A clinical study to investigate the efficacy and safety of NT 201 in the treatment of lower limb spasticity in children and adolescents with cerebral palsy.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merz Pharmaceuticals GmbH

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

12 Oct 2022 → 5 Mar 2025

Decision date (initial)

2024-05-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Merz Pharmaceuticals GmbH

External identifiers

EU CT number

2023-503420-19-00

EudraCT number

2021-001634-18

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

To demonstrate efficacy by showing superiority of NT 201 compared intra-individually to placebo in children and adolescents with LL SP caused by CP at week 4 to week 6 after a single injection.

Secondary objectives 3

1. To demonstrate the relevance of the effect of NT 201 compared to placebo in children and adolescents with LL SP caused by CP in terms of response rates in derived MAS score of plantar flexors at week 4 or week 6 after a single injection.
2. To demonstrate the relevance of the effect of NT 201 compared to placebo in children and adolescents with LL SP caused by CP assessed on a Global Impression of Change of Plantar Flexor Spasticity Scale (GICSPF) at week 4 to week 6 after a single injection.
3. To demonstrate the relevance of the effect of NT 201 compared to placebo in children and adolescents with LL SP caused by CP assessed on a Goal Attainment Scale (GAS) at week 4 to week 6 after a single injection.

Conditions and MedDRA coding

Pediatric lower limb (LL) spasticity (SP) caused by cerebral palsy (CP)

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Male and female subjects ≥ 2 to ≤ 17 years of age
2. Bilateral, symmetrical pes equinus due to LL SP caused by CP in subjects with any Gross Motor Function Classification System (GMFCS) level
3. Identical MAS plantar flexor score of ≥ 2 for pes equinus clinical patterns in both legs at neutral ankle position and knee at maximum extension
4. Main eligibility criteria for Open-Label Extension Period (OLEX): MAS plantar flexor score of ≥ 1 in both pes equinus clinical patterns
5. Main eligibility criteria for Open-Label Extension Period (OLEX): Agreement of investigator, parent(s), and subject (if applicable) to continue treatment

Exclusion criteria 6

1. Pre-dominant forms of muscle hypertonia/hyperactivity other than LL SP (e.g., rigidity, dystonia, dyskinesia)
2. Previous treatment with Botulinum neurotoxin (BoNT) of any serotype in any body region within the last four months before injection at baseline visit
3. Fixed contracture in at least one of both pes equinus
4. Significant involuntary movements or limitations that hinder MAS assessment or positioning
5. Clinically significant SP in LL clinical patterns other than pes equinus; or gait patterns drop foot, crouch gait, and equinus with jump knee
6. Limitation of hip abduction to less than 40° or pre-diagnosed migrational percentage greater than 30°

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in derived modified Ashworth Scale (MAS) score of plantar flexors from baseline at control visits at week 4 and week 6

Secondary endpoints 3

1. Response in derived MAS score of plantar flexors at control visits at week 4 or week 6 with response defined as improvement of at least 1 point on the derived MAS score as compared to study baseline
2. GICS-PF score at control visits at week 4 and week 6 by investigator
3. GAS T-score at control visits at week 4 and week 6

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merz Pharmaceuticals GmbH

Sponsor organisation

Merz Pharmaceuticals GmbH

Address

Eckenheimer Landstrasse 100, Nordend-West Nordend-West

City

Frankfurt am Main

Postcode

60318

Country

Germany

Scientific contact point

Organisation

Merz Pharmaceuticals GmbH

Contact name

Contact Point Clinical Trials

Public contact point

Organisation

Merz Pharmaceuticals GmbH

Contact name

Contact Point Clinical Trials

Third parties 6

Locations

2 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications