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A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05039099
Recruitment Status : Recruiting
First Posted : September 9, 2021
Last Update Posted : March 27, 2023
Sponsor:
Information provided by (Responsible Party):
AL-S Pharma

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: AP-101 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacodynamic Markers, and Pharmacokinetics of AP-101 in Patients With Familial Amyotrophic Lateral Sclerosis (fALS) and Sporadic Amyotrophic Lateral Sclerosis (sALS)
Actual Study Start Date : September 2, 2021
Estimated Primary Completion Date : June 15, 2023
Estimated Study Completion Date : July 30, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: AP-101
AP-101 is administered by IV.
 Drug: AP-101
Participants receive AP-101 by intravenous infusion (IV).
Placebo Comparator: Placebo
Placebo is administered by IV.
 Drug: Placebo
Participants receive placebo by IV.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: From start of the study up to Week 51 ]
    An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study intervention. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. SAE is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, other situations.

  2. Number of Participants with Abnormalities in Vital Signs, Clinical Laboratory Assessments, Physical and Neurological Examinations, Electrocardiograms (ECGs) [ Time Frame: From start of the study up to Week 51 ]

Secondary Outcome Measures :
  1. Elimination half-life (t1/2) of AP-101 in Serum [ Time Frame: Predose up to Week 51 ]
  2. Area Under the Drug Concentration-Time Curve (AUC) [ Time Frame: Predose up to Week 51 ]
  3. Concentration at End of Infusion (Cat EOI) [ Time Frame: Week 24 ]
  4. Change From Baseline in AP-101 Levels in the Cerebrospinal Fluid (CSF) up to Week 24 [ Time Frame: Baseline, up to Week 24 ]
  5. Change From Baseline in Neurofilament Light Chain and Phospho-Neurofilament Heavy Chain Levels in the Cerebrospinal Fluid (CSF) up to Week 51 [ Time Frame: Baseline, up to Week 51 ]
  6. Change From Baseline in Neurofilament Light Chain and Phospho-Neurofilament Heavy Chain Levels in Plasma up to Week 51 [ Time Frame: Baseline, up to Week 51 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • All participants must adhere to contraception restrictions
  • Female participants of childbearing potential must adhere to contraception restrictions
  • Have possible, clinically probable, clinically probable-laboratory supported or definite familial or sporadic ALS in accordance with the El-Escorial criteria or who have a diagnosis of ALS as defined by the Gold Coast Criteria; progressive motor impairment documented by history or repeated clinical examination, preceded by normal motor development, and presence of upper and lower motor neuron dysfunction in at least 1 body region or lower motor neuron dysfunction in at least 2 body regions and investigations excluding other conditions
  • In familial ALS participants, a confirmed pathogenic superoxide dismutase 1 (SOD1) mutation
  • Onset of symptoms (i.e, weakness) within past 24 months prior to screening, at the time of obtaining informed consent
  • Have slow vital capacity (SVC) of greater than or equal to (> or =) 50 percentage (%) of predicted values. Participants with SVC of <50% of predicted values may be permitted to enter the open-label extension, based on the opinion of the investigator
  • Absence of bilevel positive airway pressure (BiPAP)/proportional assist ventilation (PAV) > 4 hours for symptoms attributable to ALS. Use of a CPAP for pre-existing conditions will be allowed
  • If on riluzole, must be on a stable dose.
  • If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study
  • Able to provide informed consent which includes compliance with the requirements and restrictions
  • Have venous access sufficient to allow for blood sampling
  • Have clinical laboratory test results within the normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant by the investigator

Exclusion Criteria:

  • Have participated or currently participating in another clinical trial within 12 weeks of baseline (Day 1)
  • Have undergone a tracheostomy for ALS symptoms
  • Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day for the treatment of ALS related symptoms
  • Have other causes of neuromuscular weakness
  • Have cognitive impairment, severe disease in the cardiovascular, hematological, renal system, neurodegenerative disease, pulmonary disorder, or psychiatric illness
  • Pregnant or nursing women
  • Have been exposed to any antisense treatment targeting SOD1 within 6 months of the baseline visit
  • Have undergone stem cell therapy
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05039099


Contacts
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Contact: Study Director: AL-S Pharma SA 3176517036 choruspharma@lists.lilly.com

Locations
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United States, California
UC San Diego, ACTRI Recruiting
La Jolla, California, United States, 92037
Contact    858-243-1319    jravits@ucsd.edu   
Principal Investigator: John Ravits         
Canada, Alberta
ALS clinic at the Kaye Edmonton Clinic, University of Alberta Recruiting
Edmonton, Alberta, Canada, AB T6G 1Z1
Contact    780-248-1089    wendyj@ualberta.ca   
Principal Investigator: Wendy Johnston         
Canada, Ontario
London Health Sciences Centre - Victoria Hospital Recruiting
London, Ontario, Canada, ON N6A 5W9
Contact    519-663-3597    Christen.Shoesmith@lhsc.on.ca   
Principal Investigator: Christen Shoesmith         
ALS Research Sunnybrook Health Sciences Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact       Lorne.Zinman@sunnybrook.ca   
Principal Investigator: Lorne Zinman, Dr         
Canada, Quebec
Montreal Neurological Institute and Hospital / Dr Genge Recruiting
Montréal, Quebec, Canada, H3A 2B4
Contact    514-398-8551    rami.massie@mcgill.ca   
Principal Investigator: Rami Massie, Dr         
Germany
Charité Not yet recruiting
Berlin, Germany, 13353
Contact       thomas.meyer@charite.de   
Principal Investigator: Thomas Meyer         
Hannover Medical School Recruiting
Hanover, Germany, 30625
Contact       Petri.Susanne@mh-hannover.de   
Principal Investigator: Susanne Petri         
Ulm University Hospital Recruiting
Ulm, Germany, 89081
Contact       albert.ludolph@rku.de   
Principal Investigator: Albert Ludolph         
Korea, Republic of
Hanyang University Medical Center Recruiting
Seoul, Korea, Republic of, 04763
Contact    +82-2-2290-8371    kimsh1@hanyang.ac.kr   
Principal Investigator: SeungHyun Kim         
Sweden
Studieenheten Akademiskt specialistcentrum, SLSO Recruiting
Stockholm, Sweden, 113 61
Contact    +46851771231      
Principal Investigator: Caroline Ingre         
Norrlands universitetssjukhus/ University Hospital of Northern Sweden (NUS) Recruiting
Umeå, Sweden, SE- 901 85
Contact    +46725487410      
Principal Investigator: Peter Munch Andersen         
Sponsors and Collaborators
AL-S Pharma
Investigators
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Study Director: Study Director AL-S Pharma SA
More Information
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Responsible Party: AL-S Pharma
ClinicalTrials.gov Identifier: NCT05039099    
Other Study ID Numbers: AP101-02
2020-005971-11 ( EudraCT Number )
First Posted: September 9, 2021    Key Record Dates
Last Update Posted: March 27, 2023
Last Verified: March 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by AL-S Pharma:
Familial Amyotrophic Lateral Sclerosis
Sporadic Amyotrophic Lateral Sclerosis
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases