Overview
Sponsor-declared trial summary
Hypophosphatasia
The change in Inorganic Pyrophosphate (PPi), and Pyridoxal 5’-phosphate (PLP) levels after low, and high dose ilofotase alfa in adult hypophosphatasia (HPP) patients.
Key facts
- Sponsor
- AM-Pharma B.V.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 3 May 2023 → 12 Jul 2023
- Decision date (initial)
- 2023-05-02
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Pharmacodynamic, Pharmacokinetic
The change in Inorganic Pyrophosphate (PPi), and Pyridoxal 5’-phosphate (PLP) levels after low, and high dose
ilofotase alfa in adult hypophosphatasia (HPP) patients.
Conditions and MedDRA coding
Hypophosphatasia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | PT | 10049933 | Hypophosphatasia | 100000004850 |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- 18-85 years old, inclusive.
- Genetically confirmed variant in the tissue-nonspecific isozyme alkaline phosphatase (ALPL)-Gene.
- Clinical symptoms of HPP.
- Medical history with 1) at least two independent measures of Alkaline Phosphatase (ALP) below lower level of normal (LLN) and 2) at least one measurement of either PPi or PLP above upper level of normal (ULN).
- Provision of signed and dated informed consent form (ICF) in accordance with local regulations at screening.
- Patients must agree to use adequate double-barrier contraception when sexually active. This applies for the period between trial drug administration and 14 days thereafter.
Exclusion criteria 12
- Is unable or unwilling to participate in all scheduled visits and perform all protocol-mandated assessments.
- Has a known or suspected hypersensitivity to ilofotase alfa or any components of the formulation used.
- Body weight < 40 kilogram and > 120 kilogram.
- Patient has a history of clinically significant abnormalities or of any illness that, in the opinion of the trial investigator, might confound the results of the trial or pose an additional risk to the patient by their participation in the trial.
- NSAID use in the past 2 weeks.
- Use of corticosteroids in the past 4 weeks.
- Use of compounds intended to interfere with bone metabolism (e.g. Denosumab, Teriparatide, Romosozumab, Raloxifene) in the past 3 months.
- Use of bisphosphonates in the past 2 years.
- Participation in a drug trial within 60 days prior to administration of ilofotase alfa.
- Use of asfotase alfa in the previous 3 months.
- A patient who is currently pregnant or lactating.
- Use of supplements including Vitamin B6.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The effect of a low or high dose of ilofotase alfa on the biochemical profile in adult HPP patients.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10151386 · Product
- Active substance
- Ilofotase Alfa
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 3.2 mg/kg milligram(s)/kilogram
- Max total dose
- 3.2 mg/kg milligram(s)/kilogram
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- AM-PHARMA B.V.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/14/1427
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
AM-Pharma B.V.
- Sponsor organisation
- AM-Pharma B.V.
- Address
- Stadsplateau 6
- City
- Utrecht
- Postcode
- 3521 AZ
- Country
- Netherlands
Scientific contact point
- Organisation
- AM-Pharma B.V.
- Contact name
- Juliane Bernholz
Public contact point
- Organisation
- AM-Pharma B.V.
- Contact name
- Juliane Bernholz
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ended | 12 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| Germany | 2023-05-03 | 2023-07-12 | 2023-05-15 | 2023-06-26 |
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
| Title | Submission date | Status | Type |
|---|---|---|---|
| Summary of Results AP-recAP-HPP-01-01 SUM-32656
|
2024-07-03T09:55:00 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay Person Summary AP-recAP-HPP-01-01 | 2024-07-03T09:55:09 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | Lay_persons summary_25JUNE2024_v1_0 | 1 |
| Summary of results (for publication) | CTIS_Synopsis_AP-recAP-HPP-01-01 _final_27Jun24 | 1 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-01-27 | Germany | Acceptable 2023-04-26
|
2023-05-02 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2023-05-09 | Germany | Acceptable 2023-04-26
|
2023-05-09 |