What is ALS Phase 2 Study NP001?

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See also: Clinical Trial

NCT01281631 is a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS). Other study ID number is NP001-10-002

Dosages

Most common dosages (by patients currently using ALS Phase 2 Study NP001)
Dosage Patients
1 other monthly 9
1 monthly 3
3 other monthly 2
monthly 1
0.5 mg monthly 1
1 mg monthly 1
5 mg monthly 1
5 other monthly 1
30 min monthly 1
250 mg monthly 1
Why Patients Stopped Using ALS Phase 2 Study NP001 (multiple reasons could be selected)
Reason Patients
Course of treatment ended 8
Doctor's advice 1
Side effects too severe 1
Other 1
Not indicated 1
See all 11 patients who’ve stopped using ALS Phase 2 Study NP001

Duration

Currently Using ALS Phase 2 Study NP001
Duration Patients
0-1 month 0
1-3 months 0
3-6 months 0
6 months-1 year 0
1-2 years 0
2 years or more 2
Stopped Using ALS Phase 2 Study NP001
Duration Patients
0-1 month 0
1-3 months 1
3-6 months 7
6 months-1 year 2
1-2 years 1
2 years or more 0
Adherence
Always
30 94%
Usually
2 6%
Sometimes
0 0%
Never
0 0%
Burden
Very
1 3%
Somewhat
10 31%
A little
13 41%
Not at all
8 25%
Cost per month
$200+
8 31%
$100-199
5 19%
$50-99
2 8%
$25-49
2 8%
< $25
9 35%
Last updated:

3 patient evaluations for ALS Phase 2 Study NP001

HappyPhysicist
31836 thumb
HappyPhysicist
Sex: M
Data Quality: 2 stars
Bulbar: moderate
Arms: mild
Chest: mild
Legs: moderate
I am: vg
I have:
ALS

See HappyPhysicist's full ALS Phase 2 Study NP001 history

Jan 5, 2012 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Not at all
Advice & Tips: It has now been 8 weeks since my last infusion. In the past 4 weeks I have noticed a significant decline in my state. My speech is considerably worse and my left leg is showing signs of weakness to the point that my gait has changed and I feel a little off balance. My swallowing is also worse. I now have to drink water in very small sips to prevent myself form choking and I find that I am having difficulty swallowing food, even choking on food on occasion. It feels to me that my progression has now picked up where it left off just before I started the NP001 infusions.
Cost: < $25 monthly
Side effects: Fasciculations

Dec 19, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: None
  • Adherence: Always
  • Burden: Not at all
Advice & Tips: I has now been about 6 weeks since my last infusion. While my limbs seem to be stable my swallowing is declining. I choke almost every time I drink water now and I am starting to get into the habit of drinking milk with meals instead. Salivation is at this point worse than it was pre NP001. I have to work hard not to drool while I eat. My speech is about the same as pre NP001 or a little worse, but it does fluctuate from day to day. Overall I would say that I am declining, but again very slowly so far.
Cost: < $25 monthly
Side effects: Fasciculations

Nov 28, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Advice & Tips: Today was my first post infusion followup which was a week early due to physician scheduling. It has been 3 weeks since my last infusion and usually by now I am feeling pretty bad and really 'jonesing' for my next infusion. This time however I don't feel as bad as previously. Overall the effect of NP001 has been pretty incredible. I feel that while I am not as good as after my first infusion I am in most ways better than just before the first infusion. The good: I still do not have weakness in my left arm and leg in fact my left grip strength is greater, I do not often have to repeat myself, I am no longer biting my lips and I do not feel fatigued by the end of the day. The bad: my saliva/drooling is slightly worse than it was before I stared the study. I am now wiping the corners on my mouth many times a day. Before the study I would only need to do it a couple times a day. The same: After the first infusion I could reach my tongue to the lower right and clear food from between my gums there, which I could not do before the infusion. I am now not able to do that bringing me to about where I was before the study.
Cost: < $25 monthly
Side effects: Fasciculations

Nov 10, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Dosage: 100 Low dose NP001 (1mg/kg) Daily
Cost: < $25 monthly
Side effects: Fasciculations

Nov 9, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Dosage: 100 Low dose NP001 (1mg/kg) Daily
Advice & Tips: I just received my very last infusion of NP001. I think overall if I have declined during this study period it has been at a small fraction of the rate before starting the study. The only thing I can say that is worse is that I am more frequently wiping my lips. I think my lip muscles have atrophied but my tongue has not, neither have my left arm or leg. My left arm was getting weaker just before I started the study but it has held strong since. Along with the decrease in strength on my lips has been a very slight decline in my speech. I have been recording my speech weekly since August and it is very difficult to detect a change so the difference is very small. I believe overall NP001 has drastically slowed my progression if it hasn't stopped it. Overall, I am better than I was when I started the study.
Cost: < $25 monthly
Side effects: Fasciculations

Oct 14, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Dosage: Monthly
Advice & Tips: Today is Friday October 14th. I finished my fifth cycle of infusions which I received Monday, Tuesday and Wednesday September 12th, 13th and 14th. Not much has changed. I definitely see at pattern where I start to feel sluggish around 3 weeks after my infusions and the infusions perk me up again. Overall my 'vitals' have been pretty much constant since starting the study, FRS, weight, FVC dropped a little but that fluctuates a lot. My grip strength has remained constant since starting the study, although that too can fluctuate from day to day. I am having a difficult time evaluating my speech. In some ways it is better than before I started the study but in some ways it is worse. I have a much easier time talking for long periods of time and I can articulate better, however, it is more evident that my speech is affected. Before I could muster the strength to speak maybe one perfect sentence but it would take a lot out of me. Now I don't think I can pronounce all words perfectly no matter how hard I try. But I can blab on and on but my speech defects are obvious. There is no doubt people understand me better than they did before the study. An example is when I am at a store and I give my phone number, I no longer have to repeat it. The one way I think I may have declined is in drooling. I seem to have to wipe my lips more often than before and I noticed night time drooling for the first time about one month after starting the study. But the difference is very very small compared to what it was like about 9 months ago. I think perhaps my tongue is stronger but my lips are a little weaker. It is difficult to decide whether I am improving or declining, which in itself is a good thing. Before starting the study I would notice differences in my state every month and they were obvious to me. Right now I am struggling to decide whether there are changes in my state from 4 months ago.
Cost: < $25 monthly
Side effects: Fasciculations

Sep 17, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Dosage: Monthly
Advice & Tips: Today is Saturday September 17th. I finished my fourth cycle of infusions which I received Monday, Tuesday and Wednesday September 12th, 13th and 14th. I am seeing a pattern with the infusions. I seem to improve up to about 5-7 days after the infusions feeling my best the weekend following my infusions. After that I gradually slide back to a point somewhere in between where I was before my infusions started. After about 3 weeks from my first infusion I am feeling sluggish and my speech becomes labored again. I don't seem to lose the articulation I have gained but I do experience fatigue in my speech muscles again when I speak. I also begin to experience difficulty swallowing again around that 3 week period. It is a gradual transition, but it seems at that 3 week point is where I start to wonder if the NP001 effect is temporary. At that point I can't get my infusion soon enough. Then after a few days after my last infusion I am feeling invigorated again. While my state does rise and fall with the infusions, overall on average my state is constant. That is, I feel my ALS progression has stopped. (edit 9/17/2011: I did experienced increased fascinations starting during infusion and lasting approximately 2 days)
Cost: < $25 monthly
Side effects: Fasciculations

Sep 8, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: None
  • Adherence: Always
  • Burden: Not at all
Dosage: Monthly
Advice & Tips: Mid infusion update: I seem to notice a definite decline around week 3 after the infusion. That good old ALS fatigue seems to come back. This time around right at about 3 weeks I started experiencing cramping in my tongue which I have never had before. While my speech is still ok, if my tongue cramps up I have to pause until it stops. I am very much looking forward to my next infusion.
Cost: < $25 monthly
Side effects: Fasciculations

Aug 19, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: None
  • Adherence: Always
  • Burden: A little
Dosage: Monthly
Advice & Tips: A quick update. It has now been 2 days since I finished the last infusion cycle. My speech has improved considerably. I don't think my speech has been this good in about 4 months. If the past two months are any indication I expect it to get a little worse up to the next infusion and then take another jump in improvement. Each infusion seems to be an overall gain over the last infusion.
Cost: < $25 monthly
Side effects: Fasciculations

Aug 17, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: A little
Dosage: 100 Low dose NP001 (1mg/kg) Daily
Advice & Tips: I just finished my third infusion. Still no side effects. Again I have had a significant increase in the grip strength of both hands. My original grip strength scores were Left Hand: 67.5 lbs, Right Hand: 93 lbs. My values as of today are Left Hand: 110 lbs, Right Hand: 125 lbs. Before the study I was well below the average for a 44 year old male. For my left hand I was in the bottom 1% ! I am now just at the average mark for my left hand and above average for my right hand. This represents a 63% increase in the strength of my left hand and a 34% increase in the strength of my right hand! My speech has improved very slowly and fluctuates in quality form day to day. I myself have not noticed much of an improvement since about 2 weeks after the first infusion. However, I have been told by many people, especially those I don't interact with often that my speech is getting better and has improved over the last month. Today it seems especially good. There is one thing I know for sure. My progression has stopped since starting this trial. Furthermore the grip strength tests show definitively that I have improved at least in some areas. My FRS graph is deceiving. It make it look like I am a naturally slow progressor, but I was just about to start losing FRS points. I was loosing dexterity and strength in my left hand and left leg and given another couple of months my FRS score would have dropped 2-4 points. I have little doubt that NP001 prevented that. (edit 9/17/2011: I did experienced increased fascinations starting during infusion and lasting approximately 2 days)
Cost: < $25 monthly
Side effects: Fasciculations

Jul 19, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 100 Low dose NP001 (1mg/kg) Daily
Advice & Tips: Testing revealed dramatic increase in grip strength in left had of 26%, less dramatic but significant increase in grip strength in right hand of 7%. (edit 9/17/2011: I did experienced increased fascinations starting during infusion and lasting approximately 2 days)
Cost: < $25 monthly
Side effects: Fasciculations

Jun 22, 2011 (Started Jun 20, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 100 Low dose NP001 (1mg/kg) Daily
Advice & Tips: Vastly improved ability to swallow liquids and foods. No longer choking on foods or liquids. Increased tongue range of motion. Improved lip strength, improved pucker foce, improved ability to contain liquids in mouth. Reduced drooling. No longer biting lip during eating and talking. Slight improvement in speech. Regained consonant "t". Elimination of fatigue during speaking, able to speak for extended periods. Prior could only speak a few sentences before fatigue set in. Elimination of 'heavy/numb' sensation in left arm. No observable side effects. (edit 9/17/2011: I did experienced increased fascinations starting during infusion and lasting approximately 2 days) Overall vastly improved quality of life.
Side effects: Fasciculations

  • 39 helpful marks
Persevering
31042 thumb
Persevering
Sex: M
Data Quality: 3 stars
Bulbar: moderate
Arms: severe
Chest: none
Legs: moderate
I am: g
I have:
ALS

See Persevering's full ALS Phase 2 Study NP001 history

Oct 27, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: 11/28/11 I am now in the 5th week past the very last trial infusion.  Since infusion cycles are generally 28 days, I think of the first 4 weeks after the last infusion to be within the dosing portion of the trial.  I just "missed" the first repeat cycle last week.  As had occurred all 6 cycles, I sensed the drug wearing off beginning about 3 weeks after infusions.  Unfortunately, this time I get nothing to stop the resulting negative effects. This graph below shows my ALSFRS-R history, with the x-axis adjusted to 0 days as my first NP001 trial infusion. Points are plotted through the complete dosing portion.  The green vertical line was the first infusion.  Red is the last infusion. My FRS progression was historically very linear, with a correlation ceofficient = 0.94.  On this trend, my natural progression should have resulted in an FRS = 32 at the completion of the dosing phase, but I ended at 38, 3 points higher than when starting.
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Oct 6, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: It's been 6 days since my last infusion cycle, #5. I am now 120 days into the trial. My speech recovered some the first day, and more the 2nd and 3rd days. My swallowing has still been better the entire trial. My walking improved some again, from the low point last break. My hands do seem a little weaker than when I began the trial. Overall, my progression has certainly at least significantly slowed over 17 weeks, and I say this versus improved, since my hands seem weaker. But my ALSFRS-R is truly higher than when I began, by 4 points. My progression had been steady to where I should have been expected to lose 3 points.
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Sep 24, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: I am currently 4 days away from my next infusion cycle, #5. For the last three days, my speech has worsened to where I am having to repeat a lot more with family. It has been 24 days since my last infusion, and this speech worsening has very predictably occurred now 4 times, about 1 week before the next cycle. But, in May, I was exclusively using Proloquo2go to talk, and for months. So, I maintain an improved condition, even at the low points. My swallowing is still great, and I don't notice a cycle with it. My walking has gotten more stiff over the past week. I plan to, again, video record my current condition (talking, drinking water, walking, standing from sitting and crouching) and then again 1 week after dose cycle 5. I did this a out 6 weeks ago, at a high point, and long distance relatives were amazed at my improvements since seeing me months earlier.
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Sep 13, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Moderate
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: It has now been 13 days since my 4th cycle ended. I am 14 weeks into the trial, 100 days tomorrow, and 14 total doses. I am still BETTER than I was in May! I have always gotten very tired each infusion cycle, and have generally napped for about 3-4 hours later each day to recover from extreme tiredness and often a headache. Circumstances prevented naps on days 2 and 3. On day 3, I rushed to the airport and travelled solo. I had an all day meeting on the next day, then travelled home solo that same day, getting home about 10 pm. I was greeted by 9 out-of-state relatives ready to help us move out of our home the next day. The combination of all of this was brutal! The day after infusions, while incredibly excited about the meeting I participated in, I felt ill. My speech was very bad, walking very difficult, etc. I was very concerned. After finally settling in to our new home, one week after the last cycle, everything is back to the state it was. No further improvements, but I still maintain those realized earlier. My parents were among my visitors and stayed for over a week. Even as far back as December 2011, they could understand nothing I said. We had many conversations during their visit with my own voice. Repeating was necessary, but what a difference from 9 months ago!
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Aug 8, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: Completed cycle #3 three days ago (11 total infusions now) . Each cycle seems easier. I still had tiredness each day and a headache one day, but the short lived side effects seem less severe with each cycle. I mentioned previously that the effects plateau and then some regress slightly. The minor regression was quickly restored after only 1 infusion. My speech, after being improved for most of the time between cycles 2 and 3, regressed a few days before cycle #3, but improved again to where I am not using a computer to speak to family. For the first time since meeting the study nurse at the screening visit in May, I was able to converse without a computer, with some repeating. I've used my own voice to talk to my wife and mother 3 times on the telephone recently, for the first time in perhaps a year! My walking also seems more natural than it did after the last cycle. Overall, I have continued improving, but probably less significantly at cycle #3. My FVC/SVC has shown steady improvement as well.
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Jul 30, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: It has now been 22 days since the end of infusion cycle #2 (8 total infusions).  It is 4 days before cycle 3 begins.  As an update to the above: I have not used a cane to walk in 22 days.  I have no sense of imbalance any longer.  My gait is still abnormal and walking is slower, but is not a worrisome experience as it had been.  I have been using an iPad and Proloquo2go (AAC application) to speak for months now.  This had become necessary even for close family over the last couple months.  In the last 2.5 weeks, I have not needed to use my iPad to speak with family, until the last 2 days on occasion.  I even talked quite often throughout each day.  Swallowing liquids and solids has even improved slightly since the previous update. It has been months since I couldn't crouch without loosing balance, and had been impossible to stand up without assistance from that position for several months.  I now can again, and can even lie on the floor and stand up unassisted. I also believe my emotional lability is improved, as I can now contol laughter, without continuing for embarrassingly long durations. I have sensed improvement at each cycle so far, both during and shortly after.  Improvements seem to plateau soon after each infusion cycle and maybe even regress very slightly prior to the next.  But, overall the net result is definite overall sustained improvement each cycle.
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

Jul 7, 2011 (Started Jun 06, 2011)

  • Perceived effectiveness for ALS (Amyotrophic Lateral Sclerosis): Major
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Somewhat
Dosage: 1 other Monthly
Advice & Tips: Update after 7 Infusions (not quite 2 full cycles): Efficacy: Substantially improved swallowing, increased tongue strength (moving food), increased tongue range of motion (eliminated food from collecting in areas, firmer muscles around lips (restored partial pucker and eliminated slight drool and leaking when drinking, blew out candles for 41st birthday in late June, couldn't at 40th!), more productive cough, increased range of motion with right arm at shoulder (vertical possible, was limited to horizontal), increased range of motion with neck (was less than full leftward, now normal), increased leg strength (restored ability to rise from sitting without pushing with arms), increased arm strength (much improved ability to sit up from lying flat and to turn in bed), slight increased leg coordination and balance (no reliance on cane for balance), greater dexterity in left hand (was difficult to sign number 3 with all fingers together, now easy), intermittent clear enough speech to talk fluently without reliance on iPad. Side Effects: Tiredness and occasional slight headache within hours of infusion. Wake rested, but repeats at each cycle ad builds up. After multiple doses, tiredness lasts a couple days and slight hung-over feeling. Fasciculations worsen during dosing. Frequent smiling due to amazement over unanticipated improvement!
Cost: < $25 monthly
Side effects: Headaches, Drowsiness, Fatigue, Low red blood cell count

  • 40 helpful marks
Rthiede
29035 thumb
Rthiede
Sex: M
Data Quality: 1 star
Bulbar: none
Arms: none
Chest: none
Legs: moderate
I am: vg
I have:
ALS

See Rthiede's full ALS Phase 2 Study NP001 history

Oct 3, 2011 (Started Oct 03, 2011)

  • Perceived effectiveness for Participate in clinical trial: Slight
  • Perceived effectiveness for SURVIVAL: Slight
  • Perceived effectiveness for stop symtom progression: Slight
  • Side Effects: Mild
  • Adherence: Always
  • Burden: Not at all
Dosage: 5 mg Monthly
Advice & Tips: Side effects : mind fog, fatique Notable effects : in light of my participation in the Columbia University study I was given a neurological exam at the end of day 5 of the drip, notable increase in strenght in right arm, and while sitting increased result in leg strenght when lifting right and left leg Improved balance noted on day 8 can stand from sitting position without using arms ..could not prior to trial. In that I did not anticipate improvements so early I do not think it is placebo effect ... The mind is a powerful tool ..only time will tell
Cost: $100-199 monthly

  • 5 helpful marks
Last updated:
Showing 3 of 3 patient evaluations for ALS Phase 2 Study NP001