NCT02676947

Brief Summary

An open label study to assess the safety and efficacy of tocilizumab in group 1 pulmonary arterial hypertension patients

Trial Health

87
On Track

Trial Health Score

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

Enrollment
29

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2016

Geographic Reach
1 country

1 active site

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

December 21, 2015

Completed
11 days until next milestone

Study Start

First participant enrolled

January 1, 2016

Completed
1 month until next milestone

First Posted

Study publicly available on registry

February 9, 2016

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 18, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 18, 2018

Completed
Last Updated

April 19, 2018

Status Verified

April 1, 2018

Enrollment Period

2.1 years

First QC Date

December 21, 2015

Last Update Submit

April 17, 2018

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (2)

  • Safety - Incidence and severity of adverse events

    Incidence and severity of treatment emergent adverse events

    6 months

  • Pulmonary vascular resistance- dynes (cm-5)

    Invasive haemodynamic assessment by right heart catheter

    Change from baseline pulmonary vascular resistance to end of study at 6 months

Secondary Outcomes (4)

  • Six minute walk test

    Baseline and every month for 6 months

  • N-Terminal pro-B-type Natriuretic Peptide

    Baseline and every month for 6 months

  • World Health Organisation functional class assessment of patient reported symptoms

    Baseline and every month for 6 months

  • Quality of Life

    Baseline and every month for 6 months

Study Arms (1)

Open Label

EXPERIMENTAL

Intravenous Tocilizumab 8mg/kg monthly (up to a maximum dose 800mg) for 6 months

Drug: Tocilizumab

Interventions

TocilizumabDRUG
Also known as: RoActemra
Open Label

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Group 1 PAH due to: Idiopathic or Heritable PAH, PAH associated with connective tissue disease excluding SLE, RA and mixed CTD, Drug and Toxins
  • WHO functional class II-IV
  • Weight more than 40kg
  • minute walk distance of 100-500 m
  • Haemodynamic criteria measure by RHC
  • Documented negative V/Q scan or pulmonary arteriogram confirming absence of chromic thromboembolic disease
  • Resting oxygen saturations of \>85%
  • Lung function confirming absence of significant lung disease
  • Stable on unchanged PAH therapeutic regime for at least 1 month

You may not qualify if:

  • Subjects on continuous infusions either intravenously or subcutaneously
  • Hypersensitivity to Investigational Product
  • Severe hepatic impairment
  • Severe renal impairment
  • Clinically significant anaemia
  • Blood platelets \<100x10
  • Neutrophil count \<2x10/L
  • Left ventricular disease/dysfunction risk factors
  • Myocardial infarction within 90 days prior to screening
  • Female subjects who are pregnant or breastfeeding
  • History of malignancies within past 5 years

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Papworth Hospital NHS Foundation Trust

Cambridge, Cambridgeshire, CB23 3RE, United Kingdom

Location

Related Publications (1)

  • Toshner M, Church C, Harbaum L, Rhodes C, Villar Moreschi SS, Liley J, Jones R, Arora A, Batai K, Desai AA, Coghlan JG, Gibbs JSR, Gor D, Graf S, Harlow L, Hernandez-Sanchez J, Howard LS, Humbert M, Karnes J, Kiely DG, Kittles R, Knightbridge E, Lam B, Lutz KA, Nichols WC, Pauciulo MW, Pepke-Zaba J, Suntharalingam J, Soubrier F, Trembath RC, Schwantes-An TL, Wort SJ, Wilkins MR, Gaine S, Morrell NW, Corris PA; Uniphy Clinical Trials Network. Mendelian randomisation and experimental medicine approaches to interleukin-6 as a drug target in pulmonary arterial hypertension. Eur Respir J. 2022 Mar 10;59(3):2002463. doi: 10.1183/13993003.02463-2020. Print 2022 Mar.

    PMID: 34588193DERIVED

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Hypertension, PulmonaryLung DiseasesRespiratory Tract Diseases

Study Officials

  • Mark Toshner, MD

    Papworth Hospital NHS Foundation Trust

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 21, 2015

First Posted

February 9, 2016

Study Start

January 1, 2016

Primary Completion

February 18, 2018

Study Completion

February 18, 2018

Last Updated

April 19, 2018

Record last verified: 2018-04

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)