Study Purpose:
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
AP-101, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Study Director, AL-S Pharma SA
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Montreal Neurological Institute and Hospital
Study Director: AL-S Pharma SA / email hidden; JavaScript is required / 3176517036
Canada
Study Sponsor:
AL-S Pharma
Participant Duration:
40 weeks for the double blind phase, 64 weeks including the Open Label Extension
Estimated Enrollment:
63
Estimated Study Start Date:
09 / 02 / 2021
Estimated Study Completion Date:
07 / 30 / 2023
Posting Last Modified Date:
03 / 27 / 2023
Date Study Added to neals.org:
09 / 09 / 2021
AP-101 is a monoclonal antibody that binds to misfolded forms of SOD1, which are considered to be the most likely mechanism in ALS caused by SOD1 mutations, and which are also present in the CSF of sporadic ALS. The assumed mechanism of AP-101 is its specific binding to such misfolded SOD1 and its subsequent removal by phagocytosis, while not binding to the native form of SOD1 that is essential for human biological functions. AP-101 is administered by intravenous infusion.
In transgenic preclinical models, AP-101 generated highly significant therapeutic effects and the SAD study showed a very beneficial safety profile of AP-101 in humans at all dose levels tested.
In the absence of any disease-modifying treatment modality, we believe that the current AP-101 phase 2 study offers a valuable treatment option for ALS patients by its multiple dosing during 6 months with an additional 6 months open label extension.
The study is recruiting patients now, and further information on the active sites are available at clinicaltrials.gov identifier NCT05039099.
Michael Salzmann, CEO & Dr. Angela Genge, Global PI & Head of Clinical Advisory Board
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
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
UC San Diego, ACTRI | Recruiting
/ 858-243-1319 / email hidden; JavaScript is required
Principal Investigator : John Ravits
La Jolla, California
92037
United States
ALS clinic at the Kaye Edmonton Clinic, University of Alberta | Recruiting
/ 780-248-1089 / email hidden; JavaScript is required
Principal Investigator : Wendy Johnston
Edmonton, Alberta
AB T6G 1Z1
Canada
London Health Sciences Centre - Victoria Hospital | Recruiting
/ 519-663-3597 / email hidden; JavaScript is required
Principal Investigator : Christen Shoesmith
London, Ontario
ON N6A 5W9
Canada
ALS Research Sunnybrook Health Sciences Centre | Recruiting
/ email hidden; JavaScript is required
Principal Investigator : Lorne Zinman, Dr
Toronto, Ontario
M4N 3M5
Canada
Montreal Neurological Institute and Hospital / Dr Genge | Recruiting
/ 514-398-8551 / email hidden; JavaScript is required
Principal Investigator : Rami Massie, Dr
Montréal, Quebec
H3A 2B4
Canada
Charité | Not yet recruiting
/ email hidden; JavaScript is required
Principal Investigator : Thomas Meyer
Berlin 13353
Germany
Hannover Medical School | Recruiting
/ email hidden; JavaScript is required
Principal Investigator : Susanne Petri
Hanover 30625
Germany
Ulm University Hospital | Recruiting
/ email hidden; JavaScript is required
Principal Investigator : Albert Ludolph
Ulm 89081
Germany
Hanyang University Medical Center | Recruiting
/ +82-2-2290-8371 / email hidden; JavaScript is required
Principal Investigator : SeungHyun Kim
Seoul 04763
Korea, Republic of
Studieenheten Akademiskt specialistcentrum, SLSO | Recruiting
/ +46851771231
Principal Investigator : Caroline Ingre
Stockholm 113 61
Sweden
Norrlands universitetssjukhus/ University Hospital of Northern Sweden (NUS) | Recruiting
/ +46725487410
Principal Investigator : Peter Munch Andersen
Umeå SE- 901 85
Sweden