NCT01387594

Brief Summary

Study is designed to show a lack of effect on white blood cells circulating in the spinal fluid.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
49

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started May 2012

Typical duration for phase_1

Geographic Reach
5 countries

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 30, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 4, 2011

Completed
10 months until next milestone

Study Start

First participant enrolled

May 3, 2012

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2014

Completed
1.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 26, 2015

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

April 26, 2017

Completed
Last Updated

June 3, 2021

Status Verified

May 1, 2021

Enrollment Period

1.9 years

First QC Date

June 30, 2011

Results QC Date

November 15, 2016

Last Update Submit

May 11, 2021

Conditions

Crohn's DiseaseIleitisIleo-colonic and Colonic Crohn's DiseaseGranulomatous ColitisRegional EnteritisUlcerative Colitis

Keywords

Crohn's disease lumbar puncture anti-MAdCAM monoclonal antibody PF-00547659 Ulcerative Colitis

Outcome Measures

Primary Outcomes (2)

  • Cohort 2: Baseline Absolute Lymphocyte Count in Cerebrospinal Fluid (CSF)

    The primary CSF endpoint of Cohort 2 was the percent change from baseline in absolute lymphocyte counts in CSF after 3 doses of PF-00547659. The hypothesis for the primary endpoint was evaluated using the CSF evaluable population in Cohort 2. CSF samples were obtained via lumbar puncture and analyzed by fluorescence-activated cell sorting (FACS) for total lymphocyte counts. Lumbar punctures were performed by a highly qualified physician using a 20-22 gauge needle, preferably an atraumatic needle.

    Baseline

  • Cohort 2: Percent Change From Baseline in Absolute Lymphocyte Count in CSF at Month 3

    The primary CSF endpoint of Cohort 2 was the percent change from baseline in absolute lymphocyte counts in CSF after 3 doses of PF-00547659. The hypothesis for the primary endpoint was evaluated using the CSF evaluable population in Cohort 2. CSF samples were obtained via lumbar puncture and analyzed by FACS for total lymphocyte counts. Lumbar punctures were performed by a highly qualified physician using a 20-22 gauge needle, preferably an atraumatic needle.

    Baseline, Month 3

Secondary Outcomes (3)

  • Cohorts 1 and 2: Total Number of Participants With Non-Lumbar Puncture (LP) Related Treatment-Emergent Adverse Events (AEs), Withdrawals Due to AEs, and Serious Adverse Events (SAEs) During the 12-week Treatment Period

    Baseline up to Week 12

  • Cohorts 1 and 2: Number of Participants Who Developed Anti-Drug Antibodies (ADAs) to PF-00547659

    Day 1; Weeks 4, 8, 9-11 (Cohort 2 only), 12, 20, 28, and 36; Early Withdrawal

  • Cohorts 1 and 2: Number of Participants With Injection Site Reactions by Severity

    Baseline till End of Study/Early Withdrawal, up to Week 12

Study Arms (2)

Cohort 1

EXPERIMENTAL

Interventions prior to treatment. Control arm

Procedure: lumbar puncture

Cohort 2

EXPERIMENTAL

Interventions prior to and after 3 monthly injections

Drug: lumbar puncture

Interventions

lumbar puncturePROCEDURE

2 lumbar punctures prior to treatment; study drug 225mg SC once a month X 3 doses.

Cohort 1

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • males and females \>=18 and =\<75 years
  • For CD subjects: hsCRP \> 5 mg/L and Harvey-Bradshaw Index \> 8 OR when HBI cannot be determined (ie if stoma is present) or hsCRP \< 5mg/L then: active lesions on colonoscopy or flexible sigmoidoscopy or active Crohn's disease on CT or MR enterography
  • For UC subjects: diagnosis of UC \> 3 months; must have endoscopy to confirm active disease during screening; total mayo score of 6 to 12 points and moderate to severe disease on endoscopy

You may not qualify if:

  • Pregnancy or breastfeeding
  • TB or active enteric infections
  • Entero vesicular fistulae
  • Prior use of natalizumab or vedolizumab
  • Right or left heart failure including symptomatic diastolic dysfunction or unexplained elevation of troponin I (\>0.05 ng/mL)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

AKH Wien Universitaetsklinik fuer Innere Medizin III Klinische Abteilung fuer Gastroenterologie und

Vienna, 1090, Austria

Location

Hopital Erasme

Brussels, B-1000, Belgium

Location

UZ Gasthuisberg

Leuven, B-3000, Belgium

Location

Hopital Cardiologique

Lille, 59037, France

Location

Hopital Huriez, CHRU de Lille

Lille, 59037, France

Location

Hopital Saint-Louis - CIC

Paris, 75010, France

Location

Hopital Saint-Louis

Paris, 75010, France

Location

Charité, Universitaetsmedizin Berlin, Campus Virchow Klinikum,

Berlin, 13353, Germany

Location

Academic Medical Center - University of Amsterdam, Dept. of Gastroenterology

Amsterdam, 1105 AZ, Netherlands

Location

Related Publications (1)

  • Stuve O, Cataldi F, Pradhan V, Gorelick KJ. Normal intrathecal leukocyte cell number and composition do not decrease the incidence of post-lumbar puncture headache. J Neuroimmunol. 2017 Sep 15;310:69-71. doi: 10.1016/j.jneuroim.2017.06.011. Epub 2017 Jun 30.

    PMID: 28778448DERIVED

Related Links

MeSH Terms

Conditions

Crohn DiseaseIleitisColitis, Ulcerative

Interventions

Spinal Puncture

Condition Hierarchy (Ancestors)

Inflammatory Bowel DiseasesGastroenteritisGastrointestinal DiseasesDigestive System DiseasesIntestinal DiseasesEnteritisIleal DiseasesColitisColonic Diseases

Intervention Hierarchy (Ancestors)

BiopsySpecimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisDiagnostic Techniques, NeurologicalPuncturesTherapeuticsSurgical Procedures, OperativeInvestigative Techniques

Results Point of Contact

Title
Study Director
Organization
Shire
ClinicalTransparency@shire.com
+1 866 842 5335

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 30, 2011

First Posted

July 4, 2011

Study Start

May 3, 2012

Primary Completion

March 31, 2014

Study Completion

November 26, 2015

Last Updated

June 3, 2021

Results First Posted

April 26, 2017

Record last verified: 2021-05

Locations

BE(2)NL(1)DE(1)AU(1)FR(4)