ClinicalTrials.gov
ClinicalTrials.gov Menu

Clinical Assessment Study in Crigler-Najjar Syndrome (LUSTRO)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03078881
Recruitment Status : Recruiting
First Posted : March 14, 2017
Last Update Posted : August 15, 2018
Sponsor:
Information provided by (Responsible Party):
Audentes Therapeutics

Brief Summary:
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.

Condition or disease
Crigler-Najjar Syndrome

Study Type : Observational
Estimated Enrollment : 18 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
Actual Study Start Date : March 10, 2017
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019





Primary Outcome Measures :
  1. Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome [ Time Frame: Up to 2 years ]
  2. Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period [ Time Frame: Up to 2 years ]
  3. Assess phototherapy usage over the course of the study [ Time Frame: Up to 2 years ]

Secondary Outcome Measures :
  1. Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL [ Time Frame: Up to 2 years ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.
Criteria

Key Inclusion Criteria:

  • Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene
  • Subject is aged equal or greater than 1 year of age
  • Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)

Exclusion Criteria:

  • Subject is currently participating in an interventional study or has received gene or cell therapy
  • Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study
  • Subject has significant cholestatic disease, in the opinion of the investigator
  • Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening
  • Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator
  • Subject has a history of, or currently has, a clinically important condition other than Crigler-Najjar syndrome, in the opinion of the investigator

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03078881


Contacts
Contact: Kim Trant Director of Patient Advocacy +1 (415) 805-1049 trials@audentestx.com

Locations
United States, New York
Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
United States, Pennsylvania
Clinic for Special Children Recruiting
Strasburg, Pennsylvania, United States, 17579
Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel, 9103102
United Kingdom
King's College Hospital Recruiting
London, England, United Kingdom, SE5 9RS
Sponsors and Collaborators
Audentes Therapeutics
Investigators
Study Director: Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM Audentes Therapeutics

Responsible Party: Audentes Therapeutics
ClinicalTrials.gov Identifier: NCT03078881     History of Changes
Other Study ID Numbers: AT342-01
First Posted: March 14, 2017    Key Record Dates
Last Update Posted: August 15, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Cardiomyopathies
Crigler-Najjar Syndrome
Disease
Pathologic Processes
Heart Diseases
Cardiovascular Diseases
Hyperbilirubinemia, Hereditary
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases