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HD Clinical Trials

The FDA Orphan Drug Act/Huntington’s Disease

In 1983, Orphan Drug Act (ODA) was set up to encourage increased development of drugs for rare diseases. It was amended in 1984 to define rare diseases as those that affect less than 200,000 people in the United States, but it also included drugs for diseases affecting more than 200,000 people as long as there was no commercial viability—that is, that the cost of development and making available in the United States a drug for the disease would exceed revenue from the US sales. Huntington’s disease fits that criteria. Overview-of-huntingtons-disease/

Wave Life Sciences has seen the FDA grant it an orphan drug designation for its lead candidate WVE-120101. The experimental drug, which targets rs362307–a single nucleotide polymorphism that is associated with the disease-causing mutation in the huntingtin gene.

The orphan status gives it 7 years of exclusivity in the U.S., and other boosts, including tax credits related to clinical trial expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design.

To read more about the Orphan Drug Act, visit: https://www.ajmc-the-orphan-drug-act

We Can Never Lose HOPE………………

The author website for the nonfiction book I published is https://www.theresecrutchermarin.com

Watching Their Dance on many book websites like Amazon https://www.kirkusreviews.com/book-reviews/unknown/watching-their-dance/

August 27, 2019 by crutcht

HD Clinical Trials

SIGNAL Clinical Trial for Huntington’s Disease

Another Sponsor at the Huntington’s Disease Society of America in Boston last June was Vaccinex, Inc.

Vaccinex and the Huntington Study Group (HSG) launched a trial for people with the gene mutation that causes HD who are either early in the progression of the disease or are not yet diagnosed with the disease.

SIGNAL, a Phase 2, multi-center, randomized, double-blind, placebo controlled study in subjects with early manifest and late prodromal Huntington’s disease (HD) to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503 (pepinemab). Pepinemab a monoclonal antibody that is a potential treatment for Huntington’s disease (HD). What-is-hd/overview-of-huntingtons-disease/

On January 16, 2019, Vaccinex, Inc. announced the enrollment was complete and includes two cohorts with a total of 265 Huntington’s Disease subjects – 179 in group 1 (B1) who have early manifest disease and 86 in group 2 (B2) who are late prodromal. All subjects are randomized to receive monthly infusions of either VX15/2503 (pepinemab) or placebo for 18 months in double-blind fashion without crossover.

To read FAQ regarding about the trial visit: https://huntingtonstudygroup.org/signal-faq/

Please visit my Author Website to read my blogs about Huntington’s disease and my personal experience https://www.theresecrutchermarin.com

We Can Never Lose HOPE……………….

Author Therese Crutcher-Marin

August 22, 2019 by crutcht

HD Clinical Trials

WAVE Life Sciences-An Update on PRECISION-HD Clinical Program

WAVE Life Sciences had a table at the Huntington’s Disease Society of America (HDSA) Convention in Boston last month and I spoke with a rep for quite awhile. It’s very exciting what they are doing for Huntington’s disease (HD).

“We have designed stereopure oligonucleotides to selectively target the mutant HTT allele to lower the production of mHTT protein while leaving healthy HTT protein relatively intact.” https://www.wavelifesciences.com/

Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities usually during their prime working years and has no cure. http://www.hdsa.org

A little bit of information: We all have two Huntingtin genes; one from your father, one from your mother. Say, you have a CAG repeat of 42 from your father and a CAG count of 17 from your mother.

The drug WAVE life Science is conducting clinical trials on, Phase 1b/2a, only lowers the mutated Huntingtin gene and leaves the healthy one alone.

IMPORTANT NEWS

WAVE is currently enrolling adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362307 (“SNP1”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov
You may also find information about clinical trials in HD at hdtrialfinder.org

WAVE is currently enrolling adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362331 (“SNP2”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov
You may also find information about clinical trials in HD at hdtrialfinder.org

We Can Never Lose HOPE…………….

100% of the profits from my book is being donated to the nonprofit, HDSA. Since publication in 2017, I’ve donated over $14,000.00. It can be found on many book websites.

August 1, 2019 by crutcht

HD Clinical Trials

Gene Therapy AMT-130 for Huntington’s Disease

The Huntington’s disease (HD) community has never been so hopeful that a therapy will be found for HD in the next few years.

A new development in gene therapy was announced by uniQure, whose focus is to advance the future of medicine through gene therapy. http://www.uniqure.com/about/corporate-mission.php

“The U.S. Food and Drug Administration (FDA) has completed its review of our Investigational New Drug (IND) application for AMT-130, allowing us to begin our planned Phase I/II study. We expect that in the second half of 2019 AMT-130 will be the first ONE-TIME administered AAV gene therapy to enter clinical testing for the treatment of Huntington’s disease.” To read about the clinical trial, visit http://uniqure.com/gene-therapy/huntingtons-disease.php

uniQure’s gene therapy candidate for Huntington’s disease is differentiated in that:

AMT-130 targets the deep brain structures known for the disease pathology onset.
AMT-130 silences mutant huntingtin protein at levels not demonstrated in other studies.
AMT-130 targets the accumulation of the exon 1 HTT fragment, the most toxic source of abnormal protein aggregation in Huntington’s disease.

The AMT-130 trial will be based at HD clinical sites in the Unites States. It’s not known at this time what sites or how many, yet. These will be publicly announced when they come online. UniQure hopes to begin enrolling patients before the end of 2019. It is brain surgery. https://en.hdbuzz.net/274

Please sign up for EnrollHD if you would like to participate in a HD clinical trial study. https://www.enroll-hd.org/

We Can Never Lose HOPE…..

July 25, 2019 by crutcht

HD Clinical Trials, Hope

The Pharmaceutical Company, Genentech

Statement from Genentech.
“There’s an innate brilliance in each of us. It lives in the thousands of thoughts and feelings that move through our bodies each day. It comes to life in our expressions and movements. In how we connect with each other, and in the moments and memories we create together.

Neurological conditions can dim this brilliance. They can create barriers that impede our ability to
communicate. To move. To remember. Over time, these diseases can rob us of our identities.

Our hope is that through our work in neuroscience, combining new scientific understanding with clinical advancements, we can help preserve the unique qualities that make people who they are.” stories/preserving-what-makes-us-who-we-are

Author Therese Crutcher-Marin

Last December, Roche/Genentech announced the locations, including 20 sites in the U.S. and 6 in Canada, for their phase 3 study – called GENERATION HD1 – to test efficacy of the huntingtin-lowering therapy RG6042. To read full press release, visit RG6042-GENERATION-HD1-Study-update-.pdf

We Can Never Lose HOPE……………..

Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions.

June 18, 2019 by crutcht

HD Clinical Trials

Enroll -HD: A Prospective Registry Study in a Global Huntington’s Disease Cohort

Author Therese Crutcher-Marin

At this time, in the Huntington’s disease arena around the world, people are needed to participate in the exciting clinical trials being conducted. Without people, very brave people, we cannot move forward in finding a cure for the cruelest disease on the planet; Huntington’s disease (HD).

Enroll-HD is a longitudinal, observational, multinational study that will integrate two existing Huntington’s Disease (HD) registries, REGISTRY in Europe and COHORT in North America and Australia, while also expanding to include sites in Latin America and Asia. With no end date and annual assessments, the goal of Enroll-HD is to build a large and rich database of longitudinal clinical information and biospecimens. This database will serve as a basis for future studies aimed at developing tools and biomarkers for progression and prognosis, identifying clinically relevant phenotypic characteristics, and establishing clearly defined endpoints for interventional studies. To learn more about Enroll-HD, go to: https://www.enroll-hd.org/

https://chdifoundation.org/huntingtons-disease-society-of-america-endorses-enroll-hd/

https://memory.ucsf.edu/enroll-hd

https://www.hda.org.uk/research/current-research-studies/enroll-hd

Enroll-HD is a “platform”

The idea is that this one study will make all other HD research easier, speeding up the process of developing new drugs and other treatments that really work. Rather than being restricted to a select group of researchers, it will be a resource for all HD science, making it faster and more efficient for other researchers to conduct their projects. This is why it’s called a platform, it supports other work.

To find clinic locations near you, go to: https://www.enroll-hd.org/participate/clinic-locations/

As promised, HDBuzz’s Jeff Carroll finally signed up for the Enroll-HD study. Learn more about this important global HD “observational study” here. https://en.hdbuzz.net/261

We Can Never Lose Hope…….

April 23, 2019 by crutcht