NCT06351592Toboroz1/2. fázis
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2026. szept. 11.
Rövid összefoglaló (regiszter, angolul)
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms
- Állapot (regiszter)
- Toboroz
- Fázis (regiszter)
- 1/2. fázis
- Vizsgálattípus
- Beavatkozással járó
- Résztvevők száma
- 54 (tervezett)
- Első benyújtás
- 2024. ápr. 2.
- Kezdés
- 2024. aug. 28.
- Elsődleges befejezés
- 2032. márc. 21.
- Fő szponzor
- Regeneron Pharmaceuticals
- Betegségek
- Amiotrófiás laterálszklerózis (ALS)
- NCT-szám
- NCT06351592
- EU CT-szám
- 2023-510344-20-00
Mit vállalsz
- Vizsgálat típusa: Beavatkozással járó · 1/2. fázis
- Tervezett résztvevők: 54
- 2024. aug. 28. és az elsődleges befejezés (2032. márc. 21.) között
- 22 helyszín, 9 országban
- Vizitek és vizsgálatok: kérdezd a vizsgálat csapatát, a regiszter nem sorolja fel őket
A(z) ClinicalTrials.gov regisztermezőiből összeállítva. A vizsgálat felépítését írja le.
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Korábbi kezelés
- Concurrent participation in another interventional clinical trial
- Has received treatment with tofersen within 6 months prior to screening
- NOTE: Other protocol defined inclusion / exclusion criteria apply
Más betegség
- Has dementia, as assessed by the investigator
- Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days
- Has a medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) process, CSF circulation or safety assessment, as defined in the protocol
Egyéb
- Has had a tracheostomy
- Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter
- Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study
- Was hospitalized (ie, >24 hours) for any reason other than ALS within 30 days of screening
Kiket keres a vizsgálat
- Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol
- Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position
- Body Mass Index (BMI) ≤35 kg/m2 at time of screening
- If participants are taking riluzole or edaravone, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected to remain at that dose until the end of the study
- Platelet count >50,000/microliter
- Has normal blood pressure readings, as defined in the protocol
Életkor a regiszterben: 18 év – - · Nem: nők és férfiak · Egészséges önkénteseket nem fogad
Helyszínek (22)
Helyszínek a regiszter sorrendjében. Ha a profilodba mented a városodat, távolság szerint rendeződnek.
| Helyszín | Város | Ország | Helyszín állapota |
|---|---|---|---|
| Sunshine Coast University Hospital | Birtinya | Ausztrália | Toboroz |
| Concord Repatriation General Hospital | Concord | Ausztrália | Toboroz |
| Macquarie University | Sydney | Ausztrália | Toboroz |
| KU Leuven | Leuven | Belgium | Toboroz |
| University of Alberta Hospital, Edmonton, Division of Neurology | Edmonton | Kanada | Toboroz |
| University Hospital - London Health Sciences Centre | London | Kanada | Toboroz |
| Montreal Neurological Institute and Hospital | Montreal | Kanada | Toboroz |
| Sunnybrook Research Institute | Toronto | Kanada | Toboroz |
| Universitaetsklinikum Ulm AoR | Ulm | Németország | Toboroz |
| Kyoto University Hospital | Kyoto | Japán | Toboroz |
| Toho University Omori Medical Center | Ōta-ku | Japán | Toboroz |
| Hokkaido University Hospital | Sapporo | Japán | Toboroz |
| Tokushima University Hospital | Tokushima | Japán | Toboroz |
| Seoul National University Hospital | Seoul | Dél-Korea | Toboroz |
| Hanyang University Seoul Hospital | Seoul | Dél-Korea | Toboroz |
| Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Neurologii | Krakow | Lengyelország | Toboroz |
| MTZ Clinical Research powered by Pratia | Warsaw | Lengyelország | Toboroz |
| Medical University of Silesia | Zabrze | Lengyelország | Toboroz |
| Karolinska University Hospital, Karolinska Institutet-ALS Center-Department of Clinical Neuroscience | Huddinge | Svédország | Toboroz |
| Noorlands University Hospital-Department of Neurology | Umeå | Svédország | Toboroz |
| Kaohsiung Chang Gung Memorial Hospital | Kaohsiung City | Tajvan | Toboroz |
| Taipei Veterans General Hospital | Taipei | Tajvan | Toboroz |
Ez az oldal tájékoztatás, nem orvosi tanács. Bármely vizsgálatról az orvosoddal beszélj.