Beyond Achondroplasia

Growing together with Clara

December 31, 2014
by inesp.alves
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Compassionate use policy for orphan drugs

“Compassionate use is a treatment option that allows the use of an unauthorised medicine. Compassionate use programmes are for patients who have a disease with no satisfactory authorised therapies or cannot enter a clinical trial. They are intended to facilitate … Continue reading

October 17, 2014
by inesp.alves
2 Comments

Achondroplasia highlights

During three full days, the V International Congress around Achondroplasia and other Skeletal Dysplasia took place in the north of Spain, very well-organized by Alpe Foundation. Several world-renowned experts attended, giving one or more lectures, over 500 people registered, mainly parents … Continue reading

September 25, 2014
by inesp.alves
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Anual MRI

Since the surprise from the results of the first MRI, when Clara was 9 months old, that anxiety was always there on the other two times Clara was submitted to this exam. The sedation procedure with inhalatory anesthetic is smooth and painless … Continue reading

September 15, 2014
by inesp.alves
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Orphan Drugs Summit 2014 – Media partner

We are just two days of this great event and I’m honored to have been chosen to participate as a media partner. The Orphan Drugs Summit is the leading rare disease event in Northern Europe. A well-established platform where pharmaceutical companies … Continue reading

September 5, 2014
by inesp.alves
5 Comments

5th International Congress around achondroplasia and other skeletal dysplasias

Alpe Foundation, one of the most important spanish and european organisations for achondroplasia, is promoting  the 5th Congress, that will take place in north of Spain, Gijon, on the 11th, 12th and 13th October 2014. Their webpage is in spanish … Continue reading

August 17, 2014
by inesp.alves
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Neurologic signs of cervico-medullary compression

Lately, I´ve knew about other children with Clara´s age and with achondroplasia that had to undergone emergency cervico-medullary decompression surgery. I know also that some parents observe strange signs in their children without recognizing them to be neurologic alterations. I … Continue reading

July 24, 2014
by inesp.alves
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The long way up

Rarely I´ve mentioned here the hard and slow road of limb lenghtening. My first research on achondroplasia was at the day we received the genetic report confirming Clara had achondroplasia. Some moments get engraved in our memories in the hardest … Continue reading

July 17, 2014
by inesp.alves
5 Comments

Some moments of pure enchantment

 

BMN-111 protocol

July 14, 2014 by inesp.alves | 1 Comment

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

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July 14, 2014
by inesp.alves
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One importante step forward on rare diseases registry

From now on, all sponsors of clinical trials have to share and publish relevant information about the trial on the EudraCT , the European Clinical Trials Database. Trials results information will be publicly available at the EU Clinical Trials Register including summary results from … Continue reading

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