Trial Profile

A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations

Status: Discontinued

Phase of Trial: Phase I/II

Latest Information Update: 26 Feb 2024

Price :

$35 ^*

Note:

Adis is an information provider.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

Drugs LB 001 (Primary)
Indications Methylmalonic acidaemia
Focus Adverse reactions; First in man
Acronyms SUNRISE
Sponsors Alexion AstraZeneca Rare Disease; LogicBio Therapeutics

Most Recent Events

25 Apr 2023 Status changed from active, no longer recruiting to discontinued.
24 Feb 2023 Status changed from recruiting to active, no longer recruiting.
16 Aug 2022 Planned End Date changed from 30 Jun 2023 to 30 Dec 2023.

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com