Methylmalonic AcidemiaNCT05506254
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
An observational study of Methylmalonic Acidemia, run by Alexion Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Methylmalonic Acidemia
- What is being tested?
- hLB-001
- How many people
- 4 participants
- Where
- 4 sitesUnited States
- Study dates
- 15 Jul 2022to 1 Jan 2037
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.
Who may be able to join
- Participant has previously participated in a LogicBio clinical trial and received hLB-001.
Who cannot join
- None
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Clinical Trial Site
Atlanta, Georgia, 30322
Clinical Trial Site
Nashville, Tennessee, 37232
Clinical Trial Site
Pittsburgh, Pennsylvania, 15224
Clinical Trial Site
Seattle, Washington, 98105
About this study
Official title: A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001
Read the detailed description
Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
- Conditions
- Methylmalonic Acidemia
- Being tested
- hLB-001
- Medical subject headings
- Methylmalonic acidemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hLB-001Drug
hLB-001 gene therapy
| Study group | Role | What they receive |
|---|---|---|
| Patients who have previously received hLB-001 | Not described Receives: Drug: hLB-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment emergent adverse events
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2022
- Study start15 Jul 2022
- Primary completion1 Jan 2037 (estimated)
- Study completion1 Jan 2037 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Methylmalonic AcidemiaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05506254
- Sponsor's protocol ID
- LB-001LT
- Status last verified
- Feb 2026
- First submitted
- 16 Aug 2022
- Last updated
- 5 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001
- ClinicalTrials.gov ID
- NCT05506254
- Acronym / sponsor ID
- LB-001LT
- Conditions
- Methylmalonic Acidemia
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Jul 2022 (actual)
- Primary completion
- 1 Jan 2037 (estimated)
- Study completion
- 1 Jan 2037 (estimated)
- First posted
- 18 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001
Detailed description
Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients who have previously received hLB-001 | – | Drug: hLB-001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.
Key inclusion criteria (1 listed)
- Participant has previously participated in a LogicBio clinical trial and received hLB-001.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hLB-001hLB-001 gene therapy | Drug | Patients who have previously received hLB-001 |
Primary outcome
Incidence of treatment emergent adverse events
Time frame: 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Methylmalonic Acidemia, run by Alexion Pharmaceuticals, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment emergent adverse events (time frame: 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2026.