Screening Trial for Pain Relief in Schwannomatosis (STARFISH)

ConditionsSchwannomatosis Schwannomas Pain, Chronic

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorMassachusetts General Hospital

About this trial

This is a placebo-controlled, multi-arm phase II platform screening trial designed to test the safety, pain responses, and pharmacodynamic activity of multiple experimental therapies simultaneously in participants with moderate-to-severe pain due to schwannomatosis (SWN).

This Master Study is being conducted as a platform that may allow participants with pain associated with schwannomatosis to receive a novel intervention throughout this study. Embedded within the Master Study are individual drug sub-studies:

* Investigational Drug Sub-Study A: Siltuximab * Investigation Drug Sub-Study B: Erenumab-Aooe

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients must have a confirmed diagnosis schwannomatosis by fulfilling either clinical or molecular diagnosis.

Two or more non-intradermal schwannomas, one with pathological confirmation, without evidence of bilateral vestibular schwannoma (see

Disqualifiers

one pathologically confirmed schwannoma or intracranial meningioma and

An affected first-degree relative. Molecular diagnosis

A molecular diagnosis of schwannomatosis is confirmed by either (1) two or more pathologically proven schwannomas or meningiomas AND genetic studies of at least two tumors with loss of heterozygosity (LOH) for chromosome 22 and two different NF2 mutations; or (2) one pathologically proven schwannoma or meningioma and a germline SMARCB1 or LZTR1 pathogenic mutation.

Participant must be ≥ 18 years of age on Day 1 of treatment.

Trial design

Design model

Parallel

Treatments tested in this trial

Siltuximab
Drug
A chimeric immunoglobulin G mAb, via intravenous infusion.
Erenumab-Aooe
Drug
Human monoclonal antibody, single-dose prefilled SureClick® autoinjector, via subcutaneous injection.
Siltuximab Matching Placebo
Drug
Dextrose 5% in water, via intravenous infusion.
Erenumab-Aooe Matching Placebo
Drug
0.9% saline, 1 mL single-dose prefilled syringe, via subcutaneous injection.

Treatment groups

40 Participants

are divided into 2 treatment groups

Group A: Sub-study A: SiltuximabExperimental treatment 2 interventions

Group B: Sub-study B: Erenumab-AooeExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

Change in worst pain intensity for each drug sub-study

Defined as the change in the worst pain intensity between baseline and day 85. Worst pain is measured using the 11-point Pain Intensity Numerical Rating Scale (NRS), where scores range from 0 (no pain) to 10 (worst pain ever) of the maximum SWN-related pain experienced over the past week

Time frame

Baseline to week 12

Secondary outcomes

Number of Participants with Adverse Events in each drug sub-study

Graded according to NCI CTCAE v5.0. All participants will be evaluable for toxicity from the time of first treatment.

Time frame

Randomization/1st treatment to 4 weeks post final treatment

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Massachusetts General Hospital

Lead sponsor

United States Department of Defense

Collaborator

RECORDATI GROUP