Exploratory Safety Study of ALD518 in Subjects With Steroid-refractory Acute Graft Versus Host Disease (GVHD) (ALD518-010)

• ClinicalTrials.gov Identifier: NCT01530256
• Recruitment Status: Terminated
• First Posted: February 9, 2012
• Results First Posted: February 17, 2021
• Last Update Posted: February 17, 2021
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

Study Description

Brief Summary:

The purpose of this study is to determine the safe and tolerable, biologically active, and potentially effective doses(s) of ALD518 in subjects with acute GVHD, who have failed to respond to glucocorticosteroids, for further investigation in Part B.

Condition or disease	Intervention/treatment	Phase
Acute GvHD	Biological: ALD518	Phase 1; Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 3 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Exploratory Phase 1/2 Clinical Trial Evaluating ALD518 in Subjects With Glucocorticoid-Refractory Acute Graft vs. Host Disease (GVHD) After Allogeneic Hematopoietic Stem Cell Transplant (HSCT)
• Actual Study Start Date: March 2012
• Actual Primary Completion Date: March 2013
• Actual Study Completion Date: March 2013

Arms and Interventions

Arm	Intervention/treatment
Experimental: ALD518	Biological: ALD518; 160 mg IV q 4 weeks for 4 doses; Biological: ALD518; 320 mg IV q 4 weeks for 4 doses; Biological: ALD518; 640 mg IV q 4 weeks for 2 doses

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Adverse Events, Including Infections and Loss of Engraftment [ Time Frame: Through Week 24 ]
2. Number of Participants With Acute GVHD Response at Week 4 [ Time Frame: Week 4 ]
   Includes calculation of both complete and partial response. Complete response = functional grade of 0 for the GVHD grading in all evaluable organs. Partial response = improvement by one or more functional grades in one or more organs involved with GVHD symptoms without progression in others, or a very good partial response which is a partial response plus all of the following: (1) skin - active erythematous rash involving less than 25% of body surface, (2) liver - total bilirubin <2 mg/dL or a 25% reduction from baseline (if >2 mg/dL), (3) gut - greater than or equal to 75% reduction in daily stool volume.

Secondary Outcome Measures :

1. Number of Participants With Relapse of Underlying Malignant Disease [ Time Frame: Through Week 24 ]
2. Number of Participants With Overall Survival [ Time Frame: Through Week 24 ]
3. Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score [ Time Frame: Up to 20 weeks ]
   The FACT-BMT assesses bone marrow transplant related concerns. The total score is the sum of sub-scale scores for 5 domains: Physical Well-Being, Social/Family Well-Being, Emotional Well-Being, Functional Well-Being, and Bone Marrow Transplantation Subscale. Within each domain, a 5-point Likert-type scale (from 0-4) is used to measure the responses for each question. After taking into account reverse scores for questions constructed in a negative form, the subscale score for each domain is calculated by multiplying the sum of the item scores by the number of items in the subscale, then dividing by the number of items answered. The final score for FACT-BMT ranges from 0 to 148. Higher scores indicate better quality of life.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Glucocorticosteroid refractory acute GVHD Grade 2-4
• Prior corticosteroid therapy at > 1.0mg/kg methylprednisolone or equivalent for minimum of 3 and maximum of 14 days
• Karnofsky Performance Satus Scale ≥ 50%
• Adequate laboratory testing at screening

Exclusion Criteria:

• Subjects having Stage 1 skin acute GVHD
• Subjects with Stage 3 or 4 liver GVHD
• Prior EBV associated malignancy
• Prior or current Hepatitis B or C
• Prior or active tuberculosis

Contacts and Locations

Locations

United States, California

City of Hope

Duarte, California, United States, 91010

United States, Illinois

Loyola Medical Center

Maywood, Illinois, United States, 60153

United States, Missouri

Washington University

Saint Louis, Missouri, United States, 63110

United States, Ohio

The Ohio State University

Columbus, Ohio, United States, 43210

United States, Virginia

Virginia Commonwealth University

Richmond, Virginia, United States, 23298

Sponsors and Collaborators

CSL Behring

Investigators

• Study Director: Javier San Martin, MD FRCP; Alder Biopharmaceuticals, Inc.

More Information

• Responsible Party: CSL Behring
• ClinicalTrials.gov Identifier: NCT01530256
• Other Study ID Numbers: ALD518-CLIN-010
• First Posted: February 9, 2012
• Results First Posted: February 17, 2021
• Last Update Posted: February 17, 2021
• Last Verified: February 2021

Keywords provided by CSL Behring:

allograph; steroid refractory