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Clinical Trials & Research Studies

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 Receives FDA Clearance to Commence RT001 Phase 2/3 Clinical Trial in Patients with Infantile Neuroaxonal Dystrophy (INAD)

The U.S. Food and Drug Administration (FDA) has granted Retrotope approval to conduct an open-label Phase 2/3 clinical trial of its investigational drug RT001 to evaluate efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD). RT001 is the first-in-class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage resulting in cell death that is a hallmark of numerous neurodegenerative diseases including INAD. To date, Retrotope has enrolled two INAD patients in two separate Expanded Access trials, the first patient having begun treatment in March 2017 and the second patient having enrolled in November 2017.  Promising results from the first study were presented at the recent annual meeting of the American Academy of Neurology, and treatment of both children remains ongoing. RT001 has been granted U.S. FDA orphan drug designation for the treatment of PLA2G6 associated neurodegeneration (PLAN), which includes INAD.

Read the full press release here:

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, INAD, ALS, Friedreich’s ataxia (FA), Late Onset Tay Sachs (LOTS), Familial Encephalopathy with Neuroserpin Inclusion Bodies (FEIN or neuroserpinosis), mitochondrial myopathies, and retinopathies. RT001, Retrotope’s first lead candidate, is being tested in clinical trials for the treatment of FA, a fatal orphan disease, and in compassionate use studies for the fatal, neurodegenerative diseases such as INAD, LOTS, FEIN, and a genetic form of Alzheimer’s disease. For more information about Retrotope, please visit

About RT001 – RT001 is a patented, first-in-class, orally available D-PUFA, a deuterated polyunsaturated fatty acid, that incorporates into mitochondrial and cellular membranes and stabilizes them. Retrotope and others have discovered that lipid peroxidation, the free-radical damage of polyunsaturated fats (PUFAs) in mitochondrial and cellular membranes, may be the primary source of cell death in several degenerative diseases, including Friedreich’s Ataxia (FA) and INAD. The presence of D-PUFAs (RT001) can help protect (“fireproof”) against this attack and potentially restore cellular health.

Additional information 

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:

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:

For INAD Patients and Families:

For additional information please direct all questions to:

Sarah Endemann
Clinical Coordinator
Retrotope, Inc.
Tel: 619.206.5944


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:


Retrotope is Recruiting Participants for Natural History Study

Retrotope is currently recruiting participants for a natural history study about Infantile Neuroaxonal Dystrophy (INAD).  A natural history study is a study that follows a group of people over time who have, or are at risk of developing, a specific medical condition or disease. A natural history study collects health information in order to understand how the medical condition or disease develops and how to treat it. In this study we plan to collect information about INAD through a combination medical records and interviewing patients’ families. The purpose of this study is to define the natural history of INAD and demonstrate how INAD progresses. No treatment will be offered through participation in this study and participation is completely voluntary.  Study participants may not benefit directly from partaking in this study, but the information generated from this study could be useful in developing future clinical trials and potential treatments for INAD.

If you or someone you know is interested in participating in the study, please contact Sarah Endemann at tel:619-206-5944 or by email at