March 28, 2016
by inesp.alves
1 Comment
Article credits: “the pharma letter” Chugai and BioMarin ink exclusive sublicense agreement Japanese drug major Chugai Pharmaceutical (TYO: 4519) has concluded an exclusive sublicense agreement with US rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) on the patent of Chugai’s … Continue reading
June 10, 2015
by inesp.alves
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Bone turnover – total volume of bone that is resorbed and formed over a period of time, usually expressed as percent/year. It can be estimated by measuring relevant bone biomarkers [1]. Bone remodelling – It’s a dynamic physiological event and … Continue reading
January 7, 2015
by inesp.alves
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In “Achondroplasia: A view to the future options emerging from the benchside”, Narayana, J., Horton, W.A, 2013, there is a very interesting point that reflects the challenges of bringing FGFR3-based therapies to the bedside. “Despite the considerable recent progress in the … Continue reading
September 25, 2014
by inesp.alves
1 Comment
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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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
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
1 Comment
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
1 Comment
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
July 14, 2014 by inesp.alves | 1 Comment
Data from the EU clinical trial registry
BMN-111 complete registry at Clinical Trials Register
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Summary
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| 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 | |
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A. Protocol Information
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|---|---|---|---|
| A.1 | Member State Concerned | UK – MHRA | |
| A.2 | EudraCT number | 2013-004137-32 | |
| A.3 | Full title of the trial |
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| A.3.1 | Title of the trial for lay people, in easily understood, i.e. non-technical, language |
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| 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 | ||
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B. Sponsor Information
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|---|---|---|
| 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 | clinicaltrials@bmrn.com | |
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D. IMP Identification
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|---|---|---|
| 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 |
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D.8 Information on Placebo
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E. General Information on the Trial
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F. Population of Trial Subjects
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|---|---|---|---|
| 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) |
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G. Investigator Networks to be involved in the Trial
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N. Review by the Competent Authority or Ethics Committee in the country concerned
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|---|---|---|
| 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 |
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P. End of Trial
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|---|---|---|
| P. | End of Trial Status | Ongoing |
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