Was reading through the criteria for inclusion and exclusion in the trial and it's clear the 'Goldilocks' zone for candidates is quite limited, so I think it's understandable that without a lot of luck, recruitment could be quite staggered. Some of the reasons for exclusion could be removed given a few months of abstinence from certain substances, so maybe some who are keen to participate have to wait for the all-clear period.
See details below.
There appear to be 11 centers actively recruiting now so as is that would only require 4 per facility, although things often don't work out with that even symmetry.
Anyway, currently waiting for the hopefully positive primate results, I think they will bring a broader interest base to ATL-1102, and hence a slightly greater interest in share ownership, as the term platform is enhanced by official FDA safety approval, for everything ATL-1102 (within dose parameters).
These things can discretely leak, not necessarily from the company, creating increased share volumes, and I would like to see people in the not-too-distant future complaining about insider trading, lol.
You know things are going well when people complain about that haha
And, Merry Christmas and a Happy New Year to all.
Description
Key Inclusion Criteria:
- Has a clinical diagnosis of DMD confirmed by validated genetic testing
- Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening.
- Male aged 10 to less than 18 years, at the time of Screening.
- Body weight of at least 25 kg at Screening.
- If receiving corticosteroid therapy, therapy was initiated at least six months prior to the baseline visit and a stable daily dose for at least 3 months prior to baseline
- Participant has a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score ≥2.
- Able to perform spirometry and has sufficient Respiratory function defined as reproducible percent predicted FVC ≥50%.
- Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) ≥45% by echocardiogram and if receiving cardiac medication, must be currently on a stable regimen and doses of cardiac therapy (at least 3 months prior to baseline Day 1)
- Participant and their parent/guardian/carer are willing and able to comply with scheduled visits, study medication administration and study procedures.
Key Exclusion Criteria:
- Participation in another clinical trial (non-interventional) or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1.
- Exposure to more than 3 investigational products within the 12 months prior to Day 1.
- History of clinically significant bleeding or coagulation abnormalities or clinically significant abnormal coagulation parameters.
- Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1
- Any evidence of clinically significant structural or functional heart abnormality (cardiomyopathy that is managed by ACEi or beta blockers is acceptable provided the LVEF inclusion criterion is met).
- Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening.
- Evidence of renal impairment and/or cystatin C >1.4 mg/L.
- Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period.
- Asthma (if requiring regular medication), bronchitis/chronic obstructive pulmonary disease (COPD), bronchiectasis, emphysema, pneumonia or the presence of any non-DMD respiratory illness that affects PEF and FVC or other respiratory measures.
- Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night time NIV is permitted).
- Chronic use (daily intake >14 days), within one month of Day 1, of beta-2 agonists or any use of other bronchodilating medication (e.g., inhaled steroids, sympathomimetics, anticholinergics).
- Used carnitine, creatine, glutamine, oxatomide, idebenone or other forms of coenzyme Q10 or vitamin E or any other nutritional or antioxidant supplements or herbal medicines or anabolic steroids other than standard corticosteroids or puberty testosterone supplementation within 4 weeks of Day 1.
- Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function
(20min delay)
