Trial Outcomes & Findings for Clinical Trial in Patients Diagnosed With Immune Thrombocytopenic Purpura (NCT NCT00699140)

NCT ID: NCT00699140

Last Updated: 2016-02-08

Results Overview

The primary efficacy endpoint was the proportion of patients who reached a platelet count ≥ 50x10\^9/L.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 18 participants
• Primary outcome timeframe: At any time during the study period (The platelet count was measured at Days 1-6, 10, 14. 21, 30, 60, 90).
• Results posted on: 2016-02-08

Participant Flow

Participant milestones

Measure	1 Treatment Group With IGIV3I Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses
Overall Study STARTED	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Clinical Trial in Patients Diagnosed With Immune Thrombocytopenic Purpura

Baseline characteristics by cohort

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	43 years n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Russian Federation	12 participants n=5 Participants
Region of Enrollment United Kingdom	2 participants n=5 Participants
Region of Enrollment Spain	4 participants n=5 Participants
Subjects with medical history of splenectomy Yes	9 participants n=5 Participants
Subjects with medical history of splenectomy No	9 participants n=5 Participants
Subjects with haemorrhagic history Yes	13 participants n=5 Participants
Subjects with haemorrhagic history No	5 participants n=5 Participants
Platelet count	8.5 platelets x 10^-9/L n=5 Participants

PRIMARY outcome

Timeframe: At any time during the study period (The platelet count was measured at Days 1-6, 10, 14. 21, 30, 60, 90).

Population: All 18 subjects received at least one infusion (at any dose) of IGIV3I Grifols and were included in the intent-to-treat (ITT) population for efficacy and safety analysis.

The primary efficacy endpoint was the proportion of patients who reached a platelet count ≥ 50x10^9/L.

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Responder Patients	72.2 percentage of subjects Interval 50.2 to 88.4

SECONDARY outcome

Timeframe: During the follow-up period (time points: Days 6, 10, 14, 21, 30, 60, 90 post-first infusion day [Day 1])

Population: The maximum platelet counts were taken from the population who responded to treatment (platelet count ≥ 50x10\^9/L). If any patient received banned medication due to ITP progression during the study, the values obtained after patients received treatment were excluded.

Platelet count was measured at various time points in the follow-up period after infusion.

Outcome measures

Measure	1 Treatment Group With IGIV3I n=13 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Maximum Platelet Level Reached During the Follow-up Period	146.0 platelets x 10^-9/L Interval 71.0 to 763.0

SECONDARY outcome

Timeframe: At any time during the study period (time points: Days 1-6, 10, 14, 21, 30, 60, 90 post-first infusion day [Day 1])

Population: From all 18 subjects who received at least one infusion and were included in the intent-to-treat (ITT) population but only 13 responded to the treatment

The time taken for the platelet count to reach ≥ 50x10^9/L from first dose

Outcome measures

Measure	1 Treatment Group With IGIV3I n=13 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Time to Reach Platelet Count ≥ 50x10^9/L (≤ Days)	2 days Interval 0.0 to 5.0

SECONDARY outcome

Timeframe: At any time during the study period (up to 3 months [90 days])

Population: From all 18 subjects who received at least one infusion and were included in the intent-to-treat (ITT) population only 13 responded to the treatment

Length of time platelet count remained ≥ 50x10^9/L from first dose (Day 1)

Outcome measures

Measure	1 Treatment Group With IGIV3I n=13 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Length of Time Platelet Count Remains ≥ 50x10^9/L (≥ Days)	9 days Interval 3.0 to 61.0

SECONDARY outcome

Timeframe: First 10 to14 days since the first infusion day (Day 1)

Population: ITT population: patients who received at least one infusion of the study drug

Percentage of subjects with regression of hemorrhages of Types 1 to 3:

• Type 0: Patients without symptoms of bleeding at the first infusion continue without presenting spontaneous bleeding
• Type 1: Patients with bleeding symptoms at the first infusion had a reduction of the size of large ecchymoses, and no spontaneous appearance of new ecchymoses
• Type 2: Patients with bleeding symptoms at the first infusion had a decrease in the number of cutaneous petechiae, or the extent of the affected area of the body decreased
• Type 3: Patients had active mucosal bleedings at the first infusion, these episodes stopped without re-bleeding, and there was no occurrence of new spontaneous mucosal hemorrhages (e.g., gingival bleeding, epistaxis)

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Regression of Hemorrhages.	83.3 percentage of subjects Interval 62.3 to 95.3

SECONDARY outcome

Timeframe: At any time during the study period (from patient's signature of the informed consent form until 3 months of follow-up)

Population: ITT population: patient who received one infusion with the study drug.

All adverse events (AEs) are tabulated and summarized. The incidence, severity, and causal relationship of the AEs to IGIV3I Grifols are presented by system organ class after medical coding according to the version 15.0 of Medical Dictionary for Regulatory Activities (MedDRA). The frequency of patients with at least one AE and adverse drug reactions are estimated.

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Frequency of Adverse Reactions During and After Infusions by Percentage of Patients Frequency of patients with AEs	88.9 percentage of patients
Frequency of Adverse Reactions During and After Infusions by Percentage of Patients Frequency of patients with adverse drug reactions	38.9 percentage of patients

SECONDARY outcome

Timeframe: At any time during the study period (from patient's signature of the informed consent form until 3 months of follow-up)

Population: ITT population: patient who received at least one infusion with the study drug.

All adverse events (AEs) are tabulated and summarized. The incidence, severity, and causal relationship of the AEs to IGIV3I Grifols are presented by system organ class after medical coding according to the version 15.0 of Medical Dictionary for Regulatory Activities (MedDRA). The frequency of infusions associated with at least one AE and adverse drug reactions are estimated.

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Frequency of Adverse Reactions During and After Infusions by Percentage of Infusions Frequency of infusions with adverse events	26.3 percentage of infusions
Frequency of Adverse Reactions During and After Infusions by Percentage of Infusions Frequency of infusions with adverse drug reactions	24.6 percentage of infusions

SECONDARY outcome

Timeframe: At any time during the study period (from patient's signature of the informed consent form until 3 months of follow-up)

Population: Intent-to-treat population

Laboratory parameters at each treatment day and visit are summarized by patient. Results were marked as normal/abnormal (whether the result is below, within or above the respective reference range) and relevant/irrelevant (as determined by the investigator). The number of abnormal values considered clinically relevant changes (based on the investigator's judgment) was listed.

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Changes in Vital Signs and Clinically Relevant Changes in Laboratory Parameters After the Infusions, Including Renal Function (Creatinine Levels) Clinically relevant changes in vital signs	0 participants
Changes in Vital Signs and Clinically Relevant Changes in Laboratory Parameters After the Infusions, Including Renal Function (Creatinine Levels) Clinically relevant changes in lab parameters	7 participants

SECONDARY outcome

Timeframe: At any time during the study period (from patient's signature of the informed consent form until 3 months of follow-up)

Population: Intent-to-treat population

The results of HIV-1 and -2 antibodies, HCV antibody, HBsAg, HBV antibodies, HAV antibodies, HIV nucleic acid amplification test [NAT], and HCV NAT on Day 1, Day 14, and at Month 1, Month 2 and Month 3 were recorded for several of these markers (as appropriate). A comparison of negative viral markers on Day 1 and Month 3 was performed

Outcome measures

Measure	1 Treatment Group With IGIV3I n=18 Participants Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human) Each patient received a total dose of 2 g/kg IGIV3I Grifols, given intravenously over either 2 days or 5 days in divided doses.
Viral Safety Through the Investigation of Patients Virology Status (Hepatitis A Virus [HA	0 seroconversions

Adverse Events

1 Treatment Group With IGIV3I Grifols

Serious events: 2 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	1 Treatment Group With IGIV3I Grifols n=18 participants at risk Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human)
Vascular disorders Thrombosis	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Investigations Haemoglobin decreased	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Blood and lymphatic system disorders Leukopenia	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)

Other adverse events

Measure	1 Treatment Group With IGIV3I Grifols n=18 participants at risk Open label, non-randomized treatment group with IGIV3I Grifols IGIV3I Grifols: Immune Globulin Intravenous (Human)
Skin and subcutaneous tissue disorders Petechiae	66.7% 12/18 • Number of events 15 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Skin and subcutaneous tissue disorders Ecchymosis	44.4% 8/18 • Number of events 11 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Skin and subcutaneous tissue disorders Palmar erythema	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Skin and subcutaneous tissue disorders Rash	11.1% 2/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
General disorders Pyrexia	22.2% 4/18 • Number of events 4 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
General disorders Chills	11.1% 2/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
General disorders Fatigue	5.6% 1/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Gastrointestinal disorders Abdominal distension	5.6% 1/18 • Number of events 3 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Gastrointestinal disorders Gingival bleeding	11.1% 2/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Gastrointestinal disorders Haemorrhoidal haemorrhage	5.6% 1/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Gastrointestinal disorders Nausea	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Gastrointestinal disorders Vomiting	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Nervous system disorders Headache	38.9% 7/18 • Number of events 15 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Nervous system disorders Radicular syndrome	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Vascular disorders Haematoma	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Vascular disorders Hypertension	11.1% 2/18 • Number of events 3 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Respiratory, thoracic and mediastinal disorders Cough	5.6% 1/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Respiratory, thoracic and mediastinal disorders Epistaxis	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Musculoskeletal and connective tissue disorders Limb discomfort	5.6% 1/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Musculoskeletal and connective tissue disorders Myalgia	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Investigations Alanine aminotransferase increased	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Investigations Blood cholesterol increased	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Investigations Blood triglycerides increased	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Investigations Lipids abnormal	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Immune system disorders Drug hypersensitivity	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Infections and infestations Nasopharyngitis	5.6% 1/18 • Number of events 2 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Blood and lymphatic system disorders Bicytopenia	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)
Renal and urinary disorders Haematuria	5.6% 1/18 • Number of events 1 • 3 months At any time during the study period (from patient´s signature of the informed consent until 3 months of follow-up)

Additional Information

Marta Carretero, PhD, Clinical Project Leader

Instituto Grifols, S.A

Phone: + 34 93 571 22 00

Email: marta.carretero@grifols.com

Results disclosure agreements

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