New Trial – Phase II Umbilical-Cord Stem Cells for Amyotrophic Lateral Sclerosis

Trial at a glance

• Sponsor: Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.
• Phase/Status: Phase 2; not yet recruiting.
• Indication: Amyotrophic lateral sclerosis in approximately 150 participants.
• Arms/Dosing: ALT001 1.0 μg/kg: allogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex, supplied as 130 μg/vial lyophilized powder, reconstituted and administered by intravenous infusion at 1.0 μg/kg body weight. ALT001 2.0 μg/kg: the same protein complex and formulation, reconstituted and administered by intravenous infusion at 2.0 μg/kg body weight. During the open-label extension period, the active treatment groups will continue their original doses under blinded conditions.
• Control: Matching placebo containing no active ingredient and identical in appearance, packaging, labeling and post-reconstitution infusion presentation to ALT001.
• Endpoints: Primary: change from baseline in ALSFRS-R total score through Week 24. Secondary: weekly rate of change in ALSFRS-R total score from baseline through Week 24 and from baseline through Week 48.
• Timing: Estimated start: September 1, 2026; estimated primary completion: January 1, 2028; estimated study completion: December 31, 2028.

Trial Summary

ClinicalTrials.gov lists the following summary for the linked study.

ALT001 is an allogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex under investigation for the treatment of amyotrophic lateral sclerosis (ALS). This is a multicenter, randomized, double-blind, placebo-controlled, Phase 2 dose-ranging study in patients with ALS.

Approximately 150 participants will be randomized 1:1:1 to receive ALT001 1.0 μg/kg, ALT001 2.0 μg/kg, or matching placebo by intravenous infusion during the 24-week double-blind (DB) treatment period. During the OLE period, the active treatment groups will continue to receive their original dose under blinded conditions, whereas the placebo group will undergo re-randomization in a 1:1 ratio to receive 1.0 or 2.0 μg/kg ALT001.

The primary objective is to evaluate the dose-response relationship and efficacy of ALT001 at 1.0 and 2.0 μg/kg and to determine the recommended dose for subsequent studies. Secondary objectives include assessment of biomarkers related to efficacy and exploration of changes in cytokines, immune function, proteomics, and immunogenicity (anti-drug antibodies, ADA), as well as evaluation of the safety of ALT001.

• Contact: Pengfei Zhang, zhangpengfei@darwincell.net, +86-13911241040
• Contact: Yilong Wang, MD, yilong528@gmail.com, +86 139 1166 6571

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