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1 for Amyotrophic Lateral Sclerosis

Phase 1 & 2
Waitlist Available
Led By Robert Ferrante, PhD MSc
Research Sponsored by US Department of Veterans Affairs
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.

Eligible Conditions
  • Amyotrophic Lateral Sclerosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2017 Phase 4 trial • 88 Patients • NCT01257737
22%
Hyperammonaemia
16%
Upper respiratory tract infection
13%
Vomiting
13%
Headache
9%
Ammonia increased
7%
Diarrhoea
7%
Ear infection
7%
Amino acid level decreased
7%
Cough
7%
Amino acid level increased
7%
Pyrexia
4%
Dehydration
4%
Nausea
4%
Oropharyngeal pain
2%
Gastroenteritis
2%
Influenza
2%
Hypokalaemia
2%
Bronchitis
2%
Sinusitis
2%
Pain
2%
Dizziness
100%
80%
60%
40%
20%
0%
Study treatment Arm
HPN-100- Pediatric
HPN-100 - Adult

Trial Design

1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
ALS patient
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Phenylbutyric acid
FDA approved

Find a Location

Who is running the clinical trial?

US Department of Veterans AffairsLead Sponsor
872 Previous Clinical Trials
497,800 Total Patients Enrolled
5 Trials studying Amyotrophic Lateral Sclerosis
32,298 Patients Enrolled for Amyotrophic Lateral Sclerosis
Muscular Dystrophy AssociationOTHER
37 Previous Clinical Trials
60,711 Total Patients Enrolled
10 Trials studying Amyotrophic Lateral Sclerosis
16,459 Patients Enrolled for Amyotrophic Lateral Sclerosis
Robert Ferrante, PhD MScPrincipal InvestigatorEdith Nourse Rogers Memorial Veterans Hospital, Bedford
~2 spots leftby Sep 2025
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