Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)
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: NCT06069934 |
Expanded Access Status :
Available
First Posted : October 6, 2023
Last Update Posted : November 18, 2023
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This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials. Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube).
Each patient will be followed for 2 years with regularly scheduled visits. The screening and baseline visits will be performed in person; subsequent visits may occur in person or remotely. Recommended in-person visits will occur at Weeks 4, 12, 28, 52, 78, and end of treatment (Week 104 or early termination). If the patient is unable to complete the visits in person, these visits may also be completed remotely.
Condition or disease | Intervention/treatment |
---|---|
Amyotrophic Lateral Sclerosis | Drug: Pridopidine |
Study Type : | Expanded Access |
Expanded Access Type : | Intermediate-size Population |
Official Title: | A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2) |
- Drug: Pridopidine
Pridopidine 45 mg once daily p.o. (or via feeding tube) over a 2-week up-titration period; followed by pridopidine 45 mg twice daily p.o. (or via feeding tube) for the remainder of the treatment period (through Week 104)
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Inclusion Criteria:
- Sporadic or familial ALS.
- Patient does not qualify for clinical trials of pridopidine or other clinical trials for the treatment of ALS at the enrolling site
- Capable of providing informed consent and complying with study procedures
- Patient has established care with a physician at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP
- Life expectancy of at least 6 months.
Exclusion Criteria:
- Confirmed prolonged Fridericia-corrected QT (QTcF) interval (>450 ms for men; >470 ms for women).
- Clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, asymptomatic sustained ventricular tachycardia, or left bundle branch block.
- Known history of long QT syndrome or a first degree relative with long QT syndrome.
- Use of Nuedexta (>20 mg dextromethorphan and >10 mg quinidine twice daily); citalopram >20 mg/day; escitalopram >10 mg/day.
- Known allergy to pridopidine or any of the exipients (silicified microcrystalline cellulose, magnesium stearate).
- History of any clinically significant or unstable medical condition or laboratory abnormality that may interfere with assessment of the study objectives.
- Female who is pregnant or nursing or who plans to get pregnant during the course of the EAP
- Female of child-bearing potential or male unwilling or unable to use accepted methods of birth control.
- Use of investigational treatments for ALS (as part of participation in a clinical trial or another EAP) within 5 half-lives (if known) or 30 days (whichever is longer) prior to screening (other than pridopidine).
- Patient receives or has received any gene therapy
- Active cancer or history of cancer, except for basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years
- Patients who chose to take experimental medications and/or supplements, and for whom this is the only reason they are not eligible for trials.
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): NCT06069934
Contact: Victoria Wang | +1-857-282 ext 5374 | vjwang@mgh.harvard.edu | |
Contact: NN |
Responsible Party: | Prilenia |
ClinicalTrials.gov Identifier: | NCT06069934 |
Other Study ID Numbers: |
PL101-ALS501 |
First Posted: | October 6, 2023 Key Record Dates |
Last Update Posted: | November 18, 2023 |
Last Verified: | November 2023 |
Amyotrophic Lateral Sclerosis Pridopidine Lou Gehrig's disease |
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 |