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

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: NCT04581785
Recruitment Status : Recruiting
First Posted : October 9, 2020
Last Update Posted : December 8, 2022
Information provided by (Responsible Party):
LogicBio Therapeutics, Inc.

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

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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 : December 30, 2023
Estimated Study Completion Date : December 30, 2023

Arm Intervention/treatment
Experimental: Dose Level 1 Part A
3 year-olds to 12 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 1 Part C
6 month to 12 year-olds
Biological: hLB-001
hLB-001 via IV infusion

Experimental: Dose Level 2 Part A
3 year-olds to 12 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

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 ]

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

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

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

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

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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: Elizabeth A McCracken    412-692-5662    elizabeth.mccracken@chp.edu   
United States, Tennessee
Vanderbilt Children's Hospital Recruiting
Nashville, Tennessee, United States, 37232
Contact: Steven Klintworth    615-875-7596    Steven.klintworth@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   
Sponsors and Collaborators
LogicBio Therapeutics, Inc.
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: December 8, 2022
Last Verified: December 2022
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
mut deficiency
organic acidemia
Additional relevant MeSH terms:
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Amino Acid Metabolism, Inborn Errors
Acid-Base Imbalance
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn