CompletedPhase 2

Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis

United States14 participantsStarted 2021-12-23

Plain-language summary

This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of C9ORF72-associated ALS with BOTH of the following:
✓. Documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72. If there is a strong clinical suspicion for C9ALS based on C9-positive family history and El Escorial Criteria consistent with a diagnosis of ALS, clinical testing for the C9ORF72 repeat expansion may be performed with study screening labs at the discretion of the Site Investigator (SI), medical monitor, and study sponsor.
✓. Must meet possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS by revised El Escorial criteria (Brooks 2000).
✓. Age 18 or older
✓. Capable of providing informed consent at the Screening Visit and complying with study procedures throughout the study, in the SI's opinion, and at the discretion of the medical monitor and study sponsor.
✓. In the case that a participant lacks the ability to provide informed consent. Informed consent will be sought from the participant's surrogate representative.
✓. Able to safely swallow study drug capsule at screening and throughout study. May use thickened substances to assist in swallowing drug.
✓. Vital Capacity greater than and equal to 50% of predicted at the time of the Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to COVID-19 pandemic-related restrictions and with Sponsor approval, Forced Vital Capacity (FVC) measured in-person or via telemedicine.

Exclusion criteria

✕. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to the SI's judgment \[e.g., cardiovascular instability, systemic infection, or clinically significant laboratory abnormality or electrocardiogram (ECG) changes\].
✕. Gastrointestinal disease (e.g., gastric, or intestinal bypass surgery, jejunostomy tube, pancreatic enzyme insufficiency, malabsorption syndrome, symptomatic inflammatory bowel disease, chronic diarrheal illness, bowel obstruction) that might interfere with drug absorption or with interpretation of gastrointestinal AEs. Gastrostomy tube placement is allowed prophylactically or to supplement nutrition/hydration but may not be used for study drug administration.
✕. Hepatic profile showing any of the following:
✕. Presence of a neurodegenerative cognitive or motor syndrome (e.g., Alzheimer's disease, Parkinson's disease) not related to the C9ORF72 repeat expansion.
✕. Presence of unstable psychiatric disease or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
✕. Active cancer or history of cancer, except for the following: 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. Active cancer includes cancers with current disease manifestations or therapy that could adversely affect subject safety and longevity, create the potential for drug-drug interactions, or compromise the interpretation of study results.
✕. Prior solid organ transplantation.
✕. Ongoing immunosuppressive therapy including systemic or enteric corticosteroids at screening or for the duration of the trial, at the discretion of the site investigator and medical monitor.

What they're measuring

Safety of LAM-002A: occurrence of TEAEs

Timeframe: 28 Weeks

Tolerability of LAM-002A: completion of core study treatment

Timeframe: 12 Weeks

Plasma Pharmacokinetics of LAM-002A

Timeframe: 24 Weeks

CSF Pharmacokinetics of LAM-002A

Timeframe: 24 Weeks

Trial details

NCT IDNCT05163886

SponsorOrphAI Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-01-11

View on ClinicalTrials.gov More Amyotrophic Lateral Sclerosis trials