DNL343 for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Emory University, Atlanta, GA
Amyotrophic Lateral Sclerosis+2 More
DNL343 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis

See full description

Eligible Conditions

  • Amyotrophic Lateral Sclerosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Amyotrophic Lateral Sclerosis

Study Objectives

This trial is evaluating whether DNL343 will improve 1 primary outcome and 5 secondary outcomes in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of 28 Days.

19 months
Cerebrospinal fluid-to-plasma concentration ratio of DNL343 following multiple oral doses
PK parameter: Area under the concentration-time curve from time zero to 24 hours (AUC24) of DNL343 in plasma
PK parameter: Maximum concentration (Cmax) of DNL343 in plasma
PK parameter: Time to reach maximum concentration (tmax) of DNL343 in plasma
PK parameter: Trough concentration (Ctrough) of DNL343 in plasma
28 Days
Incidence of treatment-emergent adverse events (TEAEs) throughout the double-blind period

Trial Safety

Safety Progress

1 of 3

Other trials for Amyotrophic Lateral Sclerosis

Trial Design

3 Treatment Groups

DNL343 (High Dose)
1 of 3
DNL343 (Low Dose)
1 of 3
Placebo
1 of 3
Experimental Treatment
Non-Treatment Group

This trial requires 30 total participants across 3 different treatment groups

This trial involves 3 different treatments. DNL343 is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

DNL343 (High Dose)
Drug
DNL343 (Low Dose)
Drug
Placebo
Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DNL343
2020
Completed Phase 1
~130

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 19 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 19 months for reporting.

Closest Location

Emory University - Atlanta, GA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
are required Applicants must take stable doses of approved ALS treatments (riluzole and/or edaravone) for at least 2 months prior to the screening process. show original
without difficulty and be able to provide a fasting blood specimen show original
Diagnosis of sporadic or familial ALS
ALS symptoms developed less than three years ago. show original
If your vital capacity is greater than 50% at screening, it is likely that you will be able to continue participating in the study. show original
Women who do not have children must be surgically sterilized or be postmenopausal. show original
Men, and sex partner if a woman of childbearing potential, must use highly effective contraception
Key

Patient Q&A Section

What causes lateral sclerosis?

"Lateral sclerosis can be idiopathic, but may also be caused by multiple sclerosis (MS) in a small-to-moderate percentage of cases, neuromyelitis optica (NMO), adrenoleukodystrophy, and HIV infection. It is likely you will not feel, or may not feel, any symptoms of the disorder.\n" - Anonymous Online Contributor

Unverified Answer

Can lateral sclerosis be cured?

"Patients with a lesion at the level of the cervicothoracic spine can expect a cure in the long term. More research is needed in order to identify the parameters which predict whether or not a cure is possible." - Anonymous Online Contributor

Unverified Answer

What is lateral sclerosis?

"We propose to redefine lateral sclerosis as a syndrome consisting in a progressive degeneration of spinal cord, peripheral nerves and muscles that mainly reflects a profound impairment of motor units. The diagnosis of degenerative myositis should be considered only in case of an extensive, chronic bilateral or ascending atrophy." - Anonymous Online Contributor

Unverified Answer

How many people get lateral sclerosis a year in the United States?

"Only about 10% of people with MS reported having a family member with MS, but close and intimate relations with a family member with MS had an elevated risk for MS (adjusted odds ratio [OR]: 1.74; 95% confidence interval [CI], 1.29-1.96; p<0.001). A significant number of persons with MS (5.2%; 95% CI: 3.4-7.4%) reported having a family member who died secondary to MS or of a non-MS cause." - Anonymous Online Contributor

Unverified Answer

What are the signs of lateral sclerosis?

"The signs of lateral sclerosis include weakness, numbness and changes in speech, posture, and gait that are progressive. Those with symptoms typically have a history of fatigue and progressive loss of strength. The signs of lateral sclerosis are not specific and a careful history of medical problems, lifestyle and work history is necessary for diagnosis. Magnetic resonance imaging (MRI) is the imaging technique of choice for the detection of lateral sclerosis and should be performed if an initial diagnosis of aseptic meningitis is considered. Results from a recent paper of this study have confirmed that MRI may be the imaging technique of choice for the diagnosis of lateral sclerosis." - Anonymous Online Contributor

Unverified Answer

What are common treatments for lateral sclerosis?

"Lateral sclerosis is a progressive disorder without a treatment that can delay or stop the course of the disease, limiting function, especially in patients with mild deficits. There are four major treatments strategies: pharmacological interventions, physical therapies, braces or orthotics, and surgery. Patients with this illness may not tolerate pharmacological interventions. The mainstay for all patients is physical therapy that facilitates activities of daily living, walking, and ambulation to promote independent living." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of dnl343?

"Treatment with dnl343 is well-tolerated by healthy volunteers and patients with multiple sclerosis (MS). Dnl343 does not significantly reduce the production of the neurotoxic cytokine TNF-α. The low rate of side effects observed in Phase 1 studies suggests that dnl343 is safe for administration to patients with the chronic inflammatory disease MS." - Anonymous Online Contributor

Unverified Answer

How serious can lateral sclerosis be?

"Contrary to previous suggestions, the natural history of ALS has shifted significantly; it has become more prevalent, it can now affect early adulthood, it is now less likely to present with pure upper motor neuron signs, and it is rare in children and young adults. In contrast with the old-time notion that ALS was mainly a disease of older adults and was often fatal, now, more than half of all cases (51%) of ALS, or about half of adult cases, are mild. We should now understand that we can't automatically assign cases to an ALS category or a ALS subtype based on age or ALS symptomatology." - Anonymous Online Contributor

Unverified Answer

Does lateral sclerosis run in families?

"Significant clustering of lateral sclerosis among patients of the same sibship supports the theory that it is heritable, with autosomal dominant inheritance of the gene(s), possibly a modifier gene." - Anonymous Online Contributor

Unverified Answer

Is dnl343 typically used in combination with any other treatments?

"The study shows that Duloriquin A343 is a promising treatment candidate in MS therapy. However, further clinical trials are warranted to determine how to optimally utilize the mechanism of action of duloriquin A343." - Anonymous Online Contributor

Unverified Answer

Does dnl343 improve quality of life for those with lateral sclerosis?

"In a recent study, findings of this large multicenter, randomized, placebo-controlled study show that dnl343 has no significant clinical impact within the overall population of individuals with LSS. However, a subgroup of patients (those with lower pain scores and more favorable disease courses) appears to have significantly greater improvement with dnl343 injection at four and sixmonths post-treatment. These data suggest that future research should be directed at identifying those patients who may benefit most from this treatment option." - Anonymous Online Contributor

Unverified Answer

What is the latest research for lateral sclerosis?

"Lateral sclerosis is the second most common neurodegenerative disease among Americans, after Alzheimer's disease. The annual incidence is 9-40 cases per 100,000 person-years. Because it is now hard to imagine a new case from the current incidence, it is not possible to say when the latest research was carried out for lateral sclerosis or when the most recent report was made. What we can do, however, is to summarize the recent findings presented in this survey paper. The main finding is the identification of a genetic locus for familial lateral sclerosis." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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