Beyond Achondroplasia

Growing together with Clara

BMN-111 protocol

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Data from the EU clinical trial registry

BMN-111 complete registry at Clinical Trials Register

Summary
EudraCT Number: 2013-004137-32
Sponsor’s Protocol Code Number: 111-202
National Competent Authority: UK – MHRA
Clinical Trial Type: EEA CTA
Trial Status: Ongoing
Date on which this record was first entered in the EudraCT database: 2014-02-17
Trial results
Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL
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A. Protocol Information
A.1 Member State Concerned UK – MHRA
A.2 EudraCT number 2013-004137-32
A.3 Full title of the trial
A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia
A.3.1 Title of the trial for lay people, in easily understood, i.e. non-technical, language
A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia
A.4.1 Sponsor’s protocol code number 111-202
A.7 Trial is part of a Paediatric Investigation Plan No
A.8 EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1 Name of Sponsor BioMarin Pharmaceutical Inc.
B.1.3.4 Country United States
B.3.1 and B.3.2 Status of the sponsor Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1 Name of organisation providing support BioMarin Pharmaceutical Inc.
B.4.2 Country United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1 Name of organisation BioMarin Pharmaceutical Inc.
B.5.2 Functional name of contact point Clinical Trials Information
B.5.3 Address:
B.5.3.1 Street Address 105 Digital Drive
B.5.3.2 Town/ city Novato
B.5.3.3 Post code 94949
B.5.3.4 Country United States
B.5.6 E-mail clinicaltrials@bmrn.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3 IMP Role Test
D.2 Status of the IMP to be used in the clinical trial
D.2.1 IMP to be used in the trial has a marketing authorisation No
D.2.5 The IMP has been designated in this indication as an orphan drug in the Community Yes
D.2.5.1 Orphan drug designation number EU/3/12/1094
D.3 Description of the IMP
D.3.1 Product name modified recombinant human C-type natriuretic peptide
D.3.2 Product code BMN 111
D.3.4 Pharmaceutical form Lyophilisate for solution for injection
D.3.4.1 Specific paediatric formulation Yes
D.3.7 Routes of administration for this IMP Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8 INN – Proposed INN TBD
D.3.9.2 Current sponsor code BMN 111
D.3.9.3 Other descriptive name MODIFIED RHCNP
D.3.9.4 EV Substance Code SUB120857
D.3.10 Strength
D.3.10.1 Concentration unit mg/ml milligram(s)/millilitre
D.3.10.2 Concentration type range
D.3.10.3 Concentration number 0.2 to 10
D.3.11 The IMP contains an:
D.3.11.1 Active substance of chemical origin No
D.3.11.2 Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
The IMP is a:
D.3.11.3 Advanced Therapy IMP (ATIMP) No
D.3.11.3.1 Somatic cell therapy medicinal product No
D.3.11.3.2 Gene therapy medical product No
D.3.11.3.3 Tissue Engineered Product No
D.3.11.3.4 Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5 Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4 Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5 Radiopharmaceutical medicinal product No
D.3.11.6 Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7 Plasma derived medicinal product No
D.3.11.8 Extractive medicinal product No
D.3.11.9 Recombinant medicinal product Yes
D.3.11.10 Medicinal product containing genetically modified organisms No
D.3.11.11 Herbal medicinal product No
D.3.11.12 Homeopathic medicinal product No
D.3.11.13 Another type of medicinal product No

D.8 Information on Placebo

E. General Information on the Trial

F. Population of Trial Subjects
F.1 Age Range
F.1.1 Trial has subjects under 18 Yes
F.1.1 Number of subjects for this age range: 24
F.1.1.1 In Utero No
F.1.1.2 Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3 Newborns (0-27 days) No
F.1.1.4 Infants and toddlers (28 days-23 months) No
F.1.1.5 Children (2-11years) Yes
F.1.1.5.1 Number of subjects for this age range: 23
F.1.1.6 Adolescents (12-17 years) Yes
F.1.1.6.1 Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) No
F.1.3 Elderly (>=65 years) No
F.2 Gender
F.2.1 Female Yes
F.2.2 Male Yes
F.3 Group of trial subjects
F.3.1 Healthy volunteers No
F.3.2 Patients Yes
F.3.3 Specific vulnerable populations Yes
F.3.3.1 Women of childbearing potential not using contraception No
F.3.3.2 Women of child-bearing potential using contraception Yes
F.3.3.3 Pregnant women No
F.3.3.4 Nursing women No
F.3.3.5 Emergency situation No
F.3.3.6 Subjects incapable of giving consent personally No
F.3.3.7 Others No
F.4 Planned number of subjects to be included
F.4.1 In the member state 5
F.4.2 For a multinational trial
F.4.2.1 In the EEA 10
F.4.2.2 In the whole clinical trial 24
F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
In order to assess safety and efficacy of BMN 111 over the longer term, subjects may be eligible to continue receiving BMN 111 in an open-label extension study after completing 6 months of treatment in the initial study.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned
N. Competent Authority Decision Authorised
N. Date of Competent Authority Decision 2014-02-14
N. Ethics Committee Opinion of the trial application Favourable
N. Ethics Committee Opinion: Reason(s) for unfavourable opinion
N. Date of Ethics Committee Opinion 2014-04-03

P. End of Trial
P. End of Trial Status Ongoing

One Comment

  1. Great job as usual
    Fara Cisse Envoyé de mon iPhone
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