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Gene Therapy

hLB-001 for Methylmalonic Acidemia (SUNRISE Trial)

Phase 1 & 2

Waitlist Available

Research Sponsored by LogicBio Therapeutics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 52

Awards & highlights

SUNRISE Trial Summary

This trial is a study to see if a new treatment is safe and effective for children with a rare disease. The disease is called methylmalonyl-CoA mutase gene (MMUT) mutations and the new treatment is called hLB-001.

Eligible Conditions

Methylmalonic Acidemia

SUNRISE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants With Infusional Toxicities

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Secondary outcome measures

Change From Baseline in Serum Albumin-2A Level at Week 52

Change From Baseline in Serum Fibroblast Growth Factor 21 (FGF21) Level at Week 52

Change From Baseline in Serum Methylcitrate Level at Week 52

+2 more

SUNRISE Trial Design

5Treatment groups

Experimental Treatment

Group I: Dose Level 2 Part BExperimental Treatment1 Intervention

6 month to 2 year-olds

Group II: Dose Level 2 Part AExperimental Treatment1 Intervention

3 year-olds to 12 year-olds

Group III: Dose Level 1 Part CExperimental Treatment1 Intervention

6 month to 12 year-olds

Group IV: Dose Level 1 Part BExperimental Treatment1 Intervention

6 month to 2 year-olds

Group V: Dose Level 1 Part AExperimental Treatment1 Intervention

3 year-olds to 12 year-olds

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

AlexionIndustry Sponsor

246 Previous Clinical Trials

39,273 Total Patients Enrolled

Alexion Pharmaceuticals, Inc.Industry Sponsor

253 Previous Clinical Trials

41,178 Total Patients Enrolled

LogicBio Therapeutics, IncLead Sponsor

1 Previous Clinical Trials

4 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the eligibility requirements for participation in this study?

"To qualify for this research, potential participants must have an inborn error of amino acid metabolism and be aged between 6 months to 12 years. The trial is searching for a total of 8 patients."

Answered by AI

Are adults aged 45 or greater able to apply for enrollment in this investigation?

"Eligible participants for this medical trial should fall between 6 months and 12 years of age. There are 7 clinical trials specifically geared towards minors and 15 that cater to seniors over 65."

Answered by AI

Are there any remaining openings to participate in this trial?

"Affirmative. The information posted on clinicaltrials.gov attests to the active recruitment for this trial, which was initially uploaded on May 29th 2021 and lastly updated on August 16th 2022. 8 patients are being sought from 6 separate sites for enrolment in this study."

Answered by AI

How many participants are included in this scientific research project?

"This clinical trial, sponsored by LogicBio Therapeutics Inc., necessitates the recruitment of 8 participants that meet its enrolment criteria. The study will take place at Emory University in Atlanta and UPMC Children's Hospital in Pittsburgh."

Answered by AI

Are there any North American sites hosting this research endeavor?

"Patients can enroll in this research study at a multitude of sites; including Emory University, UPMC Children's Hospital of Pittsburgh and the renowned Children's Hospital Colorado. There are 6 other locations available as well."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04581785.