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A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Primary Purpose

Fragile X Syndrome

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NNZ-2566
Placebo
Sponsored by
Neuren Pharmaceuticals Limited
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome focused on measuring Fragile X disorder, Autism

Eligibility Criteria

12 Years - 45 Years (Child, Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  1. Fragile X Syndrome with a molecular genetic confirmation of the full FMR1 mutation.

    • Results from previously completed testing are acceptable with written documentation of the genetic results.
    • Results from PCR or Southern blot tests are acceptable
    • Results from cytogenetic testing are not acceptable but subject may be eligible if molecular genetic testing is redone.
    • A full mutation with mosaicism is allowed if:

      • Subject manifests full phenotypic profile of Fragile X syndrome
      • CGG Repeats >200 are detected
      • Southern blot prevails over PCR, if Southern blot shows >200 repeats and PCR results show <200 repeats.
    • The following results would not meet criteria:

      • Deletions
      • Point mutations
      • Mosaicism without detection of >200 CGG repeats or absence of full phenotypic profile in an individual with mosaicism
  2. Males, aged 12-45 years
  3. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening
  4. Subjects with a total score of 30 or greater on the Aberrant Behavior Checklist (ABC) at Screening.
  5. Current treatment with no more than 3 psychotropic medications. This includes medications used to treat problems with sleep onset and sleep continuity. Melatonin for difficulties with sleep onset is permissible and is excepted from the count of psychotropic medications. Similarly, anti-epileptic medications are permitted and are not denoted as "psychotropic medications" if they are used for treatment of seizures. Use of anti-epileptics for other indications such as the treatment of mood disorders counts towards the limit of permitted medications. Concurrent use of omega-3 fatty acids is also permissible and does not count towards the allowed number of concomitant psychotropic medications. A complete list of permitted concomitant psychotropic medications can be found in Appendix A.
  6. Concomitant medications for chronic medical conditions are permissible. Examples of chronic medical conditions include gastroesophageal reflux disease (GERD) and asthma. Every effort should be made to keep the doses and dosing regimens of these medications stable in the 4 weeks preceding Screening and during the period between Screening and the commencement of study medication.

    1. Permitted psychotropic concomitant medications (except for anti-epileptic medications-see below) must be stable, in terms of dose and dosing regimen, for at least 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
    2. Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication
  7. Behavioral treatments excluding psychotherapy (see exclusion criteria) must be stable for 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication

    a. For each enrollee, every effort should be made to maintain stable regimens of allowed concomitant medications and allowed behavioral therapies from the time of commencement of single-blind study medication until the last study assessment.

  8. Sufficient expressive language capabilities to complete the Expressive Language Sampling Task.
  9. Individuals with a history of seizures should have a stable pattern of seizure activity in the 3 months preceding Screening.

Exclusion Criteria:

  1. Treatment within the two weeks prior to Screening with monoamine (MAO) inhibitors, lithium, minocycline, acamprosate, racemic baclofen, investigational metabotropic glutamate receptor subtype 5 (mGluR5) medications, d-cycloserine, oxytocin, carbetocin, tricyclic antidepressants and bupropion.
  2. Patients planning to commence psychotherapy, including cognitive behavior therapy (CBT), during the period of the study or those who had begun psychotherapy, including CBT, within 6 weeks prior to Screening.
  3. History of, or current, cardiovascular, renal, hepatic, respiratory and/or gastrointestinal disease, which may interfere with the absorption, distribution, metabolism or excretion of the study medication, or which may interfere with the interpretation of the safety/tolerability or efficacy of the study medication.
  4. History of, or current cerebrovascular disease or clinically significant brain trauma.
  5. History of, or current clinically significant endocrine disorder, e.g. hypo or hyperthyroidism, or diabetes.
  6. History of, or current malignancy.
  7. Current major depressive disorder (patients have to be free of the most recent episode for 3 months prior to enrollment).
  8. History of a DSM-5-defined substance use disorder in the 3 months prior to Screening.
  9. Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as measured at Screening.
  10. QT/QTcF Exclusions (any of the following):

    • QTcF > 450 msec. Three ECGs should be obtained at the time of Screening, 5 minutes apart from each other, and the results should be averaged.
    • History of risk factors for torsade de pointes (e.g. heart failure, clinically significant hypokalemia or hypomagnesemia, or a family of long QT syndrome).
    • A serum potassium at screening <3.0 mmol/L.
    • QT/QTcF prolongation previously or currently controlled with medication, in which normal QT/QTcF intervals could or can only be achieved with medication
    • Current treatment with other medications that have demonstrated QT/QTc prolongation and have this risk described in the Warnings and Precautions section of their Prescribing Information
  11. Patients with significant hearing and/or visual impairments that may affect their ability to complete the test procedures.
  12. Current treatment with insulin
  13. Hgb A1C values outside of the normal reference range at Screening
  14. Current or past treatment with insulin like growth factor IGF-1
  15. Current or past treatment with growth hormone
  16. Enrollment in another clinical trial within the 30 days preceding Screening
  17. Previously randomized in this clinical trial
  18. Allergy to strawberry

Sites / Locations

  • UC Davis MIND Institute
  • Children's Hospital Colorado
  • Children's National Hospital
  • Emory University
  • Rush University Medical Center
  • Kennedy Krieger Institute
  • University of Massachusetts Medical School
  • University of Michigan
  • University of Nebraska
  • Mount Sinai School of Medicine
  • Cincinnati Children's Hospital Medical Center
  • Autism & Developmental Medicine Institute Geisinger Health System
  • Suburban Research Associates
  • Greenwood Genetic Center
  • Vanderbilt University Medical Center
  • Texas Children's Hospital
  • Seattle Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

NNZ-2566

Placebo (strawberry flavored solution)

Arm Description

Glycyl-L-2-Methylpropyl-L-Glutamic Acid

Strawberry flavored solution and Water

Outcomes

Primary Outcome Measures

Adverse events
Incidence of adverse events (AE), including Serious adverse events (SAE), will be evaluated between the two NNZ-2566 doses and placebo. Incidence of AEs from randomized dosing through to Day 56 post randomization. Incidence of SAEs from randomization through to Day 56 post randomization.

Secondary Outcome Measures

Physiological changes
Serum levels and changes of standard hematology, and chemistry parameters (including thyroid function) will be calculated from Baseline through to Day 56. Fundoscopy and tonsil size will be documented at Baseline, and Days 14, 28, 42 and 56. Flow cytometry will be used to assess the phosphorylation status of the enzymes Akt and extracellular signal-regulated kinase (ERK) in peripheral lymphocytes, on blood samples obtained on Days 14, 28, 42 and 56. Electrocardiogram (ECG) will be assessed at Screening, Days 14, 21, 28, 35, 42 and 56.
Behavior
Symptom severity according to the Fragile X Symptom Rating Scale, Clinician Domain Specific Top Three Concerns-VAS, Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scale (VABS), Child and Adolescent Symptom Inventory-Anxiety Scale (CASI-16), Children's Yale-Brown Obsessive Compulsive Scale (CYBOCS-PDD), Expressive Language Sampling and Clinical Global Impression of Severity (CGI-S).
Global and Functional outcome Measures
Global outcome as measured by the change in scores in the Clinical Global Impression - Severity and Improvement scales (CGI-S and -I) and the KiTap measure of cognition from baseline, during treatment, and post treatment. Global and Functional outcome as measured by changes in scores in the Fragile X Symptom Rating Scale, Clinician Domain Specific Top Three Concerns-VAS, Caregiver Top Three Concerns (related to the subject's Fragile X syndrome) as assessed via a Visual Analogue Scale (VAS), CASI-20, CYBOCS-PDD, Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scales (VABS) and Expressive Language Sampling will be assessed during treatment.

Full Information

First Posted
July 3, 2013
Last Updated
January 31, 2018
Sponsor
Neuren Pharmaceuticals Limited
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1. Study Identification

Unique Protocol Identification Number
NCT01894958
Brief Title
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
Official Title
A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Fixed-Dose Study of NNZ-2566 in Fragile X Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
January 2014 (undefined)
Primary Completion Date
September 2015 (Actual)
Study Completion Date
October 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neuren Pharmaceuticals Limited

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Fragile X Syndrome in adolescent and adult males.
Detailed Description
Fragile X Syndrome is a genetically determined neurological disorder in which affected individuals are intellectually handicapped to varying degrees and display a variety of associated psychiatric symptoms. Clinically, Fragile X Syndrome is characterized by intellectual handicap, hyperactivity and attentional problems, autism spectrum symptoms, emotional lability and epilepsy. The epilepsy seen in Fragile X Syndrome is most commonly present in childhood, but then gradually remits towards adulthood. Physical features such as prominent ears and jaw, and hyper-extensibility of joints are frequently present but are not diagnostic. Intellectual handicap is the most common feature defining the phenotype. Treatment for the disorder is symptomatic - focusing on the management of symptoms - and supportive, requiring a multidisciplinary approach. This study will investigate the safety and tolerability of treatment with oral administration of NNZ-2566 at 35 mg/kg or 70 mg/kg BID in adolescent or adult males with Fragile X Syndrome. The study also will also investigate measures of efficacy during treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X disorder, Autism

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
72 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NNZ-2566
Arm Type
Experimental
Arm Description
Glycyl-L-2-Methylpropyl-L-Glutamic Acid
Arm Title
Placebo (strawberry flavored solution)
Arm Type
Placebo Comparator
Arm Description
Strawberry flavored solution and Water
Intervention Type
Drug
Intervention Name(s)
NNZ-2566
Intervention Description
Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder (2g in 50mL vials or 3g in 30mL bottles) for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Strawberry flavored solution 0.5% v/v in Water for Injection
Intervention Description
Strawberry flavored solution
Primary Outcome Measure Information:
Title
Adverse events
Description
Incidence of adverse events (AE), including Serious adverse events (SAE), will be evaluated between the two NNZ-2566 doses and placebo. Incidence of AEs from randomized dosing through to Day 56 post randomization. Incidence of SAEs from randomization through to Day 56 post randomization.
Time Frame
Through to Day 56
Secondary Outcome Measure Information:
Title
Physiological changes
Description
Serum levels and changes of standard hematology, and chemistry parameters (including thyroid function) will be calculated from Baseline through to Day 56. Fundoscopy and tonsil size will be documented at Baseline, and Days 14, 28, 42 and 56. Flow cytometry will be used to assess the phosphorylation status of the enzymes Akt and extracellular signal-regulated kinase (ERK) in peripheral lymphocytes, on blood samples obtained on Days 14, 28, 42 and 56. Electrocardiogram (ECG) will be assessed at Screening, Days 14, 21, 28, 35, 42 and 56.
Time Frame
Baseline through to Day 56
Title
Behavior
Description
Symptom severity according to the Fragile X Symptom Rating Scale, Clinician Domain Specific Top Three Concerns-VAS, Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scale (VABS), Child and Adolescent Symptom Inventory-Anxiety Scale (CASI-16), Children's Yale-Brown Obsessive Compulsive Scale (CYBOCS-PDD), Expressive Language Sampling and Clinical Global Impression of Severity (CGI-S).
Time Frame
Baseline through to Day 56
Title
Global and Functional outcome Measures
Description
Global outcome as measured by the change in scores in the Clinical Global Impression - Severity and Improvement scales (CGI-S and -I) and the KiTap measure of cognition from baseline, during treatment, and post treatment. Global and Functional outcome as measured by changes in scores in the Fragile X Symptom Rating Scale, Clinician Domain Specific Top Three Concerns-VAS, Caregiver Top Three Concerns (related to the subject's Fragile X syndrome) as assessed via a Visual Analogue Scale (VAS), CASI-20, CYBOCS-PDD, Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scales (VABS) and Expressive Language Sampling will be assessed during treatment.
Time Frame
Baseline through to Day 56
Other Pre-specified Outcome Measures:
Title
Pharmacokinetics
Description
The following pharmacokinetic measures will be calculated from NNZ-2566 concentrations in whole blood: Cmax (Peak), Cmin (trough), C0-6 at steady state, and area under the curve (AUC).
Time Frame
During treatment
Title
Computerized eye-tracking
Description
Computer-based eye tracking assessments will be done on Days 14, 28 and 42.
Time Frame
Baseline through to Day 56

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Fragile X Syndrome with a molecular genetic confirmation of the full FMR1 mutation. Results from previously completed testing are acceptable with written documentation of the genetic results. Results from PCR or Southern blot tests are acceptable Results from cytogenetic testing are not acceptable but subject may be eligible if molecular genetic testing is redone. A full mutation with mosaicism is allowed if: Subject manifests full phenotypic profile of Fragile X syndrome CGG Repeats >200 are detected Southern blot prevails over PCR, if Southern blot shows >200 repeats and PCR results show <200 repeats. The following results would not meet criteria: Deletions Point mutations Mosaicism without detection of >200 CGG repeats or absence of full phenotypic profile in an individual with mosaicism Males, aged 12-45 years Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening Subjects with a total score of 30 or greater on the Aberrant Behavior Checklist (ABC) at Screening. Current treatment with no more than 3 psychotropic medications. This includes medications used to treat problems with sleep onset and sleep continuity. Melatonin for difficulties with sleep onset is permissible and is excepted from the count of psychotropic medications. Similarly, anti-epileptic medications are permitted and are not denoted as "psychotropic medications" if they are used for treatment of seizures. Use of anti-epileptics for other indications such as the treatment of mood disorders counts towards the limit of permitted medications. Concurrent use of omega-3 fatty acids is also permissible and does not count towards the allowed number of concomitant psychotropic medications. A complete list of permitted concomitant psychotropic medications can be found in Appendix A. Concomitant medications for chronic medical conditions are permissible. Examples of chronic medical conditions include gastroesophageal reflux disease (GERD) and asthma. Every effort should be made to keep the doses and dosing regimens of these medications stable in the 4 weeks preceding Screening and during the period between Screening and the commencement of study medication. Permitted psychotropic concomitant medications (except for anti-epileptic medications-see below) must be stable, in terms of dose and dosing regimen, for at least 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication. Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication Behavioral treatments excluding psychotherapy (see exclusion criteria) must be stable for 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication a. For each enrollee, every effort should be made to maintain stable regimens of allowed concomitant medications and allowed behavioral therapies from the time of commencement of single-blind study medication until the last study assessment. Sufficient expressive language capabilities to complete the Expressive Language Sampling Task. Individuals with a history of seizures should have a stable pattern of seizure activity in the 3 months preceding Screening. Exclusion Criteria: Treatment within the two weeks prior to Screening with monoamine (MAO) inhibitors, lithium, minocycline, acamprosate, racemic baclofen, investigational metabotropic glutamate receptor subtype 5 (mGluR5) medications, d-cycloserine, oxytocin, carbetocin, tricyclic antidepressants and bupropion. Patients planning to commence psychotherapy, including cognitive behavior therapy (CBT), during the period of the study or those who had begun psychotherapy, including CBT, within 6 weeks prior to Screening. History of, or current, cardiovascular, renal, hepatic, respiratory and/or gastrointestinal disease, which may interfere with the absorption, distribution, metabolism or excretion of the study medication, or which may interfere with the interpretation of the safety/tolerability or efficacy of the study medication. History of, or current cerebrovascular disease or clinically significant brain trauma. History of, or current clinically significant endocrine disorder, e.g. hypo or hyperthyroidism, or diabetes. History of, or current malignancy. Current major depressive disorder (patients have to be free of the most recent episode for 3 months prior to enrollment). History of a DSM-5-defined substance use disorder in the 3 months prior to Screening. Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as measured at Screening. QT/QTcF Exclusions (any of the following): QTcF > 450 msec. Three ECGs should be obtained at the time of Screening, 5 minutes apart from each other, and the results should be averaged. History of risk factors for torsade de pointes (e.g. heart failure, clinically significant hypokalemia or hypomagnesemia, or a family of long QT syndrome). A serum potassium at screening <3.0 mmol/L. QT/QTcF prolongation previously or currently controlled with medication, in which normal QT/QTcF intervals could or can only be achieved with medication Current treatment with other medications that have demonstrated QT/QTc prolongation and have this risk described in the Warnings and Precautions section of their Prescribing Information Patients with significant hearing and/or visual impairments that may affect their ability to complete the test procedures. Current treatment with insulin Hgb A1C values outside of the normal reference range at Screening Current or past treatment with insulin like growth factor IGF-1 Current or past treatment with growth hormone Enrollment in another clinical trial within the 30 days preceding Screening Previously randomized in this clinical trial Allergy to strawberry
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elizabeth M Berry-Kravis, MD
Organizational Affiliation
Rush University Medical Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Joseph Cubells, MD, PhD
Organizational Affiliation
Emory University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Alexander Kolevzon, MD
Organizational Affiliation
Icahn School of Medicine at Mount Sinai
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Nicole Tartaglia, MD
Organizational Affiliation
Children's Hospital Colorado
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jean Frazier, MD
Organizational Affiliation
University of Massachusetts, Worcester
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Shivkumar Hatti, MD
Organizational Affiliation
Suburban Research Associates
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Craig Erickson, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Thomas Challman, MD
Organizational Affiliation
Autism & Developmental Medicine Institute Geisinger Health System
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Kevin Sanders, MD
Organizational Affiliation
Vanderbilt University Medical Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Diane Treadwell-Deering, MD
Organizational Affiliation
Baylor College of Medicine
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jeffrey Innis, MD
Organizational Affiliation
University of Michigan
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Howard Needleman, MD
Organizational Affiliation
University of Nebraska
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Steve Skinner, MD
Organizational Affiliation
Greenwood Genetic Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Bryan King, MD
Organizational Affiliation
Seattle Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Randi Hagerman, MD
Organizational Affiliation
UC Davis MIND Institute
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Robert Findling, MD
Organizational Affiliation
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Official's Role
Principal Investigator
Facility Information:
Facility Name
UC Davis MIND Institute
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Children's Hospital Colorado
City
Denver
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Children's National Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
University of Massachusetts Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
University of Nebraska
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68588
Country
United States
Facility Name
Mount Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Autism & Developmental Medicine Institute Geisinger Health System
City
Lewisburg
State/Province
Pennsylvania
ZIP/Postal Code
17837
Country
United States
Facility Name
Suburban Research Associates
City
Media
State/Province
Pennsylvania
ZIP/Postal Code
19063
Country
United States
Facility Name
Greenwood Genetic Center
City
Greenwood
State/Province
South Carolina
ZIP/Postal Code
29646
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37235
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
32660869
Citation
Berry-Kravis E, Horrigan JP, Tartaglia N, Hagerman R, Kolevzon A, Erickson CA, Hatti S, Snape M, Yaroshinsky A, Stoms G; FXS-001 Investigators; Glass L, Jones NE. A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome. Pediatr Neurol. 2020 Sep;110:30-41. doi: 10.1016/j.pediatrneurol.2020.04.019. Epub 2020 May 23.
Results Reference
derived
Links:
URL
http://ghr.nlm.nih.gov/
Description
Genetics Home Reference
URL
http://ghr.nlm.nih.gov/condition/fragile-x-syndrome
Description
Fragile X Syndrome
URL
http://ghr.nlm.nih.gov/condition/mecp2-duplication-syndrome
Description
MECP2 duplication syndrome
URL
http://ghr.nlm.nih.gov/condition/ppm-x-syndrome
Description
PPM-X syndrome
URL
http://ghr.nlm.nih.gov/condition/renpenning-syndrome
Description
Renpenning syndrome
URL
http://ghr.nlm.nih.gov/condition/tetrasomy-18p
Description
tetrasomy 18p
URL
http://www.nlm.nih.gov/medlineplus/
Description
MedlinePlus
URL
http://www.nlm.nih.gov/medlineplus/drinkingwater.html
Description
Drinking Water
URL
http://www.nlm.nih.gov/medlineplus/fragilexsyndrome.html
Description
Fragile X Syndrome
URL
http://www.clinicaltrials.gov/ct2/info/fdalinks
Description
U.S. FDA Resources

Learn more about this trial

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

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