What side effects are associated with this type of intervention?

Common treatments for Huntington's Disease include antipsychotics, antidepressants, and medications for movement disorders. Antipsychotics, such as risperidone and olanzapine, can cause side effects like weight gain, sedation, and extrapyramidal symptoms (e.g., tremors, rigidity). Antidepressants, such as SSRIs and SNRIs, may lead to nausea, insomnia, and sexual dysfunction. Medications for movement disorders, like tetrabenazine, can cause drowsiness, depression, and parkinsonism. Pridopidine, a Sigma-1 Receptor Agonist, is being studied for its safety and efficacy, with potential side effects including mild gastrointestinal issues and dizziness.

What prior FDA approvals exist?

The FDA has approved several treatments for Huntington's Disease, primarily focusing on managing symptoms. Notably, vesicular monoamine transporter type 2 (VMAT2) inhibitors such as deutetrabenazine and tetrabenazine are approved for treating chorea associated with Huntington's Disease. These drugs help reduce involuntary movements but carry risks of depression and suicidality. Second-generation antipsychotics like risperidone, olanzapine, and aripiprazole are also used, particularly in patients with concurrent psychiatric symptoms. While Pridopidine, a Sigma-1 Receptor Agonist, is being studied for its potential benefits, it is not yet FDA-approved.

What other types of research exist?

Promising novel alternatives to Pridopidine for Huntington's Disease patients include norbenzomorphans, which have shown significant neuroprotective activity in models of neurodegeneration. Additionally, other sigma receptor ligands such as NPC 16377, which has demonstrated neuroprotective and antipsychotic potential, may also be considered.

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Pridopidine for Huntington's Disease

Phase 3

Waitlist Available

Research Sponsored by Prilenia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of HD based on clinical features and the presence of ≥36 CAG repeats in the huntingtin gene

Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of ≥7, at screening

Must not have

Gene therapy at any time

Any serious medical condition or clinically significant laboratory, or vital sign abnormality that precludes the patient's safe participation in and completion of the study e.g. significant heart disease within 12 weeks before baseline or history of certain cardiac arrhythmias

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called pridopidine in people who have early stage Huntington Disease. The goal is to see if this medication can help manage their symptoms and slow down the disease. Patients who finish the main part of the study can continue taking the medication for a longer period. Pridopidine has been shown to be well-tolerated and may reduce motor impairment in patients with Huntington's disease.

Who is the study for?

This trial is for adults with early stage Huntington's Disease, specifically stages 1 or 2, as indicated by a UHDRS-TFC score of ≥7. Participants must have a confirmed diagnosis with ≥36 CAG repeats in the huntingtin gene and onset of symptoms at age 18 or older. Those who've had gene therapy, used pridopidine within the last year, have serious medical conditions like recent heart disease or seizures in the past five years, or are pregnant can't join.

What is being tested?

The study tests if pridopidine (45mg twice daily) is effective and safe for patients with early stage Huntington's Disease compared to a placebo. Patients will be randomly assigned to receive either pridopidine or an inactive substance without knowing which one they're getting.

What are the potential side effects?

While specific side effects for this trial aren't listed here, common side effects from similar medications may include nausea, fatigue, dizziness, and mood changes. The safety profile of pridopidine will be closely monitored throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Huntington's disease based on genetic testing.

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My condition is in the early stages, with a functional score of 7 or higher.

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My Huntington's disease symptoms started when I was 18 or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have never received gene therapy.

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I don't have any serious health issues that would make it unsafe for me to join the study.

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I have not had epilepsy or seizures in the last 5 years.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2022 Phase 2 & 3 trial • 163 Patients • NCT04615923

28%

Fall

24%

Muscular weakness

20%

Neuromyopathy

12%

Diarrhoea

12%

Constipation

12%

Nausea

11%

Dysphagia

10%

Fatigue

10%

COVID-19

Dysarthria

Salivary hypersecretion

Dry mouth

Contusion

Dizziness

Oedema peripheral

Arthralgia

Musculoskeletal pain

Insomnia

Anxiety

Complication associated with device

Dyspnoea

Headache

Depression

Post lumbar puncture syndrome

Muscle contractions involuntary

Gastroesophageal reflux disease

Amyotrophic lateral sclerosis

Syncope

Cognitive disorder

Respiratory failure

Atrial fibrillation

Skin abrasion

Pain in extremity

COVID-19 pneumonia

Cerebellar stroke

Presyncope

Hip fracture

Ischaemic stroke

Pulmonary embolism

Pneumoperitoneum

Failure to thrive

Traumatic intracranial haemorrhage

100%

80%

60%

40%

20%

Study treatment Arm

Pridopidine

Matching Placebo

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PridopidineExperimental Treatment1 Intervention

45 mg pridopidine twice daily (BID)

Group II: PlaceboPlacebo Group1 Intervention

Matching placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pridopidine

2020

Completed Phase 3

~580

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Huntington's Disease (HD) include VMAT2 inhibitors like tetrabenazine, deutetrabenazine, and valbenazine, which reduce chorea by depleting monoamines such as dopamine. This is crucial for HD patients as it helps manage involuntary movements. Pridopidine, a Sigma-1 Receptor Agonist, is being studied for its potential neuroprotective effects, which may help preserve neuronal function and slow disease progression. Additionally, neuroprotective agents like cannabigerol (CBG) have shown promise in reducing mutant huntingtin aggregation, potentially mitigating neurodegeneration. These mechanisms are vital as they address both symptomatic relief and underlying disease pathology, offering a comprehensive approach to HD management.

Neuroprotective properties of cannabigerol in Huntington's disease: studies in R6/2 mice and 3-nitropropionate-lesioned mice.