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Trial record 1 of 1 for:    NCT02704273
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A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02704273
Recruitment Status : Completed
First Posted : March 10, 2016
Last Update Posted : January 21, 2020
Sponsor:
Information provided by (Responsible Party):
Audentes Therapeutics

Tracking Information
First Submitted Date March 1, 2016
First Posted Date March 10, 2016
Last Update Posted Date January 21, 2020
Study Start Date July 2016
Actual Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 4, 2016)
  • Characterize the disease course and natural history of XLMTM using respiratory assessment of strength as measured by PImax [ Time Frame: 3 months to 2 years ]
  • Characterize the disease course and natural history of XLMTM using neuromuscular assessments, as measured by CHOP INTEND/MFM-20 [ Time Frame: 3 months to 2 years ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: March 4, 2016)
Characterize quality of life as measured by PedsQL [ Time Frame: 3 months to 2 years ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
Official Title INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
Brief Summary This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population XLMTM subjects aged 3 years and younger.
Condition X-linked Myotubular Myopathy
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: January 16, 2020)
34
Original Estimated Enrollment
 (submitted: March 4, 2016)
16
Actual Study Completion Date September 2019
Actual Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Subject has a diagnosis of XLMTM resulting from a confirmed mutation in the MTM1 gene
  • Subject is male
  • Subject is aged less than 4 years
  • Subject requires some mechanical ventilatory support (eg, ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP] during sleeping hours)
  • Access to subject's medical records
  • Signed informed consent by the parent(s) or legally authorized representative(s) (LAR) (when applicable)
  • Subject and parent(s) or LAR are willing and able to comply with study visits and study procedures

Exclusion Criteria:

  • Subject is participating in an interventional study designed to treat XLMTM
  • Subject born <35 weeks gestation who is still not to term as per corrected age
  • Subject has a clinically important condition, or life-threatening disease other than XLMTM, in the opinion of the investigator
  • Subject has received pyridostigmine or any medication to treat XLMTM within 30 days of enrollment
Sex/Gender
Sexes Eligible for Study: Male
Ages up to 3 Years   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   France,   Germany,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02704273
Other Study ID Numbers ATX-MTM-009 INCEPTUS
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Audentes Therapeutics
Study Sponsor Audentes Therapeutics
Collaborators Not Provided
Investigators
Study Director: Salvador Rico, MD, PhD Audentes Therapeutics
PRS Account Audentes Therapeutics
Verification Date July 2019