Basimglurant in children, adolescents, and young adults with TSC

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Noema Pharma AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

30 Jun 2023 → 28 Apr 2025

Decision date (initial)

2024-09-09

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Sponsor - Neoma Pharma, AG

External identifiers

EU CT number

2024-512611-53-00

EudraCT number

2021-000838-34

ClinicalTrials.gov

NCT05059327

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacokinetic, Safety, Efficacy

To evaluate the efficacy of a double-blind, daily basimglurant administration, adjunctive to ongoing anticonvulsive therapy compared with placebo adjunctive to ongoing anticonvulsive therapy in patients with Tuberous Sclerosis Complex (TSC).

Secondary objectives 7

1. To evaluate the impact of treatment on functioning at school and during social activities.
2. To determine the effect of basimglurant on the severity of symptoms of TSC.
3. To determine the longest seizure free interval (i.e., seizure free days).
4. To evaluate the number of patients considered treatment responders.
5. To evaluate the safety of basimglurant in children, adolescents, and young adults with seizures associated with TSC.
6. To investigate the proportion of patients tolerating each dose during dose escalation.
7. To investigate long-term safety of basimglurant in children, adolescents, and young adults with seizures associated with TSC.

Conditions and MedDRA coding

Tuberous Sclerosis Complex

Study design 5 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1) Ability and willingness to provide informed assent or written consent or consent from their legal representative. 2) Fluency in the language of the study staff 3) Age 5 to 30 years at study entry 4) A documented history of TSC 5) Refractory seizure history 6) Currently receiving one or more anti-epileptic drugs (AEDs) 7) Stable medications or interventions for epilepsy for 30 days before study entry 7) Willingness to complete Patient Reported Outcome assessments 8) For female patients of childbearing potential: Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period; Willingness to use contraception.

Exclusion criteria 1

1. 1) Neurologic disease other than TSC 2) Recent anoxic episode 3) Patient weight below 15kg 4) Clinically significant unstable medical condition(s) 5) Pregnancy or lactation

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Monthly seizure counts of countable seizure types (per 28 days) during the 12-week treatment period in Period 2 and Period 4

Secondary endpoints 8

1. Changes from baseline in Sheehan Disability Scale(SDS) score at Week 16 in Period 2 and at Week 30 in Period 4
2. Caregiver Global Impression of Change (CGIC) score at Week 16 in Period 2 and Week 30 in Period 4.
3. Monthly seizure free days (per 28 days) during the 12-week treatment period in Period 2 and Period 4.
4. Seizure 25% response defined as ≥25% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.
5. Seizure 50% response defined as ≥50% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.
6. Seizure 75% response defined as ≥75% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.
7. >Adverse events, Absolute values and changes from baseline in vital signs, physical examination, electrocardiograms, and clinical laboratory test parameters. >Treatment delays, dose reductions, and dose discontinuations. >S-STS score for suicidal ideation. >Seizure count by types.
8. >Escalation (Yes/No) to each dose level during the dose escalation in Periods 2 and 4. >Toleration (Yes/No) of each dose level during the dose escalation in Periods 2 and 4. >Adverse events and other safety and tolerability parameters during the OLE period.

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Noema Pharma AG

Sponsor organisation

Noema Pharma AG

Address

Barfusserplatz 3

City

Basel

Postcode

4051

Country

Switzerland

Scientific contact point

Organisation

Noema Pharma AG

Contact name

Global Regulatory Affairs

Public contact point

Organisation

Noema Pharma AG

Contact name

Global Regulatory Affairs

Third parties 8

Locations

3 EU/EEA countries · 11 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 30 files

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

Application history

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