Neuraltus Pharmaceuticals NP001 - Identifier Number NCTO1281631*
This clinical trial is a Phase II double-blind, randomized, placebo-controlled multi-center study. The purposes of the trial include efficacy, safety and dosage. Patients receive a total of 20 intravenous injections of anywhere from 3 to 5 injections each month. There are to be two different dosage groups and one placebo group and will have a duration of six months plus three additional months of monitoring. The trial is currently recruiting at several locations throughout the US .
Why this trial is notable
Principal Investigator Dr. Robert G. Miller of Forbes Norris MDA/ALS Research Center in San Francisco, California, is a top echelon ALS clinician researcher. The scientific foundation of NP001 is extremely sound. Andrew Gengos, CEO of Neuraltis says, “NP001 regulates macrophages which play an important role in neuroinflammation. Our hope is that if we can reduce or eliminate the neuroinflammation, it will have a beneficial effect on the underlying progression of ALS.”
While now still in a Phase II trial, there is the potential for early approval from FDA of NP001 as a prescribed medication once the Phase III trial is underway. The original research, containing very impressive data upon which NP001 is based, was led by Dr. Stanley Appel of Methodist Hospital Research Institute, Houston and colleagues. Finally, there have been strong anecdotal reports of symptom amelioration by several research subjects in Phase II trial. However, it is uncertain how applicable these appearances of benefit (all self-observed) are translatable to others.
Issues of concern
1. This trial is still early. Phase II to the end of Phase III and FDA approval may require an additional 5-7 years of testing unless early usage allowance is granted. If it is granted, the time constraint will be dramatically shortened.
2. In order to participate, initial symptoms must be within a 36 month maximum range (recently changed from 24 months) and patients cannot be on a ventilator, Bi-Pap, C-Pap, trache/ventilation or gastronomy tube.
3. Anectodal reported side effects include lethargy, tiredness, dizziness.
Principal Investigator statement
“Thanks for asking for my view of this exciting trial. We all are very positive about the scientific underpinning of this trial—attacking neuroinflammation at the cellular level. We are also very keen on the early responses of our biomarkers in the Phase I trial—the field desperately needs biomarkers for ALS. Moreover, we all have been very happy with the safety record to date of NP001. Those of us who have been involved in clinical trials for a long time, have seen hopes rise during a trial before.
In the past, those hopes were not sustained—of course we hope it will be different this time around. We will be doing an interim analysis of efficacy, and will consult with the FDA about those results once we see the trial through to completion. We will report the trial results as soon as humanly possible. We are hearing some positive comments, but nothing that would cause any of us to deviate from usual and customary practice in bringing this trial to a swift conclusion. Thanks for all the great work you do for all patients with ALS.”
Dr. Robert G. Miller, MD
More information can be found at the article
Neuraltus Pinning Hopes on Lou Gehrig's Disease.