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A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Primary Purpose

Mucopolysaccharidosis II

Status

Recruiting

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

DNL310

Idursulfase

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis II focused on measuring Hunter Syndrome, MPS II, nMPS II, nnMPS II

Eligibility Criteria

2 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B)
Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening

Key Exclusion Criteria:

Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
Received any CNS-targeted MPS ERT within 6 months prior to screening
Have a contraindication for lumbar punctures and/or magnetic resonance imagings (MRIs)
Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

Sites / Locations

UCSF Benioff Children's Hospital OaklandRecruiting
Emory UniversityRecruiting
Ann and Robert H Lurie Children's Hospital of ChicagoRecruiting
Hackensack University Medical CenterRecruiting
UNC Children's Research InstituteRecruiting
Cincinnati Children's Hospital Medical CenterRecruiting
Children's Hospital of PhiladelphiaRecruiting
Children's Hospital of PittsburghRecruiting
The University of Texas Medical School at HoustonRecruiting
Hospital Italiano de Buenos AiresRecruiting
Universitair Ziekenhuis BrusselRecruiting
UZ AntwerpenRecruiting
University of Alberta - Faculty of Medicine & DentistryRecruiting
Hospital for Sick ChildrenRecruiting
McGill University Health CenterRecruiting
Vseobecna Fakultni Nemocnice V PrazeRecruiting
Hôpital Jeanne de FlandreRecruiting
Hopitaux de La TimoneRecruiting
CHU ToulouseRecruiting
Universitätsklinikum Hamburg EppendorfRecruiting
SphinCSRecruiting
Azienda Ospedaliera Universitaria Federico IIRecruiting
Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della MisericordiaRecruiting
Erasmus Medical Center - Sophia Children's HospitalRecruiting
Hospital Clínico Universitario de SantiagoRecruiting
Hospital Universitario Vall d'HebronRecruiting
Hospital Infantil Universitario Niño JesusRecruiting
Drottning Silvias Barn Och UngdomssjukhusRecruiting
Cukurova University Medical Faculty Balcali HospitalRecruiting
Gazi Universitesi Tip FakultesiRecruiting
Birmingham Women's and Children's NHS Foundation TrustRecruiting
Great Ormond Street Hospital for ChildrenRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Cohort A: Participants with nMPS II

Cohort B: Participants with nnMPS II

Open-label Treatment Phase

Arm Description

Participants who meet pre-specified criteria may receive DNL310 or idursulfase

Outcomes

Primary Outcome Measures

Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A only)

Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A only)

Secondary Outcome Measures

Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only)

Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only)

Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (Cohorts A and B)

Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B)

Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B)

Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B)

Full Information

NCT ID

NCT05371613

First Posted

May 9, 2022

Last Updated

September 27, 2023

Sponsor

Denali Therapeutics Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05371613

Brief Title

A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Acronym

COMPASS

Official Title

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Recruiting

Study Start Date

July 21, 2022 (Actual)

Primary Completion Date

December 2025 (Anticipated)

Study Completion Date

December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Denali Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of DNL310, an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mucopolysaccharidosis II

Keywords

Hunter Syndrome, MPS II, nMPS II, nnMPS II

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

54 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Cohort A: Participants with nMPS II

Arm Type

Experimental

Arm Title

Cohort B: Participants with nnMPS II

Arm Type

Experimental

Arm Title

Open-label Treatment Phase

Arm Type

Experimental

Arm Description

Participants who meet pre-specified criteria may receive DNL310 or idursulfase

Intervention Type

Drug

Intervention Name(s)

DNL310

Intervention Description

Intravenous repeating dose

Intervention Type

Drug

Intervention Name(s)

Idursulfase

Intervention Description

Intravenous repeating dose

Primary Outcome Measure Information:

Title

Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A only)

Time Frame

24 weeks

Title

Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A only)

Time Frame

96 weeks

Secondary Outcome Measure Information:

Title

Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only)

Time Frame

96 weeks

Title

Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only)

Time Frame

48 weeks

Title

Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (Cohorts A and B)

Time Frame

up to 48 weeks

Title

Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B)

Time Frame

48 weeks

Title

Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B)

Time Frame

48 weeks

Title

Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B)

Time Frame

48 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B) Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II) Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening Key Exclusion Criteria: Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy Received any CNS-targeted MPS ERT within 6 months prior to screening Have a contraindication for lumbar punctures and/or magnetic resonance imagings (MRIs) Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Clinical Trials at Denali Therapeutics

clinical-trials@dnli.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Lubica Trokan, MD, MPH

Organizational Affiliation

Denali Therapeutics Inc.

Official's Role

Study Director

Facility Information:

Facility Name

UCSF Benioff Children's Hospital Oakland

City

Oakland

State/Province

California

ZIP/Postal Code

94609

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Annie Sako

Phone

510-428-3885

Ext

4785

annie.sako@ucsf.edu

First Name & Middle Initial & Last Name & Degree

Paul Harmatz, MD

Facility Name

Emory University

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30322

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Gwendolyn Gunn

gwendolyn.gisiner.gunn@emory.edu

First Name & Middle Initial & Last Name & Degree

William Wilcox, MD

Facility Name

Ann and Robert H Lurie Children's Hospital of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lindsey Lang

llang@luriechildrens.org

Phone

312-227-6712

First Name & Middle Initial & Last Name & Degree

Barbara Burton, MD

Facility Name

Hackensack University Medical Center

City

Hackensack

State/Province

New Jersey

ZIP/Postal Code

07601

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Susan Mathus

susan.mathus@hmhn.org

First Name & Middle Initial & Last Name & Degree

Helio Pedro, MD

Facility Name

UNC Children's Research Institute

City

Chapel Hill

State/Province

North Carolina

ZIP/Postal Code

27514

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Heather Press

heather_preiss@med.unc.edu

First Name & Middle Initial & Last Name & Degree

Joseph Muenzer, MD

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Connie Wehmeyer

Phone

513-636-4760

connie.wehmeyer@cchmc.org

First Name & Middle Initial & Last Name & Degree

Nancy Leslie, MD

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Henry Daumeyer

daumeyerh@chop.edu

First Name & Middle Initial & Last Name & Degree

Can Ficicioglu, MD

Facility Name

Children's Hospital of Pittsburgh

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Dawn Kolar

kolardr@upmc.edu

First Name & Middle Initial & Last Name & Degree

Deepa Rajan, MD

Facility Name

The University of Texas Medical School at Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Marilyn Garcia

marilyn.garcia@uth.tmc.edu

First Name & Middle Initial & Last Name & Degree

Paul Hillman, MD

Facility Name

Hospital Italiano de Buenos Aires

City

Buenos Aires

ZIP/Postal Code

C1199ABB

Country

Argentina

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Sabina Garcia

sabinagarcia@iadin.com.ar

First Name & Middle Initial & Last Name & Degree

Javier Muntadas Rausei, MD

Facility Name

Universitair Ziekenhuis Brussel

City

Jette

State/Province

Brussels

ZIP/Postal Code

1090

Country

Belgium

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Raquel Van den Eydne

Raquel.VanDenEynde@uzbrussel.be

First Name & Middle Initial & Last Name & Degree

Luc Regal, MD

Facility Name

UZ Antwerpen

City

Antwerpen

ZIP/Postal Code

2650

Country

Belgium

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tine Maes

tine.maes@uza.be

First Name & Middle Initial & Last Name & Degree

Francois Eyskens, MD

Facility Name

University of Alberta - Faculty of Medicine & Dentistry

City

Edmonton

State/Province

Alberta

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Farah Hassan

farah.hassan@albertahealthservices.ca

First Name & Middle Initial & Last Name & Degree

Saadet Andrews, MD

Facility Name

Hospital for Sick Children

City

Toronto

State/Province

Ontario

ZIP/Postal Code

M5G1X8

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Preet Dhillon

preet.dhillon@sickkids.ca

First Name & Middle Initial & Last Name & Degree

Michal Inbar-Feigenberg, MD

Facility Name

McGill University Health Center

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H4A3J1

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Christine Gannon

christine.gannon@muhc.mcgill.ca

First Name & Middle Initial & Last Name & Degree

John Mitchell, MD

Facility Name

Vseobecna Fakultni Nemocnice V Praze

City

Praha

ZIP/Postal Code

128 08

Country

Czechia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lenka Linkova

lenka.linkova@vfn.cz

First Name & Middle Initial & Last Name & Degree

Martin Magner, MD

Facility Name

Hôpital Jeanne de Flandre

City

Lille

ZIP/Postal Code

59000

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Celine Desopper

amina.ziouche@chru-lillie.fr

First Name & Middle Initial & Last Name & Degree

Anne-Sophie Guémann, MD

Facility Name

Hopitaux de La Timone

City

Marseille

ZIP/Postal Code

13385

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Murielle Champeaux

murielle.champeaux@ap-hm.fr

First Name & Middle Initial & Last Name & Degree

Brigitte Chabrol, MD

Facility Name

CHU Toulouse

City

Toulouse

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Léa Saintouil

saintouil.l@chu-toulouse.fr

First Name & Middle Initial & Last Name & Degree

Aurélie Bourchany, MD

Facility Name

Universitätsklinikum Hamburg Eppendorf

City

Hamburg

ZIP/Postal Code

20251

Country

Germany

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Katharina Trepner

icld@uke.de

First Name & Middle Initial & Last Name & Degree

Nicole Muschol, MD

Facility Name

SphinCS

City

Hochheim

ZIP/Postal Code

65239

Country

Germany

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Loreen Gramsch

loreen.gramsch@sphincs.de

First Name & Middle Initial & Last Name & Degree

Eugen Mengel, MD

Facility Name

Azienda Ospedaliera Universitaria Federico II

City

Napoli

ZIP/Postal Code

80131

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Margherita Dell'Anno

m.dellanno@tigem.it

First Name & Middle Initial & Last Name & Degree

Giancarlo Parenti, MD

Facility Name

Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia

City

Udine

ZIP/Postal Code

33100

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Enrica Epifani

enrica.epifani@asufc.sanita.fvg.it

First Name & Middle Initial & Last Name & Degree

Maurizio Scarpa, MD

Facility Name

Erasmus Medical Center - Sophia Children's Hospital

City

Rotterdam

ZIP/Postal Code

3000

Country

Netherlands

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Dorine Heemskerk

t.heemskerk@erasmusmc.nl

First Name & Middle Initial & Last Name & Degree

Hannerieke van den Hout

Facility Name

Hospital Clínico Universitario de Santiago

City

A Coruña

ZIP/Postal Code

15706

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Roi Chans Gerpe

roi.chans.gerpe@sergas.es

First Name & Middle Initial & Last Name & Degree

Maria Luz Couce Pico, MD

Facility Name

Hospital Universitario Vall d'Hebron

City

Barcelona

ZIP/Postal Code

08035

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Marina González Riera

marina.gonzalez@vhir.org

First Name & Middle Initial & Last Name & Degree

Mireia del Toro, MD

Facility Name

Hospital Infantil Universitario Niño Jesus

City

Madrid

ZIP/Postal Code

28009

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Christiana Calvo Patino

ccpatino@salud.madrid.org

First Name & Middle Initial & Last Name & Degree

Beatriz Bermadino, MD

Facility Name

Drottning Silvias Barn Och Ungdomssjukhus

City

Göteborg

ZIP/Postal Code

416 85

Country

Sweden

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tina Kjellén

tina.kjellen@vgregion.se

First Name & Middle Initial & Last Name & Degree

Niklas Darin, MD, PhD

Facility Name

Cukurova University Medical Faculty Balcali Hospital

City

Adana

ZIP/Postal Code

1330

Country

Turkey

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Nurşah Gulseren

nursahgulseren523@hotmail.com

First Name & Middle Initial & Last Name & Degree

Neslihan Mungan, MD

Facility Name

Gazi Universitesi Tip Fakultesi

City

Çankaya

ZIP/Postal Code

06500

Country

Turkey

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Gozde Dulgar

gozde.dulgar@medex-smo.com

First Name & Middle Initial & Last Name & Degree

Faith Ezgu, MD

Facility Name

Birmingham Women's and Children's NHS Foundation Trust

City

Birmingham

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Shashi Watson-Jones

shashi.rana@nhs.net

First Name & Middle Initial & Last Name & Degree

Harprit Thakur

harprit.thakur@nhs.net

Facility Name

Great Ormond Street Hospital for Children

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Kerry Gilroy

kerry.gilroy@gosh.nhs.uk

First Name & Middle Initial & Last Name & Degree

Olivia Rosie-Wilkinson

Olivia.Rosie-Wilkinson@gosh.nhs.uk

First Name & Middle Initial & Last Name & Degree

Spyros Batzios, MD

12. IPD Sharing Statement

Plan to Share IPD

Links:

URL

https://www.engagehunter.com/clinical-trials

Description

Engage Hunter Website

Learn more about this trial

A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

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