Amyotrophic Lateral Sclerosis (ALS)Phase 2FHN-ALS2NCT07138014
FHND1002 for ALS Treatment: Phase 2
A Phase 2 study testing FHND1002 100mg and FHND1002 200mg in people with Amyotrophic Lateral Sclerosis (ALS), run by Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- FHND1002 100mg and FHND1002 200mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 180 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2025to 1 Mar 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of FHND1002 Granules in Patients With Amyotrophic Lateral Sclerosis (ALS)
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:
Read more
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).
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- FHND1002 100mg, FHND1002 200mg, Placebo
- Keywords
- Amyotrophic Lateral Sclerosis (ALS), Phase II, FHND1002
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FHND1002 100mgDrug
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.
FHND1002 200mgDrug
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.
PlaceboDrug
Placebo granules identical to active intervention in appearance, taste, packaging, and administration method. Contains inert excipients (microcrystalline cellulose, lactose monohydrate) without active
| Study group | Role | What they receive |
|---|---|---|
| FHND1002 100mg | Experimental | Participants receive FHND1002 granules 100mg orally once daily for 48 weeks. Administration may occur fasting or with food. Concurrent stable ALS medications (e.g., edaravone/riluzole) are permitted if maintained ≥4 weeks prior to enrollment and unchanged during the study. Standard ALS care continues throughout. Receives: Drug: FHND1002 100mg |
| FHND1002 200mg | Experimental | Participants receive FHND1002 granules 200mg orally once daily for 48 weeks. Administration may occur fasting or with food. Pre-existing stable ALS therapies (edaravone/riluzole) are allowed if dose-stable for ≥4 weeks pre-randomization and maintained during the trial. Standard supportive care is provided. Receives: Drug: FHND1002 200mg |
| Placebo | Placebo comparator | Participants receive matching placebo granules orally once daily for 48 weeks. Administration conditions (fasting/with food) mirror active arms. Background ALS medications (edaravone/riluzole) are permitted if stabilized ≥4 weeks before enrollment and unchanged throughout the study. Standard ALS management continues. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at 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.
Measured: Baseline to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2025
- Study start1 Oct 2025 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion1 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- None (open label)
- Enrolment
- 180 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07138014
- Sponsor's protocol ID
- FHND1002-II-01
- Acronym
- FHN-ALS2
- Status last verified
- Aug 2025
- First submitted
- 15 Aug 2025
- Last updated
- 22 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Due to privacy and confidentiality concerns, individual participant data (IPD) will not be shared. The data contains sensitive health information that is protected by regulations, and sharing it ma compromise participant confidentiality.
Publications
Todd TW, Petrucelli L. Modelling amyotrophic lateral sclerosis in rodents. Nat Rev Neurosci. 2022 Apr;23(4):231-251. doi: 10.1038/s41583-022-00564-x. Epub 2022 Mar 8.
PubMed 35260846Fang MY, Markmiller S, Vu AQ, Javaherian A, Dowdle WE, Jolivet P, Bushway PJ, Castello NA, Baral A, Chan MY, Linsley JW, Linsley D, Mercola M, Finkbeiner S, Lecuyer E, Lewcock JW, Yeo GW. Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in ALS/FTD. Neuron. 2019 Sep 4;103(5):802-819.e11. doi: 10.1016/j.neuron.2019.05.048. Epub 2019 Jul 1.
PubMed 31272829Piol D, Robberechts T, Da Cruz S. Lost in local translation: TDP-43 and FUS in axonal/neuromuscular junction maintenance and dysregulation in amyotrophic lateral sclerosis. Neuron. 2023 May 3;111(9):1355-1380. doi: 10.1016/j.neuron.2023.02.028. Epub 2023 Mar 23.
PubMed 36963381Yu CH, Davidson S, Harapas CR, Hilton JB, Mlodzianoski MJ, Laohamonthonkul P, Louis C, Low RRJ, Moecking J, De Nardo D, Balka KR, Calleja DJ, Moghaddas F, Ni E, McLean CA, Samson AL, Tyebji S, Tonkin CJ, Bye CR, Turner BJ, Pepin G, Gantier MP, Rogers KL, McArthur K, Crouch PJ, Masters SL. TDP-43 Triggers Mitochondrial DNA Release via mPTP to Activate cGAS/STING in ALS. Cell. 2020 Oct 29;183(3):636-649.e18. doi: 10.1016/j.cell.2020.09.020. Epub 2020 Oct 7.
PubMed 33031745Baughn MW, Melamed Z, Lopez-Erauskin J, Beccari MS, Ling K, Zuberi A, Presa M, Gonzalo-Gil E, Maimon R, Vazquez-Sanchez S, Chaturvedi S, Bravo-Hernandez M, Taupin V, Moore S, Artates JW, Acks E, Ndayambaje IS, Agra de Almeida Quadros AR, Jafar-Nejad P, Rigo F, Bennett CF, Lutz C, Lagier-Tourenne C, Cleveland DW. Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies. Science. 2023 Mar 17;379(6637):1140-1149. doi: 10.1126/science.abq5622. Epub 2023 Mar 16.
PubMed 36927019Mead RJ, Shan N, Reiser HJ, Marshall F, Shaw PJ. Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation. Nat Rev Drug Discov. 2023 Mar;22(3):185-212. doi: 10.1038/s41573-022-00612-2. Epub 2022 Dec 21.
PubMed 36543887Watanabe H, Atsuta N, Hirakawa A, Nakamura R, Nakatochi M, Ishigaki S, Iida A, Ikegawa S, Kubo M, Yokoi D, Watanabe H, Ito M, Katsuno M, Izumi Y, Morita M, Kanai K, Taniguchi A, Aiba I, Abe K, Mizoguchi K, Oda M, Kano O, Okamoto K, Kuwabara S, Hasegawa K, Imai T, Kawata A, Aoki M, Tsuji S, Nakashima K, Kaji R, Sobue G. A rapid functional decline type of amyotrophic lateral sclerosis is linked to low expression of TTN. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):851-8. doi: 10.1136/jnnp-2015-311541. Epub 2016 Jan 8.
PubMed 26746183Xu L, Liu T, Liu L, Yao X, Chen L, Fan D, Zhan S, Wang S. Global variation in prevalence and incidence of amyotrophic lateral sclerosis: a systematic review and meta-analysis. J Neurol. 2020 Apr;267(4):944-953. doi: 10.1007/s00415-019-09652-y. Epub 2019 Dec 3.
PubMed 31797084
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of FHND1002 Granules in Patients With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT07138014
- Acronym / sponsor ID
- FHN-ALS2 / FHND1002-II-01
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis (ALS), Phase II, FHND1002
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 180 participants (estimated)
- Lead sponsor
- Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Oct 2025 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 22 Aug 2025 (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 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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| FHND1002 100mg | Experimental | Drug: FHND1002 100mgParticipants receive FHND1002 granules 100mg orally once daily for 48 weeks. Administration may occur fasting or with food. Concurrent stable ALS medications (e.g., edaravone/riluzole) are permitted if maintained ≥4 weeks prior to enrollment and unchanged during the study. Standard ALS care continues throughout. |
| FHND1002 200mg | Experimental | Drug: FHND1002 200mgParticipants receive FHND1002 granules 200mg orally once daily for 48 weeks. Administration may occur fasting or with food. Pre-existing stable ALS therapies (edaravone/riluzole) are allowed if dose-stable for ≥4 weeks pre-randomization and maintained during the trial. Standard supportive care is provided. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants receive matching placebo granules orally once daily for 48 weeks. Administration conditions (fasting/with food) mirror active arms. Background ALS medications (edaravone/riluzole) are permitted if stabilized ≥4 weeks before enrollment and unchanged throughout the study. Standard ALS management continues. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
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 |
|---|---|---|
| FHND1002 100mgInvestigational 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. | Drug | FHND1002 100mg |
| FHND1002 200mgInvestigational 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. | Drug | FHND1002 200mg |
| PlaceboPlacebo granules identical to active intervention in appearance, taste, packaging, and administration method. Contains inert excipients (microcrystalline cellulose, lactose monohydrate) without active | Drug | Placebo |
Primary outcome
Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48
Time frame: 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2025.
- Background: PubMed 35260846Todd TW, Petrucelli L. Modelling amyotrophic lateral sclerosis in rodents. Nat Rev Neurosci. 2022 Apr;23(4):231-251. doi: 10.1038/s41583-022-00564-x. Epub 2022 Mar 8.
- Background: PubMed 31272829Fang MY, Markmiller S, Vu AQ, Javaherian A, Dowdle WE, Jolivet P, Bushway PJ, Castello NA, Baral A, Chan MY, Linsley JW, Linsley D, Mercola M, Finkbeiner S, Lecuyer E, Lewcock JW, Yeo GW. Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in ALS/FTD. Neuron. 2019 Sep 4;103(5):802-819.e11. doi: 10.1016/j.neuron.2019.05.048. Epub 2019 Jul 1.
- Background: PubMed 36963381Piol D, Robberechts T, Da Cruz S. Lost in local translation: TDP-43 and FUS in axonal/neuromuscular junction maintenance and dysregulation in amyotrophic lateral sclerosis. Neuron. 2023 May 3;111(9):1355-1380. doi: 10.1016/j.neuron.2023.02.028. Epub 2023 Mar 23.
- Background: PubMed 33031745Yu CH, Davidson S, Harapas CR, Hilton JB, Mlodzianoski MJ, Laohamonthonkul P, Louis C, Low RRJ, Moecking J, De Nardo D, Balka KR, Calleja DJ, Moghaddas F, Ni E, McLean CA, Samson AL, Tyebji S, Tonkin CJ, Bye CR, Turner BJ, Pepin G, Gantier MP, Rogers KL, McArthur K, Crouch PJ, Masters SL. TDP-43 Triggers Mitochondrial DNA Release via mPTP to Activate cGAS/STING in ALS. Cell. 2020 Oct 29;183(3):636-649.e18. doi: 10.1016/j.cell.2020.09.020. Epub 2020 Oct 7.
- Background: PubMed 36927019Baughn MW, Melamed Z, Lopez-Erauskin J, Beccari MS, Ling K, Zuberi A, Presa M, Gonzalo-Gil E, Maimon R, Vazquez-Sanchez S, Chaturvedi S, Bravo-Hernandez M, Taupin V, Moore S, Artates JW, Acks E, Ndayambaje IS, Agra de Almeida Quadros AR, Jafar-Nejad P, Rigo F, Bennett CF, Lutz C, Lagier-Tourenne C, Cleveland DW. Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies. Science. 2023 Mar 17;379(6637):1140-1149. doi: 10.1126/science.abq5622. Epub 2023 Mar 16.
- Background: PubMed 36543887Mead RJ, Shan N, Reiser HJ, Marshall F, Shaw PJ. Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation. Nat Rev Drug Discov. 2023 Mar;22(3):185-212. doi: 10.1038/s41573-022-00612-2. Epub 2022 Dec 21.
- Background: PubMed 26746183Watanabe H, Atsuta N, Hirakawa A, Nakamura R, Nakatochi M, Ishigaki S, Iida A, Ikegawa S, Kubo M, Yokoi D, Watanabe H, Ito M, Katsuno M, Izumi Y, Morita M, Kanai K, Taniguchi A, Aiba I, Abe K, Mizoguchi K, Oda M, Kano O, Okamoto K, Kuwabara S, Hasegawa K, Imai T, Kawata A, Aoki M, Tsuji S, Nakashima K, Kaji R, Sobue G. A rapid functional decline type of amyotrophic lateral sclerosis is linked to low expression of TTN. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):851-8. doi: 10.1136/jnnp-2015-311541. Epub 2016 Jan 8.
- Background: PubMed 31797084Xu L, Liu T, Liu L, Yao X, Chen L, Fan D, Zhan S, Wang S. Global variation in prevalence and incidence of amyotrophic lateral sclerosis: a systematic review and meta-analysis. J Neurol. 2020 Apr;267(4):944-953. doi: 10.1007/s00415-019-09652-y. Epub 2019 Dec 3.
- Description the protocol of the studyLink provided by the sponsor.
- Individual participant dataSharing planned: No. Due to privacy and confidentiality concerns, individual participant data (IPD) will not be shared. The data contains sensitive health information that is protected by regulations, and sharing it ma compromise participant confidentiality.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FHND1002 100mg and FHND1002 200mg in people with Amyotrophic Lateral Sclerosis (ALS), run by Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd..
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 9 inclusion and 9 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: opening soon, as of the sponsor's last update on 22 Aug 2025. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dongsheng Fan, MD, PhD, Professor (010-82265159, dsfan@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48 (time frame: Baseline to Week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07138014, which also lists 8 linked publications.
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 22 Aug 2025.