Research Studies and Clinical Trials

INADcure Foundation’s role in Clinical Trials and Research Studies:

All information posted by the INADcure Foundation on this page is for informational purposes only. As a non-profit charitable organization, we do not endorse specific studies or clinical trials, clinical trial designs, experimental drugs or procedures, or pharmaceutical companies. Additionally, the Foundation does not participate in subject recruitment or eligibility evaluation for any trial or recommendations that may be derived from any clinical trial or its outcomes. We encourage families to consult with their medical team prior to enrolling or participating in any of the below opportunities.

The INADcure Foundation does not provide financial support to the following study.

Retrotope, Inc. Assessing medical records of children with INAD in anticipation of FDA approval of clinical trail.

Retrotope, Inc. is currently assessing medical records of children with INAD in anticipation of FDA approval for an upcoming clinical trial for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).

About Retrotope – Retrotope, a privately-held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease modifying therapies for many intractable diseases such as Parkinson’s, Alzheimer’s, neuromuscular diseases, and retinopathies. RT001, Retrotope’s first lead candidate, is for the treatment of rare orphan neuromuscular diseases, and is being tested in clinical trials for Friedreich’s ataxia and INAD. For more information about Retrotope, please visit www.retrotope.com.

About RT001 – RT001 is a patented, orally available modified fatty-acid therapeutic that stabilizes (“fireproofs”) mitochondrial and cellular membranes against attack and restores cellular health. Retrotope has discovered that lipid peroxidation, the free-radical degradation of lipids in mitochondrial and cellular membranes, may be causative of a wide range of degenerative diseases. Free radicals attack and degrade polyunsaturated fats (PUFAs) that are essential membrane components. Retrotope has shown that the degradation products of these fats create toxic cascades in many illnesses of degeneration.

March 2017 – Retrotope enrolled its first patient compassionate use trial for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).

November 2, 2017 – Retrotope announced that the U.S. Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).

See full press release here: https://static1.squarespace.com/static/549af14ae4b004237f7bb71a/t/5a0ea01d085229ac6656c5d5/1510907934034/Retrotope+INAD+Orphan+Drug+Press+11-2-2017+final+wire.pdf

November 16, 2017 – Retrotope enrolled a second subject in a compassionate use trial of the ultra-rare, neurological disease, Infantile Neuroaxonal Dystrophy (INAD).

See full press release here: https://static1.squarespace.com/static/549af14ae4b004237f7bb71a/t/5a0ea1b3e4966bd1984dc50f/1510908340401/Retrotope+INAD+Patient+2+Final+Press+11-16-2017.pdf

For INAD Patients and Families: https://www.retrotope.com/inad-clinical-trial

For additional information please direct all questions to:

Frederic Heerinckx
Senior Director of Clinical Operations
(408) 834- 5729
Frederic@Retrotope.com

OHSU Natural History Study Recruiting Participants

The NBIA Research Group at Oregon Health and Science University has developed a new study called PLANready.

The purpose of this study is to help researchers better understand the natural history of PLAN, meaning how symptoms appear and change over time. By studying individuals with PLAN, the NBIA Research Group at OHSU hope to identify disease markers that can be used in future clinical trials. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. A disease marker could be an MRI finding, a protein level in the blood, or a rating scale to measure symptoms or function. Natural history studies provide data that serve as the foundation for future drug trials.

For more information and to sign up, please visit: http://nbiacure.org/our-research/in-the-clinic/planready/