Trial Outcomes & Findings for Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease (NCT NCT00911495)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

15 participants

Primary outcome timeframe

28 days

Results posted on

2020-05-11

Participant Flow

First subject enrolled 28 May 2009; last subject completed 6 July 2010.

Participant milestones

Participant milestones
Measure	GMI-1070 GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Overall Study STARTED	15
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GMI-1070 n=15 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Age, Continuous	28 years n=99 Participants
Sex: Female, Male Female	6 Participants n=99 Participants
Sex: Female, Male Male	9 Participants n=99 Participants
Region of Enrollment United States	15 participants n=99 Participants

PRIMARY outcome

Timeframe: 28 days

Population: All enrolled subjects were analyzed for safety.

Outcome measures

Outcome measures
Measure	GMI-1070 n=15 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Safety as Measured by the Number of Participants With Adverse Events	9 participants

SECONDARY outcome

Timeframe: 48 hours

Population: All subjects were analyzed for pharmacokinetics; one subject of the 15 enrolled was lost to follow-up.

Outcome measures

Outcome measures
Measure	GMI-1070 n=14 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Total Plasma Clearance	19.6 mL/h/kg Standard Deviation 6.42

SECONDARY outcome

Timeframe: 48 hours

Outcome measures

Outcome measures
Measure	GMI-1070 n=14 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Volume of the Central Compartment	120 mL/kg Standard Deviation 30.7

SECONDARY outcome

Timeframe: 48 hours

Outcome measures

Outcome measures
Measure	GMI-1070 n=14 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Intercompartmental Clearance	31.6 mL/h/kg Standard Deviation 17.2

SECONDARY outcome

Timeframe: 48 hours

Outcome measures

Outcome measures
Measure	GMI-1070 n=14 Participants GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Volume of the Peripheral Compartment	64.2 mL/kg Standard Deviation 19.6

OTHER_PRE_SPECIFIED outcome

Timeframe: 48 hours

As an exploratory outcome mean change in microvascular blood flow from baseline to each time point was measured. Microvascular blood flow was also measured as microFI, perfused vessel density, and RBC velocity.

Outcome measures

Outcome data not reported

Adverse Events

GMI-1070

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	GMI-1070 n=15 participants at risk GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening
Nervous system disorders Headache	26.7% 4/15 • 28 days
Blood and lymphatic system disorders Anemia	6.7% 1/15 • 28 days
Blood and lymphatic system disorders Leukocytosis	6.7% 1/15 • 28 days
Congenital, familial and genetic disorders sickle cell anemia with crisis	13.3% 2/15 • 28 days
Gastrointestinal disorders vomiting	6.7% 1/15 • 28 days
General disorders infusion site pain	6.7% 1/15 • 28 days
Metabolism and nutrition disorders hypokalemia	6.7% 1/15 • 28 days
Musculoskeletal and connective tissue disorders arthralgia	6.7% 1/15 • 28 days
Respiratory, thoracic and mediastinal disorders cough	6.7% 1/15 • 28 days
Respiratory, thoracic and mediastinal disorders oropharyngeal pain	6.7% 1/15 • 28 days
Skin and subcutaneous tissue disorders pruritus	6.7% 1/15 • 28 days

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 18007181021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60