Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
Amyotrophic Lateral Sclerosis (ALS), Phase II, FHND1002
Brief summary
This is a Phase II clinical trial evaluating the effectiveness and safety of an investigational drug, FHND1002 granules, in adults with Amyotrophic Lateral Sclerosis (ALS). The main goals are: To determine if FHND1002 can slow the progression of ALS compared to a placebo. To assess the safety and tolerability of two different doses of FHND1002 (100mg and 200mg) in ALS patients. Approximately 180 participants will be randomly assigned (like flipping a coin) to one of three groups: FHND1002 100mg once daily FHND1002 200mg once daily Placebo (an inactive substance) once daily Assignment will consider disease severity (ALSFRS-R score) and where symptoms started (Limb vs. Bulbar). Participants can continue taking stable doses of approved ALS medications (like riluzole or edaravone) or be on no medication. The study consists of: A Screening Period (up to 4 weeks). A Double-Blind Treatment Period (48 weeks). During the 48-week treatment period: Participants will take their assigned granules orally once daily (with or without food). They will attend clinic visits at Weeks 2, 4, 12, 24, 36, and 48 for safety checks. Effectiveness will be measured at Weeks 12, 24, 36, and 48 using standard ALS assessments, including the ALS Functional Rating Scale-Revised (ALSFRS-R), breathing tests (FVC%), and quality of life/questionnaires (ROADS, ALSAQ-5).
Interventions
DRUGFHND1002 100mg
Investigational oral granules containing 100mg FHND1002 (chemical entity: 3-n-butylphthalide derivative). Administered once daily for 48 weeks. Granules are packaged in unit-dose sachets with identical appearance across all study arms. Participants dissolve contents in water prior to ingestion. Stability testing confirms compatibility with fasting or fed conditions.
DRUGFHND1002 200mg
Investigational oral granules containing 200mg FHND1002 (3-n-butylphthalide derivative). Delivered as two 100mg sachets or one 200mg sachet to maintain blinding. Daily dosing regimen for 48 weeks. Granule composition matches 100mg formulation in excipients and organoleptic properties.
DRUGPlacebo
Placebo granules identical to active intervention in appearance, taste, packaging, and administration method. Contains inert excipients (microcrystalline cellulose, lactose monohydrate) without active
Sponsors
Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants are randomized in a 1:1:1 ratio to three parallel arms: * Arm 1: FHND1002 100mg granules + standard ALS care * Arm 2: FHND1002 200mg granules + standard ALS care * Arm 3: Matching placebo granules + standard ALS care Stratification factors: 1) Baseline ALSFRS-R (≥40 vs \<40); 2) Site of onset (limb vs bulbar). Double-blind treatment period: 48 weeks.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must be ≥18 years of age at the time of signing the informed consent form. * Subjects must have a diagnosis of Amyotrophic Lateral Sclerosis (ALS) meeting the revised El Escorial World Federation of Neurology criteria categories including Clinically Definite ALS, Clinically Probable ALS, Laboratory-supported Probable ALS, or Clinically Possible ALS. * Subjects must demonstrate a documented mean monthly decline of ≥0.5 points in the ALS Functional Rating Scale-Revised (ALSFRS-R) score since initial diagnosis. * During screening, subjects must exhibit a percent predicted forced vital capacity (FVC%) ≥70%; note that use of non-invasive ventilation (NIV) is exclusionary. * Disease duration must be ≤2 years calculated from the onset of the first ALS-related symptom. * At screening, subjects must have an ALSFRS-R total score ≥30 with a swallowing function subscore ≥2 and all respiratory-related subscores at 4 points. * Subjects must have a Body Mass Index (BMI) ≥18.5 kg/m². * Subjects may or may not be receiving stable therapeutic doses of approved ALS medications (e.g., edaravone, riluzole) prior to enrollment, provided any existing regimen remains unchanged throughout the study. * Subjects must demonstrate ability to understand study procedures, willingness to comply, voluntary participation, and provide signed informed consent.
Exclusion criteria
* Subjects with coexisting neurological disorders that may mimic ALS symptoms or interfere with efficacy assessment (e.g., cervical/lumbar spondylosis, dementia, history of seizures except childhood febrile seizures) will be excluded. * Subjects exhibiting motor conduction block or sensory nerve conduction abnormalities on electromyography (EMG) testing will be excluded. * Subjects with any history of spinal surgery within 3 months prior to screening will be excluded. * Subjects showing clinically significant laboratory abnormalities at screening including aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>1.5 × upper limit of normal (ULN), or serum creatinine (Scr) \> ULN will be excluded. * Subjects with severe/uncontrolled cardiac, hepatic, renal, hematologic, or neoplastic diseases, or active severe psychiatric disorders will be excluded. * Exclusion applies to pregnant or lactating women, and subjects planning pregnancy during the study or within 3 months post-treatment; participants of childbearing potential unwilling to use highly effective contraception throughout the study and for 3 months after last dose are excluded. * Subjects with known or suspected hypersensitivity to FHND1002 or its excipients will be excluded. * Subjects who have participated in another investigational drug/device trial within 1 month prior to screening will be excluded. * Subjects deemed by the Investigator to have any condition compromising safety, data integrity, or study compliance will be excluded.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48 | Baseline to Week 48 | The difference in least-squares mean (LSMean) change from baseline to Week 48 in ALSFRS-R total score between FHND1002 treatment groups (combined or individual doses) and placebo. Missing data due to early discontinuation will be handled using multiple imputation based on observed data from the same treatment group. Analysis will use a repeated measures mixed-effects model (MMRM) adjusted for baseline ALSFRS-R score, site of onset (limb vs. bulbar), treatment group, visit, and treatment-by-visit interaction, with subject as a random effect. |
Contacts
Primary ContactDongsheng Fan, MD, PhD, Professor
Outcome results
None listed