Progress Now: Enrolling Clinical Trial Updates

Amyotrophic lateral sclerosis (ALS)

Phase 1 Clinical Trial: Enrolling

This trial, called LUMINA, is testing the investigational therapy AMX0114 in adults with ALS. The main goal is to evaluate safety and tolerability at different dosing levels.

Inclusion criteria include:

• Definite ALS or probable ALS diagnosis
• Age 18 years or older
• ALS symptoms began within the past 24 months

Overview

AMX0114 is an antisense oligonucleotide (ASO) designed to reduce levels of calpain-2, a protein that has been linked to the degeneration and death of neurons in people living with sporadic ALS.

This is a randomized, double-blind, placebo-controlled study, meaning some participants will receive AMX0114 and others will receive a placebo (an inactive substance). AMX0114 is given by intrathecal injection (an injection into the fluid around the spinal cord in the lower back).

Timeframe

About 4 months

Location

14 sites in the US and Canada

Learn more

Visit ClinicalTrials.gov and enter NCT06665165 in the “Other terms” search box.

Contact

Medical Director, Amylyx, 857-320-6200, clinicaltrials@amylyx.com

Duchenne muscular dystrophy (DMD)

Phase 3 Clinical Trial: Results

Regenxbio released positive topline results from the phase 3 portion of the AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy for DMD. In the trial, RGX-202 raised microdystrophin levels in most boys.

Overview

In DMD, a mutation in the DMD gene prevents the body from producing enough dystrophin protein, which is essential for muscle function. The lack of dystrophin protein leads to progressive muscle weakness. RGX-202 is designed to deliver a modified gene to muscle cells, leading to production of microdystrophin, a shortened but functional dystrophin protein.

Key points

• This is a multicenter, open-label study.
• Participants are boys with DMD, ages 1 year and older, who are ambulatory (able to walk).
• RGX-202 is administered via a one-time IV infusion (a tube into the vein).
• Regenxbio is planning to ask the US Food and Drug Administration (FDA) to grant accelerated approval to RGX-202.

Learn more

Visit regenxbio.com/therapeutic-programs/rgx-202.

Friedreich ataxia (FA)

Phase 3 Clinical Trial: Enrolling

This phase 3 study, called BRAVE, seeks to better understand FA and to evaluate omaveloxolone, an investigational therapy for children and teenagers with FA.

Inclusion criteria include:

• Between 2 and 15 years old
• Genetic diagnosis of FA

Overview

Omaveloxolone is approved by the US Food and Drug Administration (FDA) to treat FA in patients ages 16 and older. BRAVE is evaluating the safety of omaveloxolone and how well it works for the treatment of FA in people under age 16.

This is a two-part study. In Part 1, participants will be randomly assigned to receive omaveloxolone or a placebo (an inactive substance) for a year. The primary objective is to evaluate changes in upright stability (a measure including how well someone can stay balanced while standing and walking) after one year of treatment. Participants who complete Part 1 may be eligible to continue into Part 2 in which all participants will receive omaveloxolone for an additional two years. Omaveloxolone is administered orally (by mouth).

Timeframe

3 years

Location

34 global sites, including 7 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT06953583 in the “Other terms” search box.

Contact

Patient Navigator, 877-223-3576 ext. 57078, biogenBRAVE_patientnavigator @thermofisher.com

Myasthenia gravis (MG)

Phase 3 Clinical Trial: Enrolling

This study, called UPSTREAM MG, will evaluate whether treatment with the investigational drug telitacicept is safe and effective at improving muscle weakness and fatigue in adults with generalized myasthenia gravis (gMG).

Inclusion criteria include:

• Age 18 years or older
• Diagnosis of gMG with generalized muscle weakness
• Positive screening test for acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK) antibodies

Overview

In MG, the immune system attacks receptors for communication between nerves and muscles, leading to muscle weakness.

Telitacicept is designed to reduce the level of B cells, a type of immune cell that produces the antibodies responsible for causing AChR-positive and MuSK-positive gMG.

The study begins with a randomized, double-blind, placebo-controlled period comparing the effects of telitacicept with a placebo (an inactive substance). After the double-blinded period, participants may enter an open-label extension period during which all participants will receive telitacicept. The drug is administered via a weekly subcutaneous injection (a shot under the skin).

Timeframe

24-week double-blind period, followed by 48-week open-label extension period

Location

111 global locations, including 26 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT06456580 in the “Other terms” search box.

Contact

Jeremy Sokolove, 617-655-6580, study@vorbio.com

Phase 3 Clinical Trial: Enrolling

This is a study for adults with gMG who are currently receiving ravulizumab treatment. It will evaluate whether gradually lowering oral corticosteroids can safely help patients reduce or stop these medications while keeping their gMG symptoms stable.

Inclusion criteria include:

• Age 18 years or older
• Clinical diagnosis of gMG
• Receiving ravulizumab as part of standard medical care

Overview

Researchers will follow participants using a planned schedule to slowly reduce their corticosteroid dose and monitor the safety and effectiveness of this approach. At clinic visits, participants will complete questionnaires about their symptoms and experiences, and provide a small number of blood samples for study assessments.

Timeframe

6 months

Location

12 global sites, including 5 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT07221838 in the “Other terms” search box.

Contact

Alexion Pharmaceuticals, Inc., 855-752-2356, clinicaltrials@alexion.com

Myotonic dystrophy (DM)

Observational Study: Enrolling

Researchers at the University of Rochester are seeking adults with DM type 2 (DM2) for a natural history study called Longitudinal Endpoint Optimization Providing an Assessment of Relevant Drugs in Myotonic Dystrophy Type 2 (LEOPARD-DM2).

Inclusion criteria include:

• Age 18 years or older
• Diagnosed with DM2

Overview

LEOPARD-DM2 participants will complete an online survey every 6 months. MDA is funding this long-term, observational study. Findings from the study could help researchers better understand DM2 disease progression and identify meaningful outcome measures for future clinical trials.

Timeframe

2.5 years

Contact

Charlotte Engebrecht, study coordinator, 585-200-7136, charlotte_engebrecht@urmc.rochester.edu

Pompe disease

Phase 2 Clinical Trial: Enrolling

This is a study for adults with late-onset Pompe disease (LOPD) who are receiving standard-of-care enzyme replacement therapy (ERT). The study will evaluate the safety and effectiveness of the investigational drug S-606001 when used as an add-on to ERT.

Inclusion criteria include:

• Age 18 years or older
• Documented diagnosis of LOPD
• Receiving ERT for at least the past 24 months

Overview

In Pompe disease, glycogen builds up in muscle cells, leading to muscle damage. S-606001 is designed to block an enzyme involved in glycogen production. By reducing glycogen production, S-606001 may help limit this buildup and slow the decline of muscle strength and lung function in people receiving ERT.

This is a randomized, double-blind, placebo-controlled study, meaning participants will be randomly assigned to receive either S-606001 or a placebo (an inactive substance). The drug will be administered orally (by mouth).

Participants who complete the study may be eligible to enroll in an extension study, in which all participants will receive S-606001.

Timeframe

53 weeks

Location

28 global sites, including 9 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT07123155 in the “Other terms” search box.

Contact

Shionogi Clinical Trials Administrator Clinical Support Help Line, 800-849-9707, ShionogiClinTrials-admin@shionogi.co.jp

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Clinical Trial Terms to Know

Double-blind: Neither researchers nor participants know which participants are taking the drug or placebo.

Open-label: Participants know what treatment they are receiving.

Placebo-controlled: Some participants receive the treatment being tested, and some receive a placebo that looks like the real treatment but has no active ingredients.

Randomized: Participants are randomly assigned to groups taking the drug or placebo.