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Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia (SUNRISE)

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ClinicalTrials.gov Identifier: NCT04581785
Recruitment Status : Recruiting
First Posted : October 9, 2020
Last Update Posted : August 25, 2021
Sponsor:
Information provided by (Responsible Party):
LogicBio Therapeutics, Inc.

Study Description
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Brief Summary:

The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non-disruptively integrate the human methylmalonyl-CoA mutase gene at the albumin locus.

The trial is expected to enroll pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years.


Condition or disease Intervention/treatment Phase
Methylmalonic Acidemia Biological: hLB-001 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
Actual Study Start Date : May 29, 2021
Estimated Primary Completion Date : June 30, 2023
Estimated Study Completion Date : June 30, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose Level 1 Part A
3 year-olds to12 year-olds
 Biological: hLB-001
hLB-001 via IV infusion
Experimental: Dose Level 1 Part B
6 month to 2 year-olds
 Biological: hLB-001
hLB-001 via IV infusion
Experimental: Dose Level 2 Part A
3 year-olds to12 year-olds
 Biological: hLB-001
hLB-001 via IV infusion
Experimental: Dose Level 2 Part B
6 month to 2 year-olds
 Biological: hLB-001
hLB-001 via IV infusion


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (AEs) [ Time Frame: 52 weeks ]
  2. Incidence of infusional toxicities [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Change in serum methylmalonic acid and methylcitrate [ Time Frame: 52 weeks ]
  2. Change in serum fibroblast growth factor 21 (FGF21) level [ Time Frame: 52 weeks ]
  3. Change in propionate oxidation rate [ Time Frame: 52 weeks ]
  4. Change in serum albumin-2A level [ Time Frame: 52 weeks ]
  5. Overall Survival: deaths of patients occurring during the study period will be collected [ Time Frame: 52 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   6 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • At the time of dosing, patient must be 6 months to 12 years of age

  • Males and females with diagnosis of severe MMA meeting all the following;

    1. Isolated MMA with genetically confirmed, pathogenic mutations in the MMUT gene
    2. Screening serum/plasma methylmalonic acid level of >100 µmol/L
    3. One or more of the following considered by the PI to be MMA-related: (i) An unscheduled ER visit, hospitalization or requirement for sick day diet in the year prior to screening visit (ii) Developmental delay, movement disorder, optic neuropathy or feeding disorder with tube feeding requirement
    4. Medically stable for the 2 months prior to the start of screening

Exclusion Criteria:

  • Patients with organic acidemias other than isolated MMA, or with any other causes of hyperammonemia
  • Having received MMA-targeted gene therapy or nucleic acid therapy
  • Patients on insulin or high dose hydroxocobalamin (> 1 mg/day OHB12 parenteral)
  • Kidney or liver transplant, including hepatocyte cell therapy
  • Estimated glomerular filtration rate (eGFR) of < 60 mL/min/1.73 m2 based on age appropriate equations, or ongoing dialysis for renal disease
  • Patient tests positive for anti-rAAV-LK03-neutralizing antibodies
Contacts and Locations
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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): NCT04581785


Contacts
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Contact: Clinical Trials (617) 758-8059 clinicaltrials@logicbio.com

Locations
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United States, California
Stanford Children's Health - Lucile Packard Children's Hospital Not yet recruiting
Palo Alto, California, United States, 94304
Contact: Hershonna S Robinson    650-497-0454    hsrobin@stanford.edu   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Andi Martinsen    720-777-4691    Andrea.Martinsen@childrenscolorado.org   
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Eleanor Botha    404-778-8517    Egeller@emory.edu   
United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Mark Tumblin    412-692-5969    mark.tumblin2@chp.edu   
United States, Tennessee
Vanderbilt Children's Hospital Recruiting
Nashville, Tennessee, United States, 37232
Contact: Diana Coman    615-875-9700    Diana.L.Coman@vumc.org   
Contact: Thomas Morgan    615-322-7601    thomas.m.morgan@vumc.org   
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Hayden Vreugdenhil    206-884-1264    Hayden.Vreugdenhil@seattlechildrens.org   
Saudi Arabia
King Abdullah Specialist Children Hospital Not yet recruiting
Riyadh, Saudi Arabia
Contact: Majid Alfadhel, MD    +966-011-801-1111 ext 53560    Fadhelma@ngha.med.sa   
Sponsors and Collaborators
LogicBio Therapeutics, Inc.
Investigators
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Study Director: Daniel Gruskin, MD LogicBio Therapeutics
More Information
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Additional Information:

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Responsible Party: LogicBio Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04581785    
Other Study ID Numbers: LB001-001
First Posted: October 9, 2020    Key Record Dates
Last Update Posted: August 25, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by LogicBio Therapeutics, Inc.:
inborn errors of metabolism
mut0
mut-
 mut deficiency
organic acidemia
SUNRISE
Additional relevant MeSH terms:
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Amino Acid Metabolism, Inborn Errors
Acidosis
Acid-Base Imbalance
 Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn