Frontotemporal DementiaNCT05483322
An Open-Label, Expanded Access Protocol of LAM-002A in C9ORF72-Associated Frontotemporal Dementia (FTD)
A study testing LAM-002A in people with Frontotemporal Dementia, run by IRulya Therapeutics Inc..
Trial summary
This is an open-label, single subject, expanded access protocol (EAP) of the LAM-002A investigational product administered orally at 125 mg BID for 52 weeks. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Frontotemporal Dementia
- What is being tested?
- LAM-002A
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- Adult, Older adult
Who may be able to join
- Diagnosis of C9ORF72-associated FTD with documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72
- Age 18 or older
- Capable of providing informed consent at the Screening Visit and complying with study procedures throughout the study, in the Principal Investigator's opinion.
- In the case that the subject lacks the ability to provide informed consent. Informed consent will be sought from the subject's surrogate representative.
- Able to safely swallow study drug capsule at screening and throughout study. May use thickened substances to assist in swallowing drug.
Who cannot join
- Clinically significant unstable medical condition (other than FTD) that would pose a risk to the subject, according to the Principal Investigator's judgment (e.g., cardiovascular instability, systemic infection, or clinically significant laboratory abnormality or 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:
- Serum alanine aminotransferase (ALT) >5 × upper limit of normal (ULN). ii. Serum aspartate aminotransferase (AST) >5 × ULN. iii. Serum bilirubin >1.5 × ULN. c. Renal profile showing an estimated creatinine clearance (eClCR) <30 mL/minute (with eClCR to be calculated by the method at the laboratory performing the serum creatinine test).
- Presence of a neurodegenerative cognitive or motor syndrome (e.g., Alzheimer's disease, Parkinson's disease) not related to the C9ORF72 repeat expansion.
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- Presence of unstable psychiatric disease or substance abuse that would impair ability of the participant to provide informed consent, in the Principal Investigator'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.
- Use within 14 days prior to randomization or for the duration of the trial of a strong inhibitor or inducer of cytochrome P450 (CYP) 3A4 or expected requirement for chronic use of a strong inhibitor or inducer of CYP3A4 during study therapy (see Table 2 for a l list of drugs known to be strong inhibitors or inducers of CYP3A4), at the discretion of the Principal Investigator or Sponsor.
- Use within 14 days prior to randomization or for the duration of the trial of drug that is a moderate-to-strong substrate of CYP2C9 (including warfarin, tolbutamide, phenytoin, glimepiride) or expected requirement for chronic use of such drugs during study therapy, at the discretion of the Principal Investigator or Sponsor.
- Use of investigational treatments for FTD (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Screening Visit.
- Exposure at any time to any gene therapies under investigation for the treatment of FTD (off-label use or investigational).
- If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for ≥30 days after discontinuing treatment.
- Anything that would place the subject at increased risk or preclude the subject's full compliance with or completion of the study, in the Principal Investigator's opinion.
- If the subject is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
About this study
Official title: An Open-Label, Expanded Access Protocol of LAM-002A (Apilimod Dimeyslate Capsules), Administered to a Single Subject With C9ORF72-Associated Frontotemporal Dementia (FTD)
This is an open-label, single subject, expanded access protocol (EAP) of the LAM-002A investigational product administered orally at 125 mg BID for 52 weeks.
Read the detailed description
LAM-002A will be provided for expanded access for an individual patient under 21 CFR 312.310. It will be administered BID orally at a dose of 125 mg. Assessment of safety will include clinical observations and monitoring following administration.
- Conditions
- Frontotemporal Dementia
- Being tested
- LAM-002A
- Medical subject headings
- Frontotemporal Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LAM-002ADrug
Also called apilimod dimesylate
25mg capsules
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Frontotemporal DementiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- IRulya Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05483322
- Sponsor's protocol ID
- LAM-002A-FTD-EAP
- Status last verified
- Jul 2022
- First submitted
- 29 Jul 2022
- Last updated
- 2 Aug 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Expanded Access Protocol of LAM-002A (Apilimod Dimeyslate Capsules), Administered to a Single Subject With C9ORF72-Associated Frontotemporal Dementia (FTD)
- ClinicalTrials.gov ID
- NCT05483322
- Acronym / sponsor ID
- LAM-002A-FTD-EAP
- Conditions
- Frontotemporal Dementia
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- IRulya Therapeutics Inc.Industry (pharma, biotech, devices)
- First posted
- 2 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, single subject, expanded access protocol (EAP) of the LAM-002A investigational product administered orally at 125 mg BID for 52 weeks.
Detailed description
LAM-002A will be provided for expanded access for an individual patient under 21 CFR 312.310. It will be administered BID orally at a dose of 125 mg. Assessment of safety will include clinical observations and monitoring following administration.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of C9ORF72-associated FTD with documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72
- Age 18 or older
- Capable of providing informed consent at the Screening Visit and complying with study procedures throughout the study, in the Principal Investigator's opinion.
- In the case that the subject lacks the ability to provide informed consent. Informed consent will be sought from the subject's surrogate representative.
- Able to safely swallow study drug capsule at screening and throughout study. May use thickened substances to assist in swallowing drug.
Key exclusion criteria (17 listed)
- Clinically significant unstable medical condition (other than FTD) that would pose a risk to the subject, according to the Principal Investigator's judgment (e.g., cardiovascular instability, systemic infection, or clinically significant laboratory abnormality or ECG changes).
- Gastrostomy tube placement is allowed prophylactically or to supplement nutrition/hydration but may not be used for study drug administration.
- 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 Principal Investigator'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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LAM-002AAlso: apilimod dimesylate25mg capsules | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing LAM-002A in people with Frontotemporal Dementia, run by IRulya Therapeutics Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Aug 2022.