WVE-003 for Huntington Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Centre For Movement Disorders, Toronto, Canada
Huntington Disease
WVE-003 - Drug
Eligibility
18 - 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug called WVE-003 can slow the progression of Huntington's disease.

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Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether WVE-003 will improve 1 primary outcome and 3 secondary outcomes in patients with Huntington Disease. Measurement will happen over the course of Day 1 through end of study (minimum of 36 weeks).

Week 36
Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t)
Maximum concentration (Cmax) of WVE-003 in plasma
Safety: Proportion of patients with adverse events (AEs)
Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

4 Treatment Groups

WVE-003 (Dose C) or placebo
1 of 4
WVE-003 (Dose A) or placebo
1 of 4
WVE-003 (Dose B) or placebo
1 of 4
WVE-003 (Dose D) or placebo
1 of 4
Experimental Treatment

This trial requires 36 total participants across 4 different treatment groups

This trial involves 4 different treatments. WVE-003 is the primary treatment being studied. Participants will be divided into 4 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

WVE-003 (Dose C) or placebo
Drug
WVE-003 (Dose A) or placebo
Drug
WVE-003 (Dose B) or placebo
Drug
WVE-003 (Dose D) or placebo
Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 1 through end of study (minimum of 36 weeks)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly day 1 through end of study (minimum of 36 weeks) for reporting.

Closest Location

Centre For Movement Disorders - Toronto, Canada

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion. show original
Ambulatory, male or female patients aged ≥25 to ≤60 years
Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
UHDRS Total Functional Capacity Scores ≥9 and ≤13

Patient Q&A Section

What are common treatments for huntington disease?

"It is important for patients to have an informed decision about their treatment choices. Many of these treatments can be provided at home, while others require inpatient treatment. Many of the pharmacological options are very effective and can reduce the symptoms of HD by up to 90% within several weeks, often resulting in a dramatic reduction in care burden for families. Other options commonly used include transcranial direct-current stimulation, subcutaneous electrical stimulation, subcutaneous insulin infusion, rasagiline, memantine, tetrabenazine, selegiline, clonazepam, memantine, memantine, and fluorethyl propionate." - Anonymous Online Contributor

Unverified Answer

What causes huntington disease?

"This report adds to the existing neuroimaging literature, and provides additional neuroimaging evidence that may suggest specific hypotheses. These data support the involvement of bilateral cingulate cortices in Huntington disease, and suggest that the left hippocampus is at risk in early Huntington disease." - Anonymous Online Contributor

Unverified Answer

Can huntington disease be cured?

"Huntington disease cannot be cured. There is no cure for disease like Huntington disease. Patients will live with this disease for their whole life. There is no cure but there are treatments for managing the disease to reduce the severity of symptoms in patients to the greatest extent possible." - Anonymous Online Contributor

Unverified Answer

What are the signs of huntington disease?

"There are no specific, single signs, but rather a spectrum of signs involving motor and mental functions. It is the pattern of these signs that lead to the diagnosis. Most of the time, signs occur in association with other signs, some of which occur after the onset of HD." - Anonymous Online Contributor

Unverified Answer

What is huntington disease?

"Huntington disease is a neurodegenerative disorder and is a leading cause of early death among Caucasians less than 60 years old. It is characterized by motor symptoms of movement and behavior, including slowness of movements and involuntary movements and is the result of a genetic mutation. The incidence of Huntington varies from 1 in 36,000 to 1 in 80,000 among Caucasians of European ancestry. The main symptoms of Huntington include motor problems, abnormal thought patterns, and behavioral problems. Approximately 80% of those with Huntington will die from Huntington disease by the age of 75 years, although the disease has a gradual onset. It is named for an ancestor of John Wesley Huntington (1810-1877), an English businessman, politician, and philanthropist." - Anonymous Online Contributor

Unverified Answer

How many people get huntington disease a year in the United States?

"An estimate is provided of how many children with HD are born in the United States each year based on estimates for the number of people with HD in the United States. This estimate is similar to an estimate of how many children with HD were born in 1981 and may provide a basis for estimating the future prevalence of HD in this population." - Anonymous Online Contributor

Unverified Answer

How does wve-003 work?

"WVE-003 is safe and well-tolerated. The most frequently reported adverse events were rash/elevated transaminases, fatigue, headaches, and nausea. While most patients were able to complete the study, treatment was discontinued in four patients due to adverse events. WVE-003 induces a significant cellular induction of Nf kappa B in patients with neuropsychiatric signs and symptoms consistent with HD. Data from a recent study provided the first evidence that WVE-003 can induce a cellular induction of nuclear factor kappa B in HD patients as a result of a short intravenous infusion." - Anonymous Online Contributor

Unverified Answer

What is wve-003?

"wve-003 is the first clinical trial to evaluate the safety and efficacy of gene editing in humans. wve-003 utilizes a new non-interdisciplinary approach to identify the most effective and safe strategy to deliver RNA that causes a targeted reversal of the HD mutation. wve-03 may one day help researchers and scientists understand how to correct the genetic mutation that causes Huntington's disease and alleviate the symptoms associated with the disease." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in wve-003 for therapeutic use?

"Findings from a recent study indicates that wve-003 delivers an improvement in motor function compared to current treatments and has also shown the efficacy of wve-003 in reducing and preventing development of tremor/Parkinsonism. Moreover, the findings of this animal study suggest that wve-003 may be a novel form of treatment/cure for WCH3. Therefore, we believe that wve-003 might be a valuable addition for the therapeutic arsenal against WCH3." - Anonymous Online Contributor

Unverified Answer

What does wve-003 usually treat?

"As far as can be expected with a small-scale clinical study design, wve-003 did not produce significant clinical improvement over existing treatments. wve-003 did not produce dramatic therapeutic effects in patients with moderate to severe PD, but the most commonly observed side effects were expected. wve-003 was not indicated for this indication because the study was small and with limited durations of follow-up time. wve-003 was not indicated for this indication. wve-023, although not formally evaluated in this study, was approved and available for commercialization by the US Food and Drug Administration." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating huntington disease?

"There have been several new treatments for HD: the anticholinesterase drugs, the antigliadin peptide, the mamajide and the tetracycline antibiotics. All of these drugs treat problems that are not related to the disease or are used mainly in preclinical or clinical research. None of these treatments is likely to ever become FDA approved. Another treatment in development is gene therapy to treat people with early-onset (before-40) Huntington's disease using an adeno-associated virus expressing a truncated version of the HD protein." - 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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