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Amyotrophic Lateral SclerosisPhase 1NCT05949294

Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)

A Phase 1 study testing ARO-SOD1 Injection in people with Amyotrophic Lateral Sclerosis, run by Arrowhead Pharmaceuticals.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive single ascending doses of ARO-SOD1 administered by intrathecal (IT) infusion. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
ARO-SOD1 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
Jan 2024to Mar 2025
Phase
Phase 1
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of ALS based on source-verifiable medical record and meets the Gold Coast Criteria
  • Pathogenic or likely pathogenic SOD1 mutation based on source-verifiable medical records or genetic testing during Screening
  • Slow Vital Capacity (SVC) ≥ 50% of predicted value adjusted for sex, age, and height (from a sitting position) at Screening
  • Body Mass Index (BMI) ≥ 18.0 kg/m2 at Screening
  • Able to complete at least 6 months of follow-up
  • If taking any medication or supplement to treat ALS or ALS symptoms, must be on stable dose for ≥ 4 weeks prior to Day 1 and expected to remain at that dose until final study visit and not expected to start these medications after the first dose of ARO-SOD1
  • Able and willing to provide written informed consent and to comply with all study assessments
  • Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Males must not donate sperm and females must not donate eggs during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later.

Who cannot join

  • Current or anticipated need of a diaphragm pacing system (DPS) during the study
  • Participants using tofersen can be enrolled only after a washout period of approximately 20 weeks from the last tofersen dose to the first planned dose of ARO-SOD1
  • History of having received stem cell therapy for ALS treatment
  • Any current or anticipated contraindications to lumbar puncture (LP)
  • The presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter
  • Human immunodeficiency virus (HIV), seropositive for hepatitis B virus (HBV), seropositive for hepatitis C virus (HCV)
Show all 11Show fewer
  • Uncontrolled hypertension
  • Severe cardiovascular disease
  • History of drug abuse or alcoholism within 6 months of study enrollment
  • Inability to comply with study requirements
  • Note: additional inclusion/exclusion criteria may apply per protocol

About this study

Official title: A Phase 1 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ARO-SOD1 in Adult Patients With Amyotrophic Lateral Sclerosis Harboring a Superoxide Dismutase-1 Mutation Considered to be Causative of Amyotrophic Lateral Sclerosis

In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive single ascending doses of ARO-SOD1 administered by intrathecal (IT) infusion. The study is primarily intended to evaluate safety, but will also evaluate the effect of ARO-SOD1 on SOD1 cerebrospinal fluid (CSF) levels as a biomarker of pharmacodynamic (PD) effect, therefore lumbar punctures will be required at timepoints throughout the study. After each participant has completed their individual final visit, participants whose SOD1 CSF levels have recovered to a satisfactory level may rescreen and enroll into higher dose cohorts; or if unable or unwilling to rescreen may enroll into an open-label study to be added by amendment when supported by nonclinical data for multidose administration.

Being tested
ARO-SOD1 Injection
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.

  • ARO-SOD1 InjectionDrug

    single doses of ARO-SOD1 Injection by IT infusion

Study groups in NCT05949294
Study groupRoleWhat they receive
ARO-SOD1ExperimentalARO-SOD1 Injection
Receives: Drug: ARO-SOD1 Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

    Measured: From first dose of study drug through the end of study (EOS; up to 168 days)

Secondary outcomes (15)

  1. Change Over Time from Baseline in CSF SOD1 Protein Levels

    Measured: Pre-dose on Day 1 (Baseline) through EOS (up to 168 days)

  2. Change Over Time from Baseline in Plasma Levels of Neurofilament Light Chain (NfL)

    Measured: Pre-dose on Day 1 (Baseline) through EOS (up to 168 days)

  3. Pharmacokinetics (PK) of ARO-SOD1: Maximum Observed Plasma Concentration (Cmax)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  4. PK of ARO-SOD1: Time to Reach Maximum Observed Plasma Concentration (Tmax)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  5. PK of ARO-SOD1: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  6. PK of ARO-SOD1: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  7. PK of ARO-SOD1: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  8. PK of ARO-SOD1: Terminal Elimination Half-Life (t1/2)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  9. PK of ARO-SOD1: Apparent Systemic Clearance (CL/F)

    Measured: Days 1, 2, 15, 29, 57, 85 and EOS (Day 168): Pre-dose, post-dose at 1, 3, 5, 7, 12 and 24 hours

  10. PK of ARO-SOD1: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae)

    Measured: Pre-dose through 24 hours post-dose

  11. PK of ARO-SOD1: Percentage of Administered Drug Recovered in Urine Over 0-24 Hours

    Measured: Pre-dose through 24 hours post-dose

  12. PK of ARO-SOD1: Renal Clearance (CLr)

    Measured: Pre-dose through 24 hours post-dose

  13. Change From Baseline in Total Protein in CSF

    Measured: Pre-dose on Day 1 (Baseline) through EOS (up to 168 days)

  14. Change From Baseline in Glucose in CSF

    Measured: Pre-dose on Day 1 (Baseline) through EOS (up to 168 days)

  15. Change From Baseline in Cell Count in CSF

    Measured: Pre-dose on Day 1 (Baseline) through EOS (up to 168 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jul 2023
  2. Study startJan 2024 (estimated)
  3. Primary completionMar 2025 (estimated)
  4. Study completionMar 2025 (estimated)

Why it stopped: Company business decision

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05949294
Sponsor's protocol ID
AROSOD1-1001
Other IDs
2023-509032-26, 2023
Status last verified
Apr 2024
First submitted
8 Jul 2023
Last updated
17 Apr 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing ARO-SOD1 Injection in people with Amyotrophic Lateral Sclerosis, run by Arrowhead Pharmaceuticals.

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 11 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: withdrawn, as of the sponsor's last update on 17 Apr 2024. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 was withdrawn before enrolling participants, so there are no results.

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?

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) (time frame: From first dose of study drug through the end of study (EOS; up to 168 days)). 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 NCT05949294.

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 17 Apr 2024.

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