Musculoskeletal

AUTODECRA 1 - Closedown/Analysis

Autologous Tolerogenic Dendritic Cells for Rheumatoid and Inflammatory Arthritis

• Study stage: Closed
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: Arthritis Research UK
• Therapeutic area: Musculoskeletal
• Type of study: ATIMP

Aim: To assess safety of intra-articular TolDC in patients with RA and other chronic inflammatory arthritis

Primary Outcome:  The proportion of patients experiencing AEs and SAEs following the intra-articular administration of TolDC.

• Population: Adults
• Clinical Phase: I
• Design: Randomised Control Trial (RCT)
• Setting: Secondary Care
• Planned Sample Size: 12

AuToDeCRA 2 - In Setup

A single-centre, experimental medicine study investigating the route of delivery and efficacy of autologous tolerogenic dendritic cell (tolDC) therapy for Rheumatoid Arthritis.

• Study stage: Set-up
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: Versus Arthritis
• Therapeutic area: Musculoskeletal
• Type of study: ATIMP

Aim: Demonstrate safety of tolDC administration for intra-articular, intra-dermal and intra-nodal routes.
Identify optimal route of administration to ensure tolDC reaches secondary lymphoid tissue.

• Population: Adults
• Clinical Phase: IIa
• Design: Randomised Control Trial (RCT)
• Setting: Secondary Care

FOR-DMD - Closedown/Analysis

Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid Regimen

• Study Stage: Closed
• Sponsor: University of Rochester (NY, USA)
• Funder: NIH
• Therapeutic area: Musculoskeletal
• Type of study: CTIMP

Aim: To establish the optimum corticosteroid regimen, of three under investigation, to address the pragmatic hypothesis that daily corticosteroids (prednisone or deflazacort) will be of greater benefit in terms of function and subject/parent satisfaction than intermittent corticosteroids (prednisone).

Primary outcome:  The primary outcome variable is a three-dimensional (multivariate) outcome consisting of the following three components (each averaged over all post-baseline follow-up visits through Month 36):

1. time to stand from lying (log-transformed)
2. forced vital capacity
3. subject/parent global satisfaction with treatment, as measured by the Treatment Satisfaction Questionnaire for Medication

• Population: Paediatric
• Clinical Phase: III
• Design: RCT
• Setting: Secondary Care
• Planned Sample Size: 225

MET-PREVENT - Closed

Metformin to prevent progression of sarcopenia and frailty in older people – a double blind, randomised controlled proof of concept trial

• Study stage: Closed
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: NIHR Newcastle Biomedical Research Centre
• Therapeutic area: Musculoskeletal
• Type of study: CTIMP

Aim: Provide proof-of-concept evidence as to whether metformin can improve physical performance in older people with sarcopenia and prefrailty

Primary Outcome:  The between-group difference in 4m walk speed at 4 months, adjusted for baseline values.

• Population: Ageing
• Clinical Phase: IIII
• Design: Randomised Control Trial (RCT)
• Setting: Secondary Care
• Planned Sample Size: 80

TRAFIC - Closedown/Analysis

TRAFIC - Targeting the Rheumatoid Arthritis synovial fibroblast via cyclin dependent kinase inhibition – an early phase trial

• Study Stage: Part 1 complete, part 2 in follow-up
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: Medical Research Council: Biomedical Catalyst: Developmental pathway Funding Scheme
• Therapeutic area: Musculoskeletal
• Type of study: CTIMP

Aim: Part 1: To determine the MTD of seliciclib in participants with active Rheumatoid Arthritis (RA) despite treatment with anti-TNF as either monotherapy or with background conventional disease-modifying anti-rheumatic drugs (DMARDs). Part 2: To assess response to treatment at 12 weeks.

Primary outcome:

Part 1: Identification of Maximum Tolerated Dose of Seliciclib to be used in part 2 as determined by the CRM algorithm developed for this study, with a target Dose Limiting Toxicity probability of 35%; or determination of unacceptable toxicity leading to cessation of trial.

Part 2: Composite response at 12 weeks based on clinical (EULAR and ACR20 response rates), histological (macrophage number in the sub lining layer of synovium) and MRI (Rheumatoid Arthritis MRI Scoring System (RAMRIS)) scores alongside evidence of PD activity.

• Population: Adult
• Clinical Phase: Ib/IIa (Part 1: Dose escalation design - Part 2: Single arm, single stage)
• Design: Adaptive
• Setting: Secondary Care
• Planned Sample Size: 39 (21 part 1 and 18 part 2)