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Effectiveness and Safety of Early Intramuscular Botulinum Toxin Injections to Prevent Shoulder Deformity in Babies With Obstetrical Brachial Plexus Palsy (POPB-TOX)

Primary Purpose

Obstetrical Brachial Plexus Palsy

Status
Recruiting
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Botulinum toxin type A injection
Sham
Sponsored by
University Hospital, Brest
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Obstetrical Brachial Plexus Palsy focused on measuring physiotherapy, children, shoulder deformation, obstetrical brachial plexus palsy

Eligibility Criteria

10 Months - 11 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male and female babies with unilateral OBPP
  • Age between 10 and 11 months
  • Presenting one of 2 risk factors for posterior subluxation of the humeral head (10° less passive external ROM of the affected shoulder compared with the contralateral shoulder and/or a score strictly less than 6 on the AMS for shoulder external rotation and abduction, elbow flexion or supination)
  • Signature of the consent form by (the) parent(s) over the age of majority

Exclusion Criteria:

  • Bilateral OBPP
  • Microsurgery or secondary muscle surgery planned between 12 and 18 months of age
  • Contraindications to the use of botulinum toxin
  • Contraindications to MRI
  • MRI not possible in the Paediatric Day Hospital setting because of contraindications to the sedation protocol or due to organisational constraints
  • Parents inapt to provide consent for the participation of their child
  • Parents under the age of 18 years

Sites / Locations

  • CHRU BrestRecruiting
  • Institut Régionnal de Réadaptation Centre de Réadaptation pour enfant
  • ESEAN (Etablissement de Santé pour Enfants et Adolescents de la région Nantaise)
  • CHU NîmesRecruiting
  • CHU RennesRecruiting
  • CHU St Etienne
  • Hôpital national de saint mauriceRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

Toxin group

Sham group

Arm Description

The babies receive a botulinum toxin type A injection at the age of 12 months

The babies receive a simulated injection procedure at the age of 12 months

Outcomes

Primary Outcome Measures

Change in the percentage posterior migration of the humeral head measured on axial MRI slices between 11 (before the BTI carried out at 12 months) and 18 months of age (6 months post BTI).

Secondary Outcome Measures

compare the effectiveness of BTI with Sham procedure in preventing an increase in glenoid retroversion and three-dimensional deformity
With 2D glenoid changes measured on axial MRI and 3D glenoid version and migration of the humeral head measured on MRI
compare the effectiveness of BTI with Sham procedure in the improvement of active and passive joint range of motion and upper limb function
By measurement of passive range of motion, Active Movement Scale and Assistive Hand Assessment.
confirm good clinical tolerance of BTI treatment
By measurement of the number of serious and non-serious adverse events
evaluate the effects of BTI on trophicity, fibrosis and fatty infiltration of the injected muscles as well as muscle balance of the OBPP shoulder
Bu measurement of the degree of trophicity, fibrosis and fatty infiltration of the injected muscles (supraspinous, infraspinous, teres minor, subscapularis, teres major, pectoralis major, deltoid and latissimus dorsi)
determine if the treatment changes the frequency and type of surgical interventions in the long term
the number and type of surgical interventions undergone by the children in each group will be recorded during routine medical follow-up (as in usual practice) until the child's 10th birthday following unblinding (9 years and 6 months after the BTI).

Full Information

First Posted
June 14, 2017
Last Updated
December 16, 2020
Sponsor
University Hospital, Brest
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1. Study Identification

Unique Protocol Identification Number
NCT03198702
Brief Title
Effectiveness and Safety of Early Intramuscular Botulinum Toxin Injections to Prevent Shoulder Deformity in Babies With Obstetrical Brachial Plexus Palsy
Acronym
POPB-TOX
Official Title
Effectiveness and Safety of Early Intramuscular Botulinum Toxin Injections to Prevent Shoulder Deformity in Babies With Obstetrical Brachial Plexus Palsy
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Recruiting
Study Start Date
May 17, 2018 (Actual)
Primary Completion Date
April 2025 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Brest

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
In children population with obstetrical brachial plexus palsy (OBPP), shoulder musculoskeletal deformity is the main cause of morbidity, with a loss of range of shoulder motion, pain and a reduction in social participation. Some uncontrolled studies shows that early injections of botulinum toxin (BTI) in the internal shoulder rotator muscles (which cause the deformity) are one of the most promising treatment for the prevention of bony deformity. The main objective of this study will be the evaluation of the effectiveness of BTI in the internal shoulder rotator muscles at the age of 12 months in preventing an increase in posterior subluxation of the glenohumeral joint in babies with OBPP (evaluated at the ages of 11 months and 18 months), compared to the Sham group.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Obstetrical Brachial Plexus Palsy
Keywords
physiotherapy, children, shoulder deformation, obstetrical brachial plexus palsy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
First group or "toxin" group: the babies will receive BOTOX injections at the age of 12 months in the shoulder muscles. Second group or "sham" group:the babies receive the same procedure but no injection.
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
62 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Toxin group
Arm Type
Experimental
Arm Description
The babies receive a botulinum toxin type A injection at the age of 12 months
Arm Title
Sham group
Arm Type
Sham Comparator
Arm Description
The babies receive a simulated injection procedure at the age of 12 months
Intervention Type
Drug
Intervention Name(s)
Botulinum toxin type A injection
Intervention Description
A total of 8UI/kg will be injected in the internal shoulder rotator muscles: 2UI/kg in the sub scapular muscle, 3UI/kg in the pectoralis major muscle and 3UI/kg in the teres major/latissimus dorsi muscle.
Intervention Type
Other
Intervention Name(s)
Sham
Intervention Description
The injection is mimed, the procedure is the same as the botulinum toxin injection.
Primary Outcome Measure Information:
Title
Change in the percentage posterior migration of the humeral head measured on axial MRI slices between 11 (before the BTI carried out at 12 months) and 18 months of age (6 months post BTI).
Time Frame
At 18 month age
Secondary Outcome Measure Information:
Title
compare the effectiveness of BTI with Sham procedure in preventing an increase in glenoid retroversion and three-dimensional deformity
Description
With 2D glenoid changes measured on axial MRI and 3D glenoid version and migration of the humeral head measured on MRI
Time Frame
At 18 month age
Title
compare the effectiveness of BTI with Sham procedure in the improvement of active and passive joint range of motion and upper limb function
Description
By measurement of passive range of motion, Active Movement Scale and Assistive Hand Assessment.
Time Frame
At 18 month age
Title
confirm good clinical tolerance of BTI treatment
Description
By measurement of the number of serious and non-serious adverse events
Time Frame
At 18 month age
Title
evaluate the effects of BTI on trophicity, fibrosis and fatty infiltration of the injected muscles as well as muscle balance of the OBPP shoulder
Description
Bu measurement of the degree of trophicity, fibrosis and fatty infiltration of the injected muscles (supraspinous, infraspinous, teres minor, subscapularis, teres major, pectoralis major, deltoid and latissimus dorsi)
Time Frame
At 18 month age
Title
determine if the treatment changes the frequency and type of surgical interventions in the long term
Description
the number and type of surgical interventions undergone by the children in each group will be recorded during routine medical follow-up (as in usual practice) until the child's 10th birthday following unblinding (9 years and 6 months after the BTI).
Time Frame
every years on 2 years old to 10 years old

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Months
Maximum Age & Unit of Time
11 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male and female babies with unilateral OBPP Age between 10 and 11 months Presenting one of 2 risk factors for posterior subluxation of the humeral head (10° less passive external ROM of the affected shoulder compared with the contralateral shoulder and/or a score strictly less than 6 on the AMS for shoulder external rotation and abduction, elbow flexion or supination) Signature of the consent form by (the) parent(s) over the age of majority Exclusion Criteria: Bilateral OBPP Microsurgery or secondary muscle surgery planned between 12 and 18 months of age Contraindications to the use of botulinum toxin Contraindications to MRI MRI not possible in the Paediatric Day Hospital setting because of contraindications to the sedation protocol or due to organisational constraints Parents inapt to provide consent for the participation of their child Parents under the age of 18 years
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sylvain Brochard, Pr
Phone
(+33) 02 98 22.33.73
Email
sylvain.brochard@chu-brest.fr
Facility Information:
Facility Name
CHRU Brest
City
Brest
ZIP/Postal Code
29609
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sylvain BROCHARD, Pr
Phone
02 98 22 33 73
First Name & Middle Initial & Last Name & Degree
Chritelle PONS, Dr
Phone
02 98 22 33 73
First Name & Middle Initial & Last Name & Degree
Sylvain BROCHARD, Pr
First Name & Middle Initial & Last Name & Degree
Christelle PONS, Dr
Facility Name
Institut Régionnal de Réadaptation Centre de Réadaptation pour enfant
City
Flavigny-sur-Moselle
ZIP/Postal Code
54630
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jennifer BASTIEN, Dr
Phone
03 54 59 19 33
Email
jennifer.bastien@ugecam.assurance-maladie.fr
First Name & Middle Initial & Last Name & Degree
Fanny DALMONT, Dr
Phone
03 54 59 19 50
Email
fanny.dalmont@ugecam.assurance-maladie.fr
First Name & Middle Initial & Last Name & Degree
Jennifer BASTIEN, Dr
First Name & Middle Initial & Last Name & Degree
Fanny DALMONT, Dr
Facility Name
ESEAN (Etablissement de Santé pour Enfants et Adolescents de la région Nantaise)
City
Nantes
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Guy LETELLIER, Dr
Email
g.letellier@esean.fr
First Name & Middle Initial & Last Name & Degree
Guy LETELLIER, Dr
Facility Name
CHU Nîmes
City
Nîmes
ZIP/Postal Code
30029
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mélanie PORTE, Dr
Phone
04 66 68 68 68
First Name & Middle Initial & Last Name & Degree
Mélanie PORTE, Dr
Facility Name
CHU Rennes
City
Rennes
ZIP/Postal Code
35033
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe VIOLAS, Dr
Phone
02 99 28 43 21
First Name & Middle Initial & Last Name & Degree
Philippe VIOLAS, Dr
First Name & Middle Initial & Last Name & Degree
Helene RAUSCENT, Dr
First Name & Middle Initial & Last Name & Degree
Floriane COLIN, Dr
Facility Name
CHU St Etienne
City
Saint Etienne
ZIP/Postal Code
42055
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Madeleine ASLAN, Dr
Phone
04 77 82 80 37
Email
madeleine.aslan@chu-st-etienne.fr
First Name & Middle Initial & Last Name & Degree
Vincent GAUTHERON, Pr
Phone
04 77 82 80 00
Email
vincent.gautheron@chu-st-etienne.fr
First Name & Middle Initial & Last Name & Degree
Vincent GAUTHERON, Pr
First Name & Middle Initial & Last Name & Degree
Madeleine ASLAN, Dr
First Name & Middle Initial & Last Name & Degree
Bruno DOHIN, Pr
Facility Name
Hôpital national de saint maurice
City
Saint-Maurice
ZIP/Postal Code
94410
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nathaly QUINTERO, Dr
Phone
01 43 96 63 50
Email
n.quintero@hopitaux-st-maurice.fr
First Name & Middle Initial & Last Name & Degree
Anne-Gaëlle PY, Dr
Email
ag.py@hopitaux-st-maurice.fr
First Name & Middle Initial & Last Name & Degree
Nathaly QUINTERO, Dr
First Name & Middle Initial & Last Name & Degree
Anne-Gaëlle PYDr, Dr
First Name & Middle Initial & Last Name & Degree
Katherine SANCHEZ, Dr

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
31575585
Citation
Pons C, Eddi D, Le Gal G, Garetier M, Ben Salem D, Houx L, Fitoussi F, Quintero N, Brochard S; POPB-TOX Group. Effectiveness and safety of early intramuscular botulinum toxin injections to prevent shoulder deformity in babies with brachial plexus birth injury (POPB-TOX), a randomised controlled trial: study protocol. BMJ Open. 2019 Sep 30;9(9):e032901. doi: 10.1136/bmjopen-2019-032901.
Results Reference
derived

Learn more about this trial

Effectiveness and Safety of Early Intramuscular Botulinum Toxin Injections to Prevent Shoulder Deformity in Babies With Obstetrical Brachial Plexus Palsy

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