Inclusion and Exclusion Criteria for the trial

Clinical Personnel Pages

POSTNATAL GROUP

Inclusion Criteria

For inclusion, all criteria should apply.

  1. Parent’s/legal guardian’s signed informed-consent form

  2. Clinical diagnosis of OI type III or IV AND

  3. Molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)

  4. Age less than 12 months (calculated from gestational week 40+0, i.e. the corrected age)

  5. Parent/legal guardian over 18 years of age

Exclusion Criteria

For exclusion, one is enough to exclude.

  1. Existence of other known disorder that might interfere with the treatment, such as, but not limited to organ dysfunction (for example liver or renal failure or bronchopulmonary dysplasia), congenital heart defect, hypoxic encephalopathy l-lll, severe neurological problems, immune deficiencies, muscle diseases, severe malformations or syndromes diagnosed by clinical examination

  2. Any contraindication for invasive procedures such as a moderate/severe bleeding tendency

  3. Known risk factors for clotting, such as, but not limited to previous blood clot, family history of clots, clotting disorder (inherited or acquired), heart failure, inflammatory disorders (for example lupus, rheumatoid arthritis, inflammatory bowel disease)

  4. Positive Donor Specific Antibody-test

  5. Known allergy/hypersensitivity to Fungizone and/or Gensumycin

  6. Abnormal karyotype or other confirmed genetic syndromes

  7. Oncologic disease (previous or current malignancy)

  8. Inability to comply with the trial protocol and follow-up schedule

  9. Inability to understand the information and to provide informed consent

PRENATAL GROUP

Inclusion Criteria

For inclusion, all criteria should apply.

  1. Woman has signed the patient consent form

  2. Only women where termination of the pregnancy is no longer possible or where the women are committed to continue the pregnancy may be included in the trial

  3. Suspicion of OI type III or IV in the fetus on ultrasound findings AND

  4. Molecular diagnosis of OI in the fetus (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)

  5. Gestation age between 16+0 and 35+6 weeks+days

  6. Pregnant woman over 18 years of age

 

After birth and before the 2nd dose, subjects in the prenatal group will be assessed for the inclusion criteria for the postnatal group. 

Exclusion Criteria

For exclusion, one is enough to exclude.

  1. Multiple pregnancy

  2. Co-existence of other disorder that might interfere with the treatment, as judged by the Investigator or the patient’s obstetrician

  3. Abnormal fetal karyotype or other confirmed genetic syndrome

  4. Any contraindication for invasive procedures such as a bleeding tendency or contagious infections, such as, but not limited to HIV, Syphilis, Hepatitis B, Hepatitis C or other known infectious diseases that can harm the fetus

  5. Known risk factors for clotting, such as, but not limited to previous blood clot, family history of clots, clotting disorder (inherited or acquired), heart failure, inflammatory disorders (for example lupus, rheumatoid arthritis, inflammatory bowel disease)

  6. Positive Donor Specific Antibody-test

  7. Known allergy/hypersensitivity to Fungizone and/or Gensumycin

  8. Oncologic disease in woman or fetus (previous or current malignancy)

  9. Unwilling to or cannot undergo delivery by Caesarean section

  10. Inability to comply with the trial protocol and follow-up schedule

  11. Inability to understand the information and to provide informed consent

 

After birth and before the 2nd dose, subjects in the prenatal group will be assessed for the exclusion criteria for the postnatal group. 

CONTROL GROUP

Inclusion Criteria

For inclusion, all criteria should apply.

a) Matched historical controls

  1. Parent’s/legal guardian’s signed informed-consent form

  2. Clinical and molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)

  3. Data on fractures and growth is available

  4. Parent/legal guardian over 18 years of age

 

b) Prospective untreated controls

Postnatal inclusion: 

The inclusion criteria for the postnatal group apply.

 

Prenatal inclusion: 

The inclusion criteria for the prenatal group apply, except inclusion criteria 2.

Exclusion Criteria

For exclusion, one is enough to exclude.

 

a)  Matched historical controls

  1. Existence of other disorder that might interfere with the trial

  2. Abnormal karyotype 

 

b) Prospective untreated controls

Postnatal exclusion: The exclusion criteria, except exclusion criterium 4,5,6 and 7 (Contraindication for invasive procedure, known risk factor for clotting, Positive Donor Specific Antibody-test and Known allergy/hypersensitivity to Fungizone and/or Gensumycin), for the postnatal group apply.

Prenatal exclusion: The exclusion criteria, except exclusion criterium 1, 4, 5, 6 and 7 (Multiple pregnancy, Contraindication for invasive procedure, known risk factor for clotting, Positive Donor Specific Antibody-test and Known allergy/hypersensitivity to Fungizone and/or Gensumycin), for the prenatal group apply

Go back

This project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement 681045

© 2019 BOOSTB4 Consortium