Trial Outcomes & Findings for Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome (NCT NCT02128269)

NCT ID: NCT02128269

Last Updated: 2017-07-13

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Treatment Period (24 weeks)

Results posted on

2017-07-13

Participant Flow

Participant milestones

Participant milestones
Measure
ALXN1007
ALXN1007 10 mg/kg IV q 2 weeks x 12 doses
Treatment Period
STARTED
9
Treatment Period
COMPLETED
7
Treatment Period
NOT COMPLETED
2
Follow-up
STARTED
9
Follow-up
COMPLETED
9
Follow-up
NOT COMPLETED
0

Reasons for withdrawal

Reasons for withdrawal
Measure
ALXN1007
ALXN1007 10 mg/kg IV q 2 weeks x 12 doses
Treatment Period
Adverse Event
1
Treatment Period
Physician Decision
1

Baseline Characteristics

Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
ALXN1007- Open Label Study
n=9 Participants
ALXN1007 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
7 Participants
n=5 Participants
Age, Categorical
>=65 years
2 Participants
n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
Sex: Female, Male
Male
6 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
7 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
2 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
2 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
6 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Region of Enrollment
United States
4 participants
n=5 Participants
Region of Enrollment
Japan
2 participants
n=5 Participants
Region of Enrollment
Italy
1 participants
n=5 Participants
Region of Enrollment
France
2 participants
n=5 Participants

PRIMARY outcome

Timeframe: Treatment Period (24 weeks)

Outcome measures

Outcome measures
Measure
ALXN1007
n=9 Participants
ALXN1007 10 mg/kg IV q 2 weeks x 12 doses
Safety and Tolerability of Intravenous (IV) ALXN1007 as Measured by Percentage of Patients Reporting Adverse Events
8 Participants

Adverse Events

ALXN1007- Open Label Study

Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
ALXN1007- Open Label Study
n=9 participants at risk
ALXN1007 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses
Injury, poisoning and procedural complications
Lumbar vertebral fracture
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Pregnancy, puerperium and perinatal conditions
Abortion missed
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Musculoskeletal and connective tissue disorders
Lumbar spinal stenosis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Injury, poisoning and procedural complications
Wrist fracture
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Hepatobiliary disorders
Cholestasis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Hepatobiliary disorders
Liver Cytolysis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).

Other adverse events

Other adverse events
Measure
ALXN1007- Open Label Study
n=9 participants at risk
ALXN1007 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses
Blood and lymphatic system disorders
Anaemia
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Blood and lymphatic system disorders
Antiphospholipid syndrome
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Blood and lymphatic system disorders
Thrombocytopenia
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Ear and labyrinth disorders
Tinnitus
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Abdominal pain
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Colitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Enterocolitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Gastrooesophageal reflux disease
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Haemorrhoids
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Hypoaesthesia oral
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Gastrointestinal disorders
Nausea
22.2%
2/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
General disorders
Oedema peripheral
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
General disorders
Pain
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Bronchitis
22.2%
2/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Cystitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Gastroenteritis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Labyrinthitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Nasopharyngitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Oral Herpes
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Paronychia
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Pharyngitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Sinusitis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Staphylococcal Infection
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Tracheitis
22.2%
2/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Upper respiratory tract infection
44.4%
4/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Infections and infestations
Urethritis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Musculoskeletal and connective tissue disorders
Back pain
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Nervous system disorders
Carpal tunnel syndrome
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Nervous system disorders
Dizziness
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Nervous system disorders
Hypoaesthesia
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Nervous system disorders
Restless legs syndrome
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Respiratory, thoracic and mediastinal disorders
Cough
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Respiratory, thoracic and mediastinal disorders
Epistaxis
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Skin and subcutaneous tissue disorders
Alopecia
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).
Vascular disorders
Varicose vein
11.1%
1/9 • Treatment Period (24 weeks) and Follow-up Period (12 weeks).

Additional Information

Alexion Medical Monitor

Alexion

Phone: 617-613-1071

Results disclosure agreements

  • Principal investigator is a sponsor employee Publication rights are tied to the completion of the multi-center publication
  • Publication restrictions are in place

Restriction type: OTHER