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Treatment of Rett Syndrome With Recombinant Human IGF-1

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Children's Hospital Boston
Sponsor:
Collaborators:
International Rett Syndrome Foundation
Autism Speaks
Information provided by (Responsible Party):
Walter Kaufmann, Harvard Medical School
ClinicalTrials.gov Identifier:
NCT01777542
First received: January 23, 2013
Last updated: September 29, 2014
Last verified: September 2014
  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:


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 ]

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 ]

Other Outcome Measures:
  • 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 ]

Estimated Enrollment: 30
Study Start Date: January 2013
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (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

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   2 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 must be post-regression (Hagberg Stage 2)
  • Subject and caregiver's primary language must be English
  • Subject must reside in North America (US and Canada)
  • 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
  • Subject's regimen of non-pharmacological interventions (physical therapy, speech therapy, etc.) is stable for at least 90 days

Exclusion Criteria:

  • Severe scoliosis (curvature >40 degrees)
  • Bone-age greater than 11 years
  • 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Contacts
Contact: Katherine Barnes 617-355-5230 rettresearch@childrens.harvard.edu
Contact: Natalie Bruck 617-355-5230 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 Children's Hospital Boston
  More Information

Publications:
Responsible Party: Walter Kaufmann, Professor of Neurology, Harvard Medical School
ClinicalTrials.gov Identifier: NCT01777542     History of Changes
Other Study ID Numbers: IRB-P00005610
Study First Received: January 23, 2013
Last Updated: September 29, 2014
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
Syndrome
Disease
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Intellectual Disability
Mental Retardation, X-Linked
Nervous System Diseases
Neurobehavioral Manifestations
Neurodegenerative Diseases
Neurologic Manifestations
Pathologic Processes
Insulin
Insulin, Globin Zinc
Mitogens
Hypoglycemic Agents
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014