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HD Clinical Trials

HD Clinical Trials, HD Community

GENERATION HD2-Clinical Trial Open to the Huntington’s Disease Community

The HDSA RESEARCH WEBINAR: The Roche/Genentech GENERATION HD2 Study of Tominersen; a new clinical trial that is now open to folks with Huntington’s disease (HD) who meet the criteria. The webinar is worth watching.  
Tominersen is an antisense therapy that acts by reducing the production of all forms of the huntingtin protein (HTT), including its mutated variant (mHTT), which is believed to be the main cause of HD.

Info on the study:
-Adults 25-50 years of age (early stage of HD)
-360 people in the study
-Clinical trial locations will be in 15 countries; more than 1 in US
-16 month treatment period
Go to: http://gene.com/patients/clinical-trial-information to read about the Tominersen study and how to get involved. Or ClinicalTrials.gov or signup for HDTrialFinder

Huntington’s disease is a rare, fatal, genetic brain disorder that has NO CURE, strikes young, is a long and progressive disease and has the symptoms of ALS, Alzheimer’s and Parkinson’s, simultaneously.  41,000 Americans are symptomatic and approximately 200,000 are at risk.

We Can Never Lose Hope…

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and author.  Visit my website  https://theresecrutchermarin.com

HD Clinical Trials

HDSA Webinar on Why Clinical Trial VIBRANT-HD Was Stopped

  Disappointing news for the Huntington’s disease community from Novartis Pharmaceutical Company regarding stopping the clinical trial VIBRANT-HD.  Read full article here

“Tucked into its latest earnings report, Novartis confirmed to investors that it has stopped developing an experimental drug for Huntington’s disease.

The reason, according to Novartis, was an “overall assessment of the risk-benefit profile” observed in a mid-stage clinical trial named VIBRANT-HD. In August, Novartis said it had temporarily suspended dosing in the study, after discovering that some patients treated with the drug, known as branaplam, were experiencing nerve damage.”

Register for Webinar on February 22, 2023

We Can Never Lose Hope…           

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.  Visit my website  https://theresecrutchermarin.com

 

HD Clinical Trials

Annexon Biosciences Good News on its Open-Label Phase 2 Clinical Trial for Huntington’s Disease

Annexon, a clinical-stage biopharmaceutical company, located in the San Francisco Bay Area, is a company with good news for the Huntington’s disease  (HD) community.  HD is a rare, fatal, genetic brain disorder that strikes young and has symptoms of ALS, Parkinson’s disease and Alzheimers at the same time.             

The local biotech company is developing a new class of complement medicines for patients with classical complement-mediated  autoimmune, neurodegenerative and ophthalmic disorders announced on June 7, 2022, the final data from its open-label Phase 2 clinical trial of ANX005 in patients with Huntington’s disease (HD).  Read the full news release here

The progress on ANX005 for Huntington’s disease is one of the green arrows below.

The following is a summary of the results of the Phase 2 clinical trail of ANX005.

-ANX005 Demonstrated Full C1q Target Inhibition and Was Generally Well-Tolerated

-Disease Progression Stabilized in Overall Patient Population Through the Nine-month Study

-Rapid Improvement in Clinical Outcome Measures Maintained in Patients with High Baseline Complement Activity Through the Nine-month Study

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.         

Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.  Find it on:  Amazon, and many online book stores.  100% of book sales is donated to Huntington’s Disease Society of America.

 

HD Clinical Trials

Bayer Unit BrainVectis to Start Huntington Gene Therapy Trial

Some good news for the Huntingtons disease community around the world from the company Bayer.

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease“.

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.         

Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.  Find it on:  Amazon, and many online book stores.  100% of book sales is donated to Huntington’s Disease Society of America.

 

 

 

HD Clinical Trials

Novartis Temporarily Suspends Clinical Trial on Liquid Branaplam-A Huntington’s Disease Drug

Author Therese Crutcher-Marin, HD Advocate

The following announcement is very disappointing to the Huntington’s disease community around the world, and I am saddened by it, but, We Can Never Lose HOPE.       

Novartis Pharmaceutical Company stated they will be gathering more data and will share information to the HD community in the next few months and decide on next steps.

August 24, 2022 Community update:

Status of VIBRANT-HD, the study of branaplam/LMI070 in Huntington’s Disease

“Dear Huntington’s Disease Community:

We are sharing this update to make sure you have the latest information regarding our ongoing clinical trial of in Huntington’s Disease. Novartis has decided to temporarily suspend dosing of study drug in the Phase 2b VIBRANT-HD study of branaplam in adults with HD. This decision was made because early signs of side effects were seen in some participants in the trial. The recommendation was made by an independent Data Monitoring Committee (DMC), following a regularly planned data review, which revealed findings that suggested possible side effects – specifically that branaplam might be causing peripheral neuropathy, which results from injury to the nerves located outside of the brain and spinal cord.”  Read the complete statement from Novartis here

Read in layman terms regarding Novartis statement-HDBuzz

HDBuzz is saddened to share the news that the VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam, has been temporarily suspended for the safety of participants.

 

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.         

Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.  Find it on:  Amazon, and many online book stores.  100% of the purchase price is donated to Huntington’s Disease Society of America.


HD Clinical Trials

Prilenia’s Oral Medication for Huntington’s Disease To Maintain Function

 

CLINICAL TRIALS

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Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is an active global phase 3 trial conducted in collaboration with the Huntington Study Group (HSG). The aim of the study is to evaluate pridopidine’s impact on Total Functional Capacity (TFC score) as the primary endpoint. TFC is a validated and clinically meaningful endpoint that measures the patient’s ability to conduct certain activities. Pridopidine is the first treatment to ever achieve maintenance of function in early HD patients (as measured by TFC) in the previous PRIDE-HD study. PROOF-HD is the only late-stage trial in HD targeting clinical progression.

The PROOF-HD study completed patient enrollment ahead of schedule and with numbers over the enrollment target. It is on track to reach its target readout in early 2023.

Watch the video from Prilenia on HD-Proof

Henk Schuring, Chief Regulatory and Commercialization Officer, Prilenia Therapeutics, discusses the PROOF-HD Huntington disease clinical trial. Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic at around the age of 40 years. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.

Author Therese Crutcher-Marin

We Can Never Lose HOPE…

HD Clinical Trials

The Pausing of Two Huntington’s Disease Clinical Drug Trials

Author Therese Crutcher-Marin, HD Advocate

Last Thursday, August 11th, I watched a HDSA Research Webinar: HD Clinical Trial Update with Arik Johnson, PsyD (HDSA’s Chief Mission Officer), Leora Fox, PhD (HDSA’s Assistant Director of Research & Patient Engagement), Jennifer Simpson, LCSW (HDSA’s Assistant Director of Youth & Community Services) and Victor Sung, MD (HDSA’s Chairman of the Board of Trustees) discussed recent Huntington’s disease (HD) clinical trial news. 

Last week Novartis Pharmaceutical Company announced VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam was temporarily suspended for the safety of participants.

Also last week, UniQure Biotech Company announced that it has paused enrollment in its higher-dose cohort receiving AMT-130 due to the occurrence of suspected unexpected severe adverse reactions.

Both trials were in the safety & dose level.  At this time, the HD community is waiting anxiously for more information from Novartis and UniQure.
Since the trials were paused and not halted, we should not lose HOPE because after the scientists/doctors evaluate the data, the trial may resume.
We Can Never Lose Hope…
HD Clinical Trials

A Series of Blogs Updating Clinical Trials on Drugs for Huntington’s Disease

Over the next several weeks I will update the progress on the drugs in clinical trials that are being conducted around the world for Huntington’s disease (HD).                               

 

                 

The companies/drugs I will be sharing  are:

Prilenia-HD Proof Improves day-to-day function, movement & behavior in HD

Roche-Tominersen  May help younger people with HD

PTC Therapeutics-PTC-518 Designed to selectively lower huntingtin mRNA and protein

Novartis-Vibrant-HD (branaplam) Lowers levels of the huntingtin protein 

UniQure-AMT-130 Gene therapy

Neurocrine-Kinect HD Treatment of chorea

Sage-Sage-718 Cognitive function

We Can Never Lose HOPE……

Author Therese Crutcher-Marin

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.  Visit my website and learn about HD:  https://theresecrutchermarin.com

 

 

 

HD Clinical Trials

Interested in Joining A Clinical Trial for Huntington’s Disease?

Author Therese Crutcher-Marin, HD Advocate

If you are considering joining a clinical trial for Huntington’s disease (HD) but don’t know how  to find the right clinical trial for you, please go to HDSA  HD Trial Finder sponsored by the nonprofit Huntington’s Disease Society of America (HDSA).

HD Trial Finder is a clinical trials matching service: a way for individuals with Huntington’s disease, caregivers, healthy volunteers, and physicians to connect with current research studies. It includes an easy-to-use website and free call center staffed by trained HD clinical trial navigators.

Hope for tomorrow begins with your involvement today!

People with Huntington’s Disease, care partners, and healthy volunteers are all needed today to participate in different aspects of HD research. By creating a profile and answering some basic questions about yourself or a friend, family member, or patient who is impacted by HD, you can match to studies and find contact information for nearby sites. You can also use HD Trial Finder to read about ongoing trials without entering any information.

We Can Never Lose HOPE….

HD Clinical Trials

uniQure AMT-130-Gene Therapy Clinical Trial for Huntington’s Disease

Silencing the mutant huntingtin gene 

A promising gene therapy for a rare, devastating disease             

uniQure has begun dosing patients in a dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease (HD).

This investigational treatment is the first one-time administered gene therapy to enter clinical testing for the treatment of HD. AMT-130 is administered only once by neurosurgical procedure.

If you live in the San Francisco Bay Area, the University of San Francisco Medical Center is involved in this clinical trial.

Read here:  UniQure press release with clinical update on HD patients in the trial Dec. 16,2021.   

Frequently asked questions about AMT-130

Animated illustration of how AMT-130 is administered into the caudate and striatum regions of the brain via a micro-catheter   Watch it here

We Can Never Lose HOPE……

Author Therese Crutcher-Marin

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.  Visit my website and learn about HD:  https://theresecrutchermarin.com