Amyotrophic Lateral Sclerosis (ALS)Phase 4NCT06849115
Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations
A Phase 4 study testing L-Carnitine Injection，1000mg once daily in people with Amyotrophic Lateral Sclerosis (ALS), run by ChaodongWang at 1 site in China.
Trial summary
The study was designed as a single-center, open-label, single-arm pilot study to estimate the safety and efficacy L-carnitine in ALS patients with CHCHD10 mutations. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- L-Carnitine Injection，1000mg once daily
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteChina
- Study dates
- 18 Jul 2024to 13 Feb 2025
- Phase
- Phase 4
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, 18-80 years old
- ALS diagnosed as definite or probable as defined by the World Federation of Neurology revised El Escorial criteria
- Carrying pathogenic CHCHD10 mutations
- Capable of providing informed consent and following trial procedures
- Forced Vital Capacity (FVC) >40% of predicted value for gender, height, and age at the Screening Visit
- Subjects develop at less moderate degree of motor dysfunction, with ALS-FRS-R total scores less than 46
- Both male and female patients must agree to use adequate birth control for the whole duration of the study within 30 weeks
- Subjects are willing and expected to complete the 30-week follow up, including 3 times hospitalization.
Who cannot join
- Presence of tracheostomy
- History of known allergy to L-carnitine
- Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal
- Renal insufficiency as defined by eGFR < 60 mL/min/1.73m2
- Pregnant women or women currently breastfeeding
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
- Anything that, in the opinion of the Site Investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
Trial locations and contacts
1 site in 1 country.
About this study
The study was designed as a single-center, open-label, single-arm pilot study to estimate the safety and efficacy L-carnitine in ALS patients with CHCHD10 mutations.
Read the detailed description
The study was designed as a single-center, open-label, single-arm pilot study and was performed at the ALS inpatient clinic of the Xuanwu Hospital in Beijing, China. ALS patients with CHCHD10 mutations were recruited to participate in the clinical trial. The patients were given L-carnitine therapy administered every 8 weeks, consisting of 1g L-carnitine diluted in 500 mL of 0.9% sodium chloride solution via intravenous infusion once daily. Each treatment cycle lasted 2 weeks, repeated at 8-week intervals for a total of 3 cycles, with L-carnitine 1g is administered orally once daily during the intervals between intravenous therapies. Primary end point was the changes of ALS-FRS-R scores, and secondary end points were the changes of plasm NfL level, FVC%, weight and ALSAQ-40 scores. Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks. At each visit, a detailed neurological examination, including assessment of general nerve function as well as quantitative and qualitative evaluation of the motor system, were performed for the patients. All adverse events (AEs) encountered and any serious events were to be recorded as true. Severity was graded according to the modified WHO criteria for toxicity (http://www.regsource. com/_Adverse_Event_Reporting/_adverse_event_ reporting.html) where applicable.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- L-Carnitine Injection，1000mg once daily
- Keywords
- Amyotrophic Lateral Sclerosis, L-carnitine, CHCHD10, flail arm syndrome
- 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.
L-Carnitine Injection，1000mg once dailyDrug
All patients were given L-carnitine therapy administered every 8 weeks, consisting of 1g L-carnitine diluted in 500 mL of 0.9% sodium chloride solution via intravenous infusion once daily. Each treatment cycle lasted 2 weeks, repeated at 8-week intervals for a total of 3 cycles, with L-carnitine 1g is administered orally once daily during the intervals between intravenous therapies.
| Study group | Role | What they receive |
|---|---|---|
| L-carnitine group | Experimental | Not described Receives: Drug: L-Carnitine Injection，1000mg once daily |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of ALS-FRS-R scores
ALS-Functional Rating Scale-Revised (ALS-FRS-R) score ranges from 0 to 48, with higher scores indicating better motor function (0 = complete loss of motor function; 48 = intact/normal motor function)
Measured: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
Secondary outcomes (4)
Changes of plasm NfL level
Measured: Eligible patients were assessed at baseline and after 10, 20 and 30 weeks.
Changes of FVC%
Predicted forced vital capacity (FVC)%
Measured: Eligible patients were assessed at baseline and after 10, 20 and 30 weeks.
Changes of Weight
Measured: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
Changes of ALSAQ-40 scores
The Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) total scores range from 0 to 120. Higher scores indicate greater disease burden or poorer quality of life.
Measured: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2025
- Study start18 Jul 2024
- Primary completion19 Dec 2024
- Study completion13 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- ChaodongWang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, ChaodongWang, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06849115
- Sponsor's protocol ID
- ALS-L-carnitine
- Status last verified
- Feb 2025
- First submitted
- 23 Feb 2025
- Last updated
- 8 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations
- ClinicalTrials.gov ID
- NCT06849115
- Acronym / sponsor ID
- ALS-L-carnitine
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis, L-carnitine, CHCHD10, flail arm syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- ChaodongWangUniversity, hospital or other
- Study start
- 18 Jul 2024 (actual)
- Primary completion
- 19 Dec 2024 (actual)
- Study completion
- 13 Feb 2025 (actual)
- First posted
- 27 Feb 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.
The study was designed as a single-center, open-label, single-arm pilot study to estimate the safety and efficacy L-carnitine in ALS patients with CHCHD10 mutations.
Detailed description
The study was designed as a single-center, open-label, single-arm pilot study and was performed at the ALS inpatient clinic of the Xuanwu Hospital in Beijing, China. ALS patients with CHCHD10 mutations were recruited to participate in the clinical trial. The patients were given L-carnitine therapy administered every 8 weeks, consisting of 1g L-carnitine diluted in 500 mL of 0.9% sodium chloride solution via intravenous infusion once daily. Each treatment cycle lasted 2 weeks, repeated at 8-week intervals for a total of 3 cycles, with L-carnitine 1g is administered orally once daily during the intervals between intravenous therapies. Primary end point was the changes of ALS-FRS-R scores, and secondary end points were the changes of plasm NfL level, FVC%, weight and ALSAQ-40 scores. Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks. At each visit, a detailed neurological examination, including assessment of general nerve function as well as quantitative and qualitative evaluation of the motor system, were performed for the patients. All adverse events (AEs) encountered and any serious events were to be recorded as true. Severity was graded according to the modified WHO criteria for toxicity (http://www.regsource. com/_Adverse_Event_Reporting/_adverse_event_ reporting.html) where applicable.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| L-carnitine group | Experimental | Drug: L-Carnitine Injection，1000mg once daily |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female, 18-80 years old
- ALS diagnosed as definite or probable as defined by the World Federation of Neurology revised El Escorial criteria
- Carrying pathogenic CHCHD10 mutations
- Capable of providing informed consent and following trial procedures
- Forced Vital Capacity (FVC) >40% of predicted value for gender, height, and age at the Screening Visit
Key exclusion criteria (7 listed)
- Presence of tracheostomy
- History of known allergy to L-carnitine
- Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal
- Renal insufficiency as defined by eGFR < 60 mL/min/1.73m2
- Pregnant women or women currently breastfeeding
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 |
|---|---|---|
| L-Carnitine Injection，1000mg once dailyAll patients were given L-carnitine therapy administered every 8 weeks, consisting of 1g L-carnitine diluted in 500 mL of 0.9% sodium chloride solution via intravenous infusion once daily. Each treatment cycle lasted 2 weeks, repeated at 8-week intervals for a total of 3 cycles, with L-carnitine 1g is administered orally once daily during the intervals between intravenous therapies. | Drug | L-carnitine group |
Primary outcome
Changes of ALS-FRS-R scores
Time frame: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
ALS-Functional Rating Scale-Revised (ALS-FRS-R) score ranges from 0 to 48, with higher scores indicating better motor function (0 = complete loss of motor function; 48 = intact/normal motor function)
4 secondary outcomes
Changes of plasm NfL level
Time frame: Eligible patients were assessed at baseline and after 10, 20 and 30 weeks.
Changes of FVC%
Time frame: Eligible patients were assessed at baseline and after 10, 20 and 30 weeks.
Changes of Weight
Time frame: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
Changes of ALSAQ-40 scores
Time frame: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2025.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing L-Carnitine Injection，1000mg once daily in people with Amyotrophic Lateral Sclerosis (ALS), run by ChaodongWang at 1 site in China.
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 8 inclusion and 7 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: finished, as of the sponsor's last update on 8 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Changes of ALS-FRS-R scores (time frame: Eligible patients were assessed at baseline and after 2, 10, 12, 20 and 30 weeks.). It is what the study was designed to measure, not a finding.
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 8 Apr 2025.