Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency

Description

A Phase 1/2, open-label dose-finding safety study of single ascending doses of DTX301 in adults with late-onset OTC Deficiency

Study Start Date

January, 01 2017

Estimated Completion Date

November 2018

Interventions

  • Genetic: scAAV8OTC

Study ID

Dimension Therapeutics -- 301OTC01

Status

Recruiting

Trial ID

NCT02991144

Study Type

Interventional

Trial Phase

Phase 1/Phase 2

Enrollment Quota

9

Sponsor

Dimension Therapeutics

Inclusion Criteria

    1. Males and females ?18 years of age with documented diagnosis of late onset (defined as first manifestation of signs and symptoms at ?1 month of age) OTC deficiency, confirmed via enzymatic, biochemical, or molecular testing 2. Documented history of ?1 symptomatic hyperammonemia event with ammonia ?100 µmol/L. 3. Subject's OTC deficiency is stable as evidenced by either a) no clinical symptoms of hyperammonemia OR b) an ammonia level <100 µmol/L within the 4 week period preceding the Screening visit. 4. On stable dose of ammonia scavenger therapy for ?4 weeks. 5. Males and all females of childbearing potential must be willing to use effective contraception at the time of administration of gene transfer and for the 52 weeks following administration of DTX301

Exclusion Criteria

    1. Screening or Baseline (Day 0) ammonia level ?100 µmol/L or signs and symptoms indicative of hyperammonemia during the 4-week period preceding Day 0 2. Liver transplant, including hepatocyte cell therapy/transplant. 3. History of liver disease 4. Serum creatinine >2.0 mg/dL. 5. Participation in another investigational medicine study (including another gene transfer trial) within 3 months of Screening 6. History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer treated with watchful waiting or surgically removed non-melanoma skin cancer.

Gender

All

Ages

18 Years and older

Accepts Healthy Volunteers

No

Study Locations and Contact Information (24)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles WenHann Tan MD 617-355-6000 wen-hann.tan@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles WenHann Tan MD 617-355-6000 wen-hann.tan@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles WenHann Tan MD 617-355-6000 wen-hann.tan@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles WenHann Tan MD 617-355-6000 wen-hann.tan@childrens.harvard.edu
University Hospital Cleveland Medical CenterCase Western Reserve University - Cleveland, Ohio 544.0 miles Shawn E McCandless MD 216-844-3936 shawn.mccandless@case.edu
University Hospital Cleveland Medical CenterCase Western Reserve University - Cleveland, Ohio 544.0 miles Shawn E McCandless MD 216-844-3936 shawn.mccandless@case.edu
University Hospital Cleveland Medical CenterCase Western Reserve University - Cleveland, Ohio 544.0 miles Shawn E McCandless MD 216-844-3936 shawn.mccandless@case.edu
University Hospital Cleveland Medical CenterCase Western Reserve University - Cleveland, Ohio 544.0 miles Shawn E McCandless MD 216-844-3936 shawn.mccandless@case.edu
The Childrens Hospital Colorado - Aurora, Colorado 1,759.8 miles Janet A Thomas MD 303-724-2334 janet.thomas@childrenscolorado.org
The Childrens Hospital Colorado - Aurora, Colorado 1,759.8 miles Janet A Thomas MD 303-724-2334 janet.thomas@childrenscolorado.org
The Childrens Hospital Colorado - Aurora, Colorado 1,759.8 miles Janet A Thomas MD 303-724-2334 janet.thomas@childrenscolorado.org
The Childrens Hospital Colorado - Aurora, Colorado 1,759.8 miles Janet A Thomas MD 303-724-2334 janet.thomas@childrenscolorado.org
Oregon Health and Science University - Portland, Oregon 2,538.3 miles Cary O Harding MD 503-494-7608 hardingc@ohsu.edu
Oregon Health and Science University - Portland, Oregon 2,538.3 miles Cary O Harding MD 503-494-7608 hardingc@ohsu.edu
Oregon Health and Science University - Portland, Oregon 2,538.3 miles Cary O Harding MD 503-494-7608 hardingc@ohsu.edu
Oregon Health and Science University - Portland, Oregon 2,538.3 miles Cary O Harding MD 503-494-7608 hardingc@ohsu.edu
Ronald Reagan University of Califonria Los Angeles Medical Center - Los Angeles, California 2,605.2 miles Gerald Lipshutz MD 310-267-9592 glipshutz@mednet.ucla.edu
Ronald Reagan University of Califonria Los Angeles Medical Center - Los Angeles, California 2,605.2 miles Gerald Lipshutz MD 310-267-9592 glipshutz@mednet.ucla.edu
Ronald Reagan University of Califonria Los Angeles Medical Center - Los Angeles, California 2,605.2 miles Gerald Lipshutz MD 310-267-9592 glipshutz@mednet.ucla.edu
Ronald Reagan University of Califonria Los Angeles Medical Center - Los Angeles, California 2,605.2 miles Gerald Lipshutz MD 310-267-9592 glipshutz@mednet.ucla.edu
University of California San Francisco - San Francisco, California 2,696.4 miles Renata Gallagher MD PhD 415-476-3572 renata.gallagher@ucsf.edu
University of California San Francisco - San Francisco, California 2,696.4 miles Renata Gallagher MD PhD 415-476-3572 renata.gallagher@ucsf.edu
University of California San Francisco - San Francisco, California 2,696.4 miles Renata Gallagher MD PhD 415-476-3572 renata.gallagher@ucsf.edu
University of California San Francisco - San Francisco, California 2,696.4 miles Renata Gallagher MD PhD 415-476-3572 renata.gallagher@ucsf.edu

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