Trial Outcomes & Findings for Exploratory Safety Study of ALD518 in Subjects With Steroid-refractory Acute Graft Versus Host Disease (GVHD) (NCT NCT01530256)

NCT ID: NCT01530256

Last Updated: 2021-02-17

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: Through Week 24
• Results posted on: 2021-02-17

Participant Flow

The trial was terminated after only 3 participants received the 160 mg dose.

Participant milestones

Measure	ALD518 ALD518: 160 mg intravenous (IV) once every (q) 4 weeks for 4 doses
Overall Study STARTED	3
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	ALD518 ALD518: 160 mg intravenous (IV) once every (q) 4 weeks for 4 doses
Overall Study Death	2

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	ALD518 n=3 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Through Week 24

Outcome measures

Measure	ALD518 n=3 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Number of Participants With Adverse Events, Including Infections and Loss of Engraftment	3 participants

PRIMARY outcome

Timeframe: Week 4

Population: 2 of 3 participants died before Week 4 so could not be assessed

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.

Outcome measures

Measure	ALD518 n=1 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Number of Participants With Acute GVHD Response at Week 4	0 participants

SECONDARY outcome

Timeframe: Through Week 24

Outcome measures

Measure	ALD518 n=3 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Number of Participants With Relapse of Underlying Malignant Disease	0 participants

SECONDARY outcome

Timeframe: Through Week 24

Outcome measures

Measure	ALD518 n=3 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Number of Participants With Overall Survival	1 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	ALD518 n=2 Participants ALD518: 160 mg IV q 4 weeks for 4 doses
Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Participant 1 (Day 0)	93 score on a scale
Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Participant 1 (Week 4)	96 score on a scale
Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Participant 1 (Week 12)	102 score on a scale
Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Participant 1 (Week 20)	113 score on a scale
Change in Patient Reported Outcomes as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Participant 2 (Day 0)	55 score on a scale

Adverse Events

ALD518

Serious events: 3 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Measure	ALD518 n=3 participants at risk ALD518: 160 mg IV q 4 weeks for 4 doses
Renal and urinary disorders Renal failure acute	66.7% 2/3 • Number of events 3 • Up to death or termination of study (approximately 139 days).
Infections and infestations Sepsis	33.3% 1/3 • Number of events 2 • Up to death or termination of study (approximately 139 days).
Eye disorders Retinal detachment	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Infections and infestations Sinusitis	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Infections and infestations Fungal infection	33.3% 1/3 • Number of events 2 • Up to death or termination of study (approximately 139 days).

Other adverse events

Measure	ALD518 n=3 participants at risk ALD518: 160 mg IV q 4 weeks for 4 doses
Investigations Activated partial thromboplastin time prolonged	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Blood bilirubin increased	66.7% 2/3 • Number of events 3 • Up to death or termination of study (approximately 139 days).
Respiratory, thoracic and mediastinal disorders Dyspnoea	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Blood creatinine increased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Vascular disorders Hypotension	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Psychiatric disorders Confusional state	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations International normalized ratio increased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Metabolism and nutrition disorders Hyponatraemia	33.3% 1/3 • Number of events 2 • Up to death or termination of study (approximately 139 days).
Infections and infestations Cytomegalovirus viraemia	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Alanine aminotransferase increased	33.3% 1/3 • Number of events 2 • Up to death or termination of study (approximately 139 days).
Investigations Blood alkaline phosphatase increased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Aspartate aminotransferase increased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Platelet count decreased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Metabolism and nutrition disorders Hypertriglyceridaemia	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Investigations Blood cholesterol increased	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Metabolism and nutrition disorders Oedema peripheral	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Respiratory, thoracic and mediastinal disorders Cough	33.3% 1/3 • Number of events 2 • Up to death or termination of study (approximately 139 days).
Metabolism and nutrition disorders Hyperuricaemia	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Metabolism and nutrition disorders Decreased appetite	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
General disorders Fatigue	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
General disorders Pyrexia	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Psychiatric disorders Anxiety	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
Gastrointestinal disorders Gastric haemorrhage	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).
General disorders Pain	33.3% 1/3 • Number of events 1 • Up to death or termination of study (approximately 139 days).

Additional Information

Study Director

CSL Behring

Phone: 610-878-4000

Email: clinicaltrials@cslbehring.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place