Safety, PK, PD, Dosing, and Efficacy of RLS-0071for the Treatment of Hospitalized Patients With Steroid-Refractory Acute Graft-versus-Host Disease

NCT06343792 | Recruiting Phase 2 FDA Drug

• 1 other identifier: RLS-0071-203
• Study Type: interventional
• Target: 66
• Locations: 3 countries
• Sites: 11

Brief Summary

This study is a Open Label Prospective Dose-Ranging Escalation and Expansion Trial to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Dosing, and Efficacy of RLS-0071 for the secondary treatment of acute Graft-versus-Host Disease (aGvHD) in hospitalized patients who are steroid-refractory.

Conditions

Steroid Refractory GVHD

Outcome Measures

Primary Outcomes (2)

• Number of participants with treatment-related adverse events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

  Day 1 to Day 180

• Overall Response Rate (ORR) of RLS-0071

  Day 1 to Day 28

Secondary Outcomes (9)

• Incidence of refractoriness (to RLS-0071 +/- ruxolitinib)

  Days 7, 14, 28, 56, and 180

• Overall corticosteroid use

  Days 7, 14, 28, 56, and 180

• Initiation of additional or alternative treatment(s) for aGvHD (including an increase in steroid dose to >2 mg/kg methylprednisolone equivalent)

  Day 1 - Day 180

• Change or shift in Stage for lower GI aGvHD, liver aGvHD, skin aGvHD, or upper GI aGvHD from baseline based on MAGIC Criteria

  Days 7, 14, 28, 56, and 180

• Attainment of Stage 0 or 1 lower GI aGvHD, liver aGvHD, skin aGvHD, or upper GI aGvHD

  Days 7, 14, 28, 56, and 180

Study Arms (7)

RLS-0071Cohort 1

EXPERIMENTAL

10 mg/kg Q8H RLS-0071 for 7 days

Drug: RLS-0071

RLS-0071 Cohort 2

EXPERIMENTAL

10 mg/kg Q8H RLS-0071 for 14 days with concurrent ruxolitinib

Drug: RLS-0071

RLS-0071 Cohort 3

EXPERIMENTAL

20 mg/kg Q8H RLS-0071 for 14 days with concurrent ruxolitinib

Drug: RLS-0071

RLS-0071 Cohort 4

EXPERIMENTAL

10 mg/kg Q8H RLS-0071 for 7 days and then 10 mg/kg QD RLS-0071 for 7 days with concurrent ruxolitinib

Drug: RLS-0071

RLS-0071 Cohort 5

EXPERIMENTAL

20 mg/kg Q8H RLS-0071 for 7 days and then 20 mg/kg QD RLS-0071 for 7 days with concurrent ruxolitinib

Drug: RLS-0071

RLS-0071 Expansion Cohort 1

EXPERIMENTAL

12 participants will receive 10 mg/kg Q8H RLS-0071 for 14 days

Drug: RLS-0071

RLS-0071 Expansion Cohort 2

EXPERIMENTAL

12 participants will receive 20 mg/kg Q8H RLS-0071 for 14 days

Drug: RLS-0071

Interventions

RLS-0071 DRUG

RLS-0071 will be administered for 7 or 14 days according to the assigned dose group.

RLS-0071 Cohort 2; RLS-0071 Cohort 3; RLS-0071 Cohort 4; RLS-0071 Cohort 5; RLS-0071 Expansion Cohort 1; RLS-0071 Expansion Cohort 2; RLS-0071Cohort 1

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female adults or adolescents (\>12 years old).
• Hospitalized with steroid-refractory aGvHD (grade II-IV) after allo-HSCT
• Anticipated hospital length-of-stay of at least 1 week from the time of RLS-0071 initiation.
• No plans to add additional GvHD treatment medications or to add, dose-adjust, or discontinue GvHD prophylactic medications during the 7-days of RLS-0071 treatment.
• Neutrophil recovery following the stem-cell transplantation, defined as blood neutrophil count \>500/mL for at least 3 consecutive measurements and not supported by growth factor supplementation
• Weight ≥40 kg and ≤ 140 kg at screening.

You may not qualify if:

• Has received more than 1 allo-HSCT
• Current, previous, or planned use of any systemic treatment in addition to or other than corticosteroids or ruxolitinib for aGvHD
• Previous failure of ruxolitinib treatment
• Uncontrolled GI infection
• Endoscopic and biopsy testing (if performed) that definitively rules out lower GI aGvHD
• Chronic GvHD
• Participants with evidence of relapsed primary disease, or participants who have been treated for relapse after the allo-HSCT was performed.
• Unresolved toxicity or complications (other than aGvHD) due to the allo-HSCT
• Any corticosteroid therapy for indications other than aGvHD at doses of methylprednisolone or equivalent \>1 mg/kg per day within 7 days of enrollment.
• Severe organ dysfunction unrelated to underlying aGvHD
• Known hypersensitivity, allergy, or anaphylactic reaction to polyethylene glycol (PEG)
• Significant liver disease that is unrelated to GvHD
• Severe kidney disease
• Currently breast feeding.
• Known pregnancy, a positive pregnancy test at screening, or lactation for WOCBP.

Sponsors & Collaborators

• ReAlta Life Sciences, Inc.: lead
• CTI Clinical Trial and Consulting Services: collaborator

Study Sites (11)

Site 1091

Duarte, California, 91010, United States

ACTIVE NOT RECRUITING

Site 1343

Los Angeles, California, 90095, United States

ACTIVE NOT RECRUITING

Site 1318

Atlanta, Georgia, 30041, United States

ACTIVE NOT RECRUITING

Site 1068

St Louis, Missouri, 63110, United States

ACTIVE NOT RECRUITING

Site 1100

Cincinnati, Ohio, 45229, United States

ACTIVE NOT RECRUITING

Site 1382

Columbus, Ohio, 43210, United States

ACTIVE NOT RECRUITING

Site 3242

Freiburg im Breisgau, 79106, Germany

RECRUITING

Site 3101

Seville, SE, 41013, Spain

RECRUITING

Site 3360

Madrid, Spain

RECRUITING

Site 3227

Salamanca, Spain

RECRUITING

Site 3101

Seville, Spain

RECRUITING

MeSH Terms

Interventions

RLS-0071

Central Study Contacts

Linda Dell, MBA

CONTACT

201-675-4044; ldell@realtals.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2024
• First Posted: April 3, 2024
• Study Start: August 31, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: November 24, 2025

Record last verified: 2025-11

Locations

DE (1); ES (4); US (6)