A Study of TCD601 (Siplizumab) in Newly Diagnosed Adult Amyotrophic Lateral Sclerosis (ALS) Patients (AURORA)

A 52-week, Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Trial of Siplizumab in Newly Diagnosed Adult Amyotrophic Lateral Sclerosis (ALS) Patients (AURORA)

The purpose of this study is to investigate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of TCD601 (siplizumab) in newly diagnosed adult ALS patients.

Study Overview

• Status: Recruiting
• Conditions: ALS
• Intervention / Treatment: Biological: TCD601

Detailed Description

The purpose of this study is to investigate how safe and tolerable the study drug siplizumab is when given to adult patients with ALS. The study will examine how the drug is distributed in the body and what effects it has on different immune cells in the blood and in the fluid surrounding the motor neurons (cerebrospinal fluid). In addition, the study will monitor the progression of ALS and look for any changes related to the disease that may occur during the study.

• Study Type: Interventional
• Enrollment (Estimated): 48
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Fredrik Juhlin; Phone Number: +46 (0)76 394 9872; Email: fredrik.juhlin@itb-med.com

Study Locations

• Sweden
  • Malmo, Sweden, 205 02
    • Recruiting
    • Skane University Hospital Malmo
    • Contact: Research Coordinator
  • Stockholm, Sweden, 171 77
    • Recruiting
    • Studieenheten Akademiskt Specialistcentrum
    • Contact: Research Coordinator
  • Umeå, Sweden, 901 85
    • Recruiting
    • Umea University Hospital
    • Contact: Research Coordinator

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Male or female patients ≥ 18 to 80 years of age.
• Diagnosis of ALS by revised El Escorial Criteria, at study entry within 24 months of first symptoms.
• Patients on existing ALS treatment must have been on a stable dose for 28 days.

Key Exclusion Criteria:

• Patient with severe systemic infections, current or within the two weeks prior to randomization.
• Subjects who, in the opinion of the investigator, are not capable of giving informed consent for the study or who are unable or unwilling to adhere to the study requirements outlined in the protocol.
• Use of other investigational products or treatment in another investigational drug study within 30 days of screening
• Pregnant or nursing (lactating) women.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment; TCD601 (siplizumab) administered with the contemporary standard of care regimen.	Biological: TCD601; Investigational Product; Other Names: siplizumab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the safety of TCD601 in adult patients with ALS	Assess the incidence of treatment-emergent Adverse Events [Safety and Tolerability]	12 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the pharmacokinetics (PK)	Siplizumab concentration over time	12 Months
Evaluation of levels of biomarkers	Neurofilament light chain levels over time	12 Months
Assessment of clinically relevant changes in laboratory measurements	Incidence of abnormal laboratory values	12 Months
Evaluation of pharmacodynamics (PD) of TCD601	Lymphocyte counts by subset over time	12 Months
Assessment of clinically relevant changes in vital signs	Incidence of abnormal vital signs	12 Months
Correlation of ALS disease status with leucocyte phenotypic profiles	The ALS disease status measured by ALSFRS and Kings staging will be compared with the leucocyte phenotypes measured by FACS analysis.	12 Months
Length of time from trial entry to tracheostomy / death	The time from study enrollment to patient death or tracheostomy will be measured.	12 Months

Collaborators and Investigators

• Sponsor: ITB-Med LLC
• Investigators: Study Director: Fredrik Julin, ITB-Med LLC

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2024
• Primary Completion (Estimated): November 1, 2029
• Study Completion (Estimated): November 1, 2029

Study Registration Dates

• First Submitted: May 6, 2024
• First Submitted That Met QC Criteria: June 10, 2024
• First Posted (Actual): June 12, 2024

Study Record Updates

• Last Update Posted (Actual): March 31, 2026
• Last Update Submitted That Met QC Criteria: March 25, 2026
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Amyotrophic Lateral Sclerosis; Neuroinflammatory
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; siplizumab
• Other Study ID Numbers: TCD601H101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No