes ]
Echocardiogram [ Time Frame: At the beginning and end of study (twice over 50 week span) ] [ Designated as safety issue: Yes ]
Evaluation of cortical function using Visual Evoked Potentials [ Time Frame: At the beginning and end of each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Evaluation of hand stereotypies with the QSensor© accelerometer [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: No ]
Visual Analog Scale - Hand Stereotypy [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ] [ Designated as safety issue: No ]
RNA expression profiling [ Time Frame: Optional; Every 10 weeks during each of the two 20-week treatment periods ] [ Designated as safety issue: No ]

Estimated Enrollment:	30
Study Start Date:	January 2013
Estimated Study Completion Date:	July 2014
Estimated Primary Completion Date:	July 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Treatment Period 1 One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) , and the other half of subjects will be randomly assigned to receive placebo.	Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1) Subjects will receive twice daily subcutaneous injections of IGF-1. Other Names: mecasermin [rDNA] injection Increlex Drug: Placebo Subjects will receive twice daily subcutaneous injections of a saline solution (placebo). Other Name: saline
Placebo Comparator: Treatment Period 2 Subjects that initially received Recombinant Human Insulin Growth Factor 1 (rhIGF-1) will now receive placebo, and subjects that initially received placebo will now receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1).	Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1) Subjects will receive twice daily subcutaneous injections of IGF-1. Other Names: mecasermin [rDNA] injection Increlex Drug: Placebo Subjects will receive twice daily subcutaneous injections of a saline solution (placebo). Other Name: saline

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Eligibility

Ages Eligible for Study:	5 Years to 10 Years
Genders Eligible for Study:	Female
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Diagnosis of "classic" or "typical" Rett Syndrome
Genetic documentation of MECP2 mutation
Subject and caregiver's primary language must be English
Subject must reside in the USA
Caregiver must have internet access and be able to complete questionnaires online and communicate via email
Subject is stable on current medications for at least 4 weeks

Exclusion Criteria:

Severe scoliosis (curvature >40 degrees)
Bone-age greater than 11
Cardiomegaly (enlarged heart)
Tanner stage 2 or higher breast development
Allergy to IGF-1
Prior use of IGF-1, growth hormone, or sex steroids

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01777542

Contacts

Contact: Katherine Barnes	617-355-5230	rettresearch@childrens.harvard.edu
Contact: Nicole Cantwell	617-355-5173	rettresearch@childrens.harvard.edu

Locations

United States, Massachusetts
Boston Children's Hospital	Recruiting
Boston, Massachusetts, United States, 02215

Sponsors and Collaborators

Children's Hospital Boston

International Rett Syndrome Foundation

Autism Speaks

Investigators

Principal Investigator:

Walter E. Kaufmann, MD

Boston Children's Hospital

More Information

No publications provided

Responsible Party:	Walter Kaufmann, Visiting Professor of Neurology, Children's Hospital Boston
ClinicalTrials.gov Identifier:	NCT01777542 History of Changes
Other Study ID Numbers:	IRB-P00005610
Study First Received:	January 23, 2013
Last Updated:	January 24, 2013
Health Authority:	United States: Institutional Review Board United States: Food and Drug Administration

Keywords provided by Children's Hospital Boston:

Rett syndrome
RTT
IGF-1
autism spectrum disorder

Additional relevant MeSH terms:

Rett Syndrome
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked

Genetic Diseases, Inborn
Mitogens
Insulin
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Hypoglycemic Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 19, 2013