Overview
Sponsor-declared trial summary
Phase
Therapeutic use (Phase IV)
Status
Ended
Participants planned
50
Countries
1
Sites
1
X-linked adrenoleukodystrophy
To determine the prevalence of RLS in women with ALD.
Key facts
- Sponsor
- Amsterdam UMC Stichting
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 4 Feb 2024 → 1 Dec 2025
- Decision date (initial)
- 2024-12-17
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- amsterdam umc
External identifiers
- EU CT number
- 2024-518989-27-01
- EudraCT number
- 2022-001203-40
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
To determine the prevalence of RLS in women with ALD.
Secondary objectives 1
- To determine whether in a blinded crossover study a 8-week pramipexole treatment course will significantly reduce RLS symptoms compared to placebo by self-report (IRLS) and objective leg movement activity using the Suggested Immobilization Test (SIT) in women with ALD.
Conditions and MedDRA coding
X-linked adrenoleukodystrophy
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-518989-27-00 | Treating Leg Symptoms in Women with X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance | Amsterdam UMC Stichting |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 9
- Phase 1: 1. Women
- Phase 1: 2. Ability to provide verbal consent
- Phase 1: 3. A willingness and ability to comply with study procedures.
- Phase 1: 4. Age ≥18
- Phase 1: 5. Metabolically or genetically confirmed diagnosis of ALD
- Phase 2: 1. Participation in Phase 1
- Phase 2: 2. Ability to provide written informed consent
- Phase 2: 3. Women with ALD who have Restless Leg Syndrome (IRLS > 15)
- Phase 2: 4. Use of adequate contraception
Exclusion criteria 10
- Phase 1: 1. Inflammatory brain demyelination
- Phase 2: 1. Pregnant. Research staff perform pregnancy tests upon visit to center.
- Phase 2: 2. Participants with active or unstable major psychiatric disorder other than ALD, who, in the investigators’ judgement, require further treatment
- Phase 2: 3. Use of dopaminergic agonists or antagonists within the last 30 days
- Phase 2: 4. Alcohol use disorder within the last 30 days
- Phase 2: 5. Methamphetamine or benzodiazepine dependence in the last 30 days
- Phase 2: 6. Neurological disorder or cardiovascular disease raising safety concerns about use of pramipexole and/or judged to interfere with ability to assess efficacy of the treatment
- Phase 2: 7. Medical instability considered to interfere with study procedures
- Phase 2: 8. Renal disease judged to interfere with drug metabolism and excretion
- Phase 2: 9. Patients who are deemed poor candidates by PI discretion
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 6
- 1. Subjective total sleep time
- 2. Subjective sleep latency
- 3. Subjective time awake after sleep onset
- 4. Depressive (PHQ-9) and anxiety (GAD-7) symptoms
- 5. Quality of life measures (SF-36)
- 6. Functional measures: EDSS, 25-FW, and TUG
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Pramipexole Viatris 0,088 mg tabletten
PRD10873926 · Product
- Active substance
- Pramipexole Dihydrochloride Monohydrate
- Substance synonyms
- PRAMIPEXOLE HYDROCHLORIDE MONOHYDRATE
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 0.5 mg milligram(s)
- Max total dose
- 56 mg milligram(s)
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Authorised
- ATC code
- N04BC05 — PRAMIPEXOLE
- Marketing authorisation
- BE376144
- MA holder
- VIATRIS GX
- MA country
- Belgium
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Amsterdam UMC Stichting
- Sponsor organisation
- Amsterdam UMC Stichting
- Address
- De Boelelaan 1117
- City
- Amsterdam
- Postcode
- 1081 HV
- Country
- Netherlands
Scientific contact point
- Organisation
- Amsterdam UMC Stichting
- Contact name
- M. Voermans
Public contact point
- Organisation
- Amsterdam UMC Stichting
- Contact name
- MMC Voermans
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ended | 50 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Amsterdam UMC Stichting
Neurology, Meibergdreef 9, 1105 AZ, Amsterdam
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Netherlands | 2024-02-04 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 4 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-518989-27-00_Unredacted clean | 5 |
| Recruitment arrangements (for publication) | Blanc document | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_2024-518989-27-00_NL _Redacted | 3.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC pramipexole | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-19 | Netherlands | Acceptable 2024-12-17
|
2024-12-17 |